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Rare Diseases and Orphan Products: Accelerating Research and Development (2010)

Chapter:Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs

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Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

C
Medicare Part D Coverage and Reimbursement of Orphan Drugs

Laura Faden and Haiden Huskamp*

INTRODUCTION

Given the small potential market for medications that treat rare conditions, pharmaceutical manufacturers may have reduced incentives to develop new medications for rare diseases. To increase incentives for manufacturers, the Orphan Drug Act (P.L. 97-414) provides the following provisions for drugs that receive an orphan drug indication1 from the U.S. Food and Drug Administration (FDA): a 7-year period of market exclusivity, a tax credit of 50 percent of the cost of conducting clinical trials, eligibility for federal research grants, and a waiver of user fees (21 USC 360bb, OIG, 2001). However, health plan coverage and reimbursement also influence a pharmaceutical firm’s decisions to invest in the development of a drug or biologic for a rare disease.

The purpose of this report is to examine stand-alone Medicare Part D prescription drug plan (PDP) coverage of a set of drugs and biologics that

*

Laura Faden., M.P.H., is a doctoral student in the Harvard University Program in Health Policy. Haiden Huskamp, Ph.D., is Professor of Health Care Policy in the Department of Health Care Policy at Harvard Medical School. Responsibility for the content of this paper rests with the author and does not necessarily represent the views of the Institute of Medicine or its committees and convening bodies.

1

The Orphan Drug Act of 1983 defines an orphan indication as follows: “in the case of a drug, any disease or conditions which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug” (21 USC 360bb).

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

treat rare diseases or conditions—which we will refer to collectively as “orphan drugs”—in the Medicare population. This report does not address Medicare coverage and reimbursement of medical devices.

We focus on the Medicare population because the program covers approximately 15 percent of the U.S. population, including adults who have a disabling rare condition and who have Medicare coverage based on their qualification for Social Security Disability Insurance (SSDI). Furthermore, data on Medicare prescription drug coverage and reimbursement are more readily available than similar data from Medicaid and private plans—because Medicare is a public, federal program, the data are public and centrally collected.

There have been no comprehensive studies of Medicare PDP coverage and reimbursement of orphan drugs. In 2005, the National Organization for Rare Disorders (NORD) conducted a similar study that examined coverage of orphan drugs in 10 national Medicare Part D plans (NORD, 2006). This report extends the NORD report by including drugs approved since 2005 and by analyzing coverage of all Medicare prescription drug plans. Furthermore, this report goes beyond the NORD analysis, which only analyzed plan coverage, by also analyzing factors that may reduce access to covered drugs (i.e., formulary tier placement, utilization management).2

Medicare Beneficiaries

Medicare, which was created by the Social Security Act of 1965, is a federally administered health insurance program for people who are 65 years of age or older or who qualify for SSDI. There is typically a two-year waiting period before people who qualify for SSDI can receive Medicare benefits. Congress has waived that requirement for people with end-stage renal disease or Lou Gehrig’s disease.

As of January 2010, Medicare covers 46 million Americans, 17 percent of whom are under 65 years and are permanently disabled (KFF, 2010c). Almost half (47 percent) of Medicare beneficiaries have low income (below 200 percent poverty), and 7 million beneficiaries meet income and asset criteria to quality for Medicaid—these beneficiaries are known as “dual-eligibles.” Among the Medicare population, there is a high prevalence of comorbid conditions (44 percent suffer from three or more chronic conditions), and 29 percent have a cognitive or mental impairment (KFF, 2010c).

It is not known how many Medicare beneficiaries have a rare disease or, conversely, what proportion of people with a rare disease is covered by Medicare. However, the Social Security Compassionate Allowances program—

2

Although the NORD report notes the tier placement and utilization management tools used for each drug, the authors do not provide any analysis of these aspects of drug coverage.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

which guarantees immediate SSDI benefits for people who suffer from certain conditions—covers many rare diseases (Social Security Online, 2010).

Currently, more than 27 million Medicare beneficiaries are enrolled in a Medicare prescription drug plan, two-thirds of whom are enrolled in a stand-alone PDP (KFF, 2009d, 2010b).3 In 2009, 36 percent of these beneficiaries received low-income subsidies (LIS) that cover their premiums and deductibles; LIS beneficiaries are responsible only for a small copayment that is determined by their income level (KFF, 2009a). Dual-eligibles and those eligible for Supplemental Security Income cash assistance are automatically eligible for LIS, and other low-income beneficiaries can apply for the subsidies. All LIS beneficiaries are enrolled in plans that have monthly premiums below the benchmark premium amount established for each region (hereafter referred to as “benchmark plans”).

Medicare Prescription Drug Plans

Until 2006, Medicare did not cover outpatient prescription drugs. It covered hospital and physician services (Part A and B), which included coverage of inpatient drugs and drugs administered by a physician (e.g., infusions). The Balanced Budget Act of 1997 created an option for Medicare beneficiaries to receive insurance coverage from private health plans that contract with Medicare (Part C)—these plans are currently referred to as “Medicare Advantage Plans.” The Medicare Prescription Drug Improvement and Modernization Act of 2003 created the Medicare Part D program, a voluntary drug benefit that is administered through private health plans or pharmaceutical benefit managers. As of January 1, 2006, Medicare beneficiaries could voluntarily enroll in either a stand-alone PDP or a Medicare Advantage plan with prescription drug coverage (MA-PD). Dual-eligibles are automatically enrolled in a benchmark plan.

The legislation does not require PDPs to have uniform cost sharing requirements or formulary design. However, PDPs are required to offer a plan that is at least actuarially equivalent to a standard benefit package as determined by the Centers for Medicare and Medicaid Services (CMS) (CMS, 2010). In 2010, the standard benefit package is

  • $310 deductible;

  • 25 percent coinsurance up to $2,830 of total drug costs;

3

Of the remaining Medicare beneficiaries not covered by a Part D plan—approximately 19 million—most have drug coverage, either through a retiree drug plan (through an employer or union) or another form of drug coverage (e.g., Veterans Affairs, Indian Health Services, state pharmacy assistance programs, employer benefits for active workers, etc.). Approximately 10 percent of beneficiaries have no prescription drug coverage (KFF, 2009d, 2010b).

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
  • no coverage from $2,830 to $6,330 of total drug costs—a coverage gap that is commonly referred to as the “doughnut hole,” which, starting in 2010, will be partially subsidized (beneficiaries will receive a $250 rebate);4 and

  • 5 percent coinsurance, or a flat copayment of $2.50 for a generic drug and $6.30 for a brand drug, above $4,550 out-of-pocket expenses (i.e., catastrophic out-of-pocket spending limit) with no maximum limit on out-of-pocket expenses.

In addition, CMS requires that PDP and MA-PD formularies include at least two drugs in every drug class5 and all, or substantially all, drugs in the following six “protected” therapeutic categories: antidepressants; antipsychotics; anticonvulsants; immunosuppressants (to prevent rejection of organ transplants); antiretrovirals (for the treatment of infection by retroviruses, primarily HIV); and antineoplastics (only those chemotherapy drugs that are generally are not covered under Medicare Part B) (CMS, 2010).

Even with these requirements, PDPs and MA-PDs have a considerable amount of flexibility in formulary design. First, plans decide whether or not to cover a drug. Second, plans can use a tiered formulary structure to create financial incentives for beneficiaries to choose lower-cost or preferred drugs. In 2010, approximately three-fifths of plans have the following fourtier structure (KFF, 2009c).

  • Tier 1: generic drugs

  • Tier 2: preferred brand-name drugs

  • Tier 3: nonpreferred brand-name drugs

  • Tier 4 (“specialty tier”): specialty drugs6

Each tier has a different cost sharing requirement—most plans assign a flat copayment to the first three tiers and a coinsurance to the specialty tiers (although a growing number of plans are requiring a coinsurance for the first three tiers) (KFF, 2009c). Almost all (94 percent) of plans have a

4

The Patient Protection and Affordable Care Act of 2010 (P.L. 111-148) included provisions to reduce cost sharing in the doughnut hole. Starting in 2011, Medicare and manufacturers will phase in subsidies for generic and brand drugs with the goal of reducing out-of-pocket expenditure in the doughnut hole in 2010 to the same 25 percent coinsurance that applies to costs below the lower threshold of the coverage gap.

5

The U.S. Pharmacopeia has developed a therapeutic classification system that serves as a guideline for Part D formularies. Model guidelines are publicly available: see http://www.usp.org/pdf/EN/mmg/modelGuidelinesV4.0WithFKDTs.pdf.

6

CMS guidelines stipulate that drugs placed on the specialty tier must cost at least $600 per month and prohibit enrollees from requesting cost sharing exceptions for specialty drugs (CMS, 2009b).

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

specialty tier (KFF, 2009c).7 In 2010, the specialty tier coinsurance ranges from 25 to 33 percent of the full cost of the drug (KFF, 2009c).

In addition to coverage decisions and tiered formularies, a third way in which plans influence prescription drug utilization is by employing utilization management tools such as prior authorization (PA) requirements, step therapy (ST) requirements, and quantity limits (QL). PA requirements create an administrative barrier to accessing a drug—a patient or provider must follow a certain procedure, created by the plan, to request coverage of the drug and then await the plan’s approval of coverage. ST requirements establish a chronological course of recommended treatments for a condition that must be tried before coverage of the drug is approved. QL requirements set explicit criteria for the quantity of a drug that will be covered during a given period of time.

METHODS

Medicare Part D Plans Characteristics and Orphan Drug Coverage

We used the CMS Formulary and Pharmacy Network Information File (January 2010 quarterly release) to determine the coverage, tier placement, and utilization management requirements for each orphan drug. We classified drug plans as stand-alone drug plans (PDPs) and MA-PD plans.8 Within PDPs, we identified national plans (i.e., plans offered in every region of the country) and benchmark plans. National plans were identified by contract number (CMS, 2009b), and benchmark plans were identified using the regional premium limits (CMS, 2009a).

We calculated the “plan coverage rate” for a particular drug as the percentage of plans that cover the drug. Similar to the NORD report (NORD, 2006), we categorized each drug by level of coverage rate, which we classified as the following:

  • No or low coverage: <25 percent plan coverage rate

  • Low coverage: 25-49 percent plan coverage rate

  • Medium coverage: 50-74 percent plan coverage rate

  • High coverage: 75-99 percent plan coverage rate

  • Complete coverage: 100 percent plan coverage rate

We also examined tier placement and utilization management among drugs covered by PDPs. For each drug we calculated the “tier placement

7

This excludes plans that offer the standard benefit package (i.e., plans that have no tiering).

8

MA-PDs include both regional and local plans. However, for the analyses we removed duplicate MA-PDs by including each unique contract and plan combination only once.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

rate” as the percentage of plans that cover that drug on a given tier. For example, if 20 percent of the plans that cover a drug have placed that drug on tier 4, the drug has a tier 4 placement rate of 20 percent. The tiers range from one to four. A few plans have more than four tiers—for these plans we included all tiers greater than four in the tier 4 category.9

Similarly, we examined rates of step therapy requirements, quantity limits, and prior authorization requirements (“utilization management rate”).10 We categorized each covered drug by the rate of tier placement and use of utilization management, which we classified into the following categories:

  • No or low placement (or use): <25 percent of plans

  • Low placement (or use): 25-49 percent of plans

  • Medium placement (or use): 50-74 percent of plans

  • High placement (or use): 75-99 percent of plans

  • Complete placement (or use): 100 percent of plans

In terms of beneficiary access to drugs, a higher plan coverage rate will likely improve access, whereas a higher utilization management or tier 4 placement rate may pose barriers to access. These categories are subjective and were created only to simplify the interpretation of the results for all drugs along the three dimensions of access (i.e., plan coverage, tier placement, and utilization management). Therefore we also report the raw rates.

9

For the purposes of this report, we refer to tiers 4-6 as “tier 4” and assume that tier 4 is equivalent to a specialty tier. However, given the heterogeneity of the PDPs’ formulary structures, this assumption does not hold for all plans. As previously noted, three-fifths of the plans have a four tier structure—for these plans tier 4 is the specialty tier (CMS, 2009b). Some plans have no tiers (11 percent) or fewer than four tiers (7 percent)—for the latter, the specialty tier may actually be tier 3. Also, 21 percent of the plans have more than four tiers—for these plans, tier 4 may not be a specialty tier but rather a nonpreferred brand tier (CMS, 2009b) or a tier for injectible drugs (KFF, 2010a). Therefore, by collapsing tiers 4-6 and labeling this as a specialty tier, we may be misclassifying up to 28 percent of the plans. However, this misclassification may have a negligible effect in terms of concerns about beneficiary cost sharing because a large share (34 percent) of PDPs now use coinsurance rates for nonpreferred brand tiers (CMS, 2009b) and these coinsurance rates may actually be higher than specialty-tier coinsurance rates. Likewise, cost sharing for drugs placed in an injectible tier is also likely to be high because these drugs are quite expensive.

10

Note that for these analyses, the rates are based on only the number of plans that cover each drug (i.e., the denominator is different for each drug). Since each drug is associated with multiple entries in the National Drug Code (NDC) directory, a drug may appear on multiple tiers of a plan’s formulary (e.g., both the brand and generic version are covered, but the generic is on lower tier). For the purposes of our analyses, we assign drugs to the lowest tier on which they appear. A plan is counted as having a utilization management tool for a drug if the tool is applied to at least one of the covered NDCs associated with the drug.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

To determine if coverage policies differed by type of plan, we repeated these analyses for several subgroups of plans: all PDPs, benchmark PDPs, national PDPs and MA-PDs. Although we present data on all analyses, we focus on the analyses of all PDPs in the results and discussion sections.

List of Orphan Drugs

We created a list of drugs that were approved by the FDA with an orphan indication between 1983 and December 2008. We included only drugs approved prior to 2009 in order to provide adequate time for marketing and plan coverage decisions. We included only outpatient drugs (i.e., not covered by Medicare Part B as of July 2010) that have an approved orphan indication relevant to the Medicare population (i.e., not for a pediatric indication11) and that have not been discontinued or withdrawn.

Our list excludes drugs that are covered by Medicare Part A or B and blood products. We also excluded drugs that are available on the market (for other FDA-approved indications) and that have been granted an orphan designation but have not yet received FDA approval for an orphan indication. Lastly, we excluded drugs for the treatment of rare diseases or conditions that appear in Medicare compendia unless the FDA has approved the drugs for the same orphan indication (see Chapter 6 of this report).

The National Drug Codes (NDCs) for each drug were obtained from First Data Bank (FDB), which was up-to-date as of January 2010. We noted which drugs are available in generic form and which are biologics versus new chemical entities.

RESULTS

List of Orphan Drugs

Ninety-nine orphan drugs met our inclusion criteria (see Addendum Table C-A1). Drugs are listed in chronological order of the date of approval of the first orphan indication relevant to the Medicare population (some drugs have multiple relevant orphan indications). Twenty-nine (29 percent) of the drugs are available in generic form and eleven (11 percent) are biologics.

Medicare Plan Characteristics—Part D and Medicare Advantage Plans

In 2010, there are 1,620 stand-alone PDPs and 2,418 MA-PDs. Of the PDPs, 1,295 (77 percent) are national plans and 398 (23 percent) are

11

We performed a separate analysis of Medicare Part D coverage of orphan drugs with only a pediatric indication orphan approval. See Addendum Tables C-A3 and C-A4.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

benchmark plans. The 1,295 national plans represent 12 plan sponsor organizations, 26 unique contracts, and 88 unique formularies (a sponsor may use the same formulary for multiple plans).

The average monthly premium is $46.39 (range: $1.50 to $120.20; standard deviation: $19.75) (see Table C-1). More than half (60 percent) of the plans have deductibles. The median deductible for all plan types is $310.00 (range: $10.00 to $310.00). Benchmark plans and nonnational plans are more likely to have a deductible and to have a higher deductible than nonbenchmark and national plans. Compared to stand-alone PDPs, MA-PDs have a lower average premium and are less likely to have a deductible.

Medicare Plans’ Coverage of Orphan Drugs— Stand-Alone PDPs and MA-PDs

The coverage rate (percentage of plans covering a drug) for orphan drugs among Medicare prescription drug plans is high. On average, an orphan drug is covered by 84 percent (standard deviation: 24 percent) of stand-alone PDPs. Table C-2 shows a breakdown of coverage rate category by plan type. Of the 99 drugs, 44 (44 percent) are covered by all 1,620 PDPs (i.e., complete coverage category). An additional 29 drugs (29 percent) are covered by at least 75 percent of the plans (i.e., high-coverage category).

Table C-2 shows that 19 (19 percent) of the drugs fall into the medium-coverage category (i.e., only covered by 50-75 percent of plans) and that 7 (7 percent) are covered by less than half of the plans (i.e., no or very low coverage and low-coverage categories). As of January 2010 4 drugs are not covered by any PDP: citric acid, glucono-delta-lactone, and magnesium car-

TABLE C-1 Average Premium and Use of Deductible for Different Types of Medicare Prescription Drug Plans (99 drugs)

 

N

Average Premium (std. dev.)

% with Deductiblea

All stand-alone PDPs

1,620

46.39 (19.75)

60

Benchmark PDPs

398

28.70 (5.69)

94

Nonbenchmark PDPs

1,222

52.15 (19.27)

49

National PDPs

1,295

46.70 (20.14)

57

Nonnational PDPs

325

45.15 (18.07)

72

MA-PDs

2,418

20.12 (18.81)

23

a The median deductible across all plan types is $310.

NOTE: 2010 Data.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-2 Orphan Drug Coverage by Type of Medicare Prescription Drug Plan (99 drugs)

 

All Stand-alone PDPs (N)

MA-PDPs (N)

Stand-alone National PDPs (N)

Stand-alone Non-national PDPs (N)

Stand-alone Benchmark PDPs (N)

Stand-alone Non-benchmark PDPs (N)

No or very low coverage (<25% plan coverage rate)

4

4

4

10

4

4

Low coverage (25-49% plan coverage rate)

3

0

0

13

7

2

Medium coverage (50-74% plan coverage rate)

19

17

19

8

15

19

High coverage (75-99% plan coverage rate)

29

36

19

25

24

29

Complete coverage (100% plan coverage rate)

44

42

57

43

49

45

NOTE: Number of drugs that fall into each coverage rate category. Because the number of drugs in the analysis is 99, the numbers and percentages of drugs are identical; the percentages have therefore not been included in the table.

bonate (Renacidin Irrigation); clofazimine (Lamprene); glutamine (Nutrestore); zinc acetate (Galzin). Three other drugs—lodoxamide tromethamine (Alomide Ophthalmic Solution), tinidazole (Tindamax), and metronidazole topical (Metrogel)—are covered by 45 to 50 percent of PDPs (see Table C-3). As explained in the note for the table, a search of formularies conducted in late spring 2010 found a few plans had initiated coverage of Galzin and Renacidin Irrigation.

Overall, MA-PD plans have slightly better coverage of orphan drugs than stand-alone PDPs. Compared to PDPs, the percentage of drugs falling into the no-very low and low-coverage categories is slightly lower among MA-PDs (4 percent in MA-PDPs versus 7 percent in PDPs). On average, an orphan drug is covered by 87 percent (standard deviation: 22 percent) of MA-PDPs, compared to 84 percent of stand-alone PDPs.

Within PDPs, there is some variation in coverage rates between benchmark and nonbenchmark plans, with nonbenchmark plans having slightly higher coverage rates. On average, an orphan drug is covered by 83 percent (standard deviation: 26 percent) of benchmark plans and 85 percent (standard deviation: 24 percent) of nonbenchmark plans. The benchmark plans have a higher percentage of drugs that fall within the no-very low

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-3 Orphan Drugs with No, Very Low, or Low Plan Coverage (less than 50% coverage among all standalone PDPs) (7 drugs)

Generic Name

Trade Name

Stand-alone PDP Coverage (% plans that cover drug)

Route of Administration

Orphan Designation(s) (year of orphan approval)

Citric acid, glucono-deltalactone, and magnesium carbonate

Renacidin Irrigation

0a

Irrigation

Treatment of renal and bladder calculi of the apatite or struvite variety (1990)

Clofazimine

Lamprene

0

Oral

Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum (1986)

Glutamine

Nutrestore

0

Oral

For use with human growth hormone in the treatment of short bowel syndrome (nutrient malabsorption from the gastrointestinal tract resulting from an inadequate absorptive surface) (2004)

Zinc acetate

Galzin

0a

Oral

Treatment of Wilson’s disease (1997)

Lodoxamide tromethamine

Alomide Ophthalmic Solution

46

Ophthalmic

Treatment of vernal keratoconjunctivitis (1993)

Tinidazole

Tindamax

47

Oral

(1) Treatment of giardiasis; (2) treatment of amebiasis (2004)

Metronidazole (topical)

Metrogel

49

Topical

Treatment of acne rosacea (1988)

a These drugs had 0% coverage according to our analysis, which was limited to coverage of the drugs’ NDCs (listed in the FDB) in the January determined that Renacidin and Galzin are in fact covered by some PDPs as of June 2010. Galzin, which was a “high priority access problem drug” in the NORD analysis, is not on any national PDP formularies but appears on at least two nonnational formularies. Renacidin also appears on two national formularies and a few nonnational PDP formularies.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

and low-coverage categories (11 percent in benchmark versus 6 percent in nonbenchmark plans).

There is considerably more variation in coverage rates between national and nonnational plans, with national plans having higher coverage rates. On average, an orphan drug is covered by only 77 percent (standard deviation: 32 percent) of nonnational plans, compared to 86 percent (standard deviation: 24 percent) of national plans. Within nonnational plans, almost a quarter (23 percent) of the drugs are classified as having a no-very low or low-coverage rate, compared to only 4 percent of drugs within national plans. Aside from the four drugs covered by no PDPs, no other drug is covered by less than 50 percent of the national plans. Conversely, 19 of the 95 covered drugs are covered by less than 50 percent of the nonnational plans.

Formulary Tier Placement by Stand-Alone PDPs

The orphan drugs are commonly placed on high cost sharing tiers. Table C-4 shows the tier 4 placement rate by plan type. For these analyses, and the utilization management analyses below, we excluded the four drugs not covered by any plan. Of the 95 remaining drugs, 84 (88 percent) are placed on tier 4 or higher by at least one PDP. Twenty-eight (29 percent) are placed on tier 4 by at least 75 percent of the plans (i.e., high tier 4 placement), and another 15 (16 percent) are placed on tier 4 by at least 50 percent of the plans (i.e., medium tier 4 placement).

Utilization Management Tools Used by Stand-Alone PDPs

PDPs rarely use step therapy to manage orphan drugs. ST is used by at least one plan only for 18 (19 percent) of the covered orphan drugs. Of these 18 drugs, half (9) have a ST use rate of less than 10 percent. The drug with the highest use of ST—interferon beta-1b—is given ST requirements by almost one-quarter (23 percent) of PDPs. The use of quantity limits to manage utilization of orphan drugs is more common among PDPs than the use of ST, although most plans do not use quantity limits for these drugs. QLs are used by at least one plan for 57 (60 percent) of the covered drugs. Twenty-seven (28 percent) of these drugs have QL use rates greater than 20 percent, and an additional 4 drugs (4 percent) have QL use rates greater than 50 percent (interferon beta-1a, lidocaine patch, raloxifene, and modafinil).

Prior authorization is the most widely used form of utilization management employed by PDPs. Table C-5 shows PA rates by plan type. PA is used by at least one plan for 80 (84 percent) of the covered orphan drugs. Thirty-three (35 percent) of the drugs are given a PA requirement by at least

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-4 Orphan Drugs by Rate of Tier 4 Placement and Type of Medicare Prescription Drug Plan (95 drugs)

 

Stand-alone PDPs N (%)

MA-PDPs N (%)

Stand-alone National PDPs N (%)

Stand-alone Non-national DPs N (%)

Stand-alone Benchmark PDPs N (%)

Stand-alone Non-benchmark PDPs N (%)

No or very low tier 4 placement (<25% plans)

47 (49)

52 (55)

47 (49)

43 (45)

49 (52)

47 (49)

Low tier 4 placement (25-49% plans)

5 (5)

2 (2)

5 (5)

12 (13)

4 (4)

5 (5)

Medium tier 4 placement (50-74% plans)

15 (16)

8 (8)

12 (13)

29 (31)

35 (37)

12 (13)

High tier 4 placement (75-99% plans)

28 (29)

33 (35)

31 (33)

11 (12)

7 (7

31 (33)

Complete tier 4 placement (100% plans)

0 (0)

0 (0)

0 (0)

0 (0)

0 (0)

0 (0)

NOTE: Number (percentage) of drugs that fall into each coverage rate category; excludes 4 drugs not covered by any plan.

TABLE C-5 Orphan Drugs by Rate of Prior Authorization Use and Type of Medicare Prescription Drug Plan (95 drugs)

 

Stand-alone PDPs N (%)

MA-PDPs N (%)

Stand-alone National PDPs N (%)

Stand-alone Non-national PDPs N (%)

Stand-alone Benchmark PDPs N (%)

Stand-alone Non-benchmark PDPs N (%)

No or very low use of PA (<25% plans)

47 (49)

52 (55)

46 (48)

56 (59)

45 (47)

48 (51)

Low tier use of PA (25-49% plans)

15 (16)

13 (14)

15 (16)

19 (20)

14 (15)

17 (18)

Medium use of PA (50-74% plans)

19 (20)

17 (18)

15 (16)

14 (15)

18 (19)

16 (17)

High use of PA (75-99% plans)

14 (15)

13 (14)

9 (9)

6 (6)

13 (14)

14 (15)

Complete use of PA (100% plans)

0 (0)

0 (0)

10 (11)

0 (0)

5 (5)

0 (0)

NOTE: Number (percentage) of drugs that fall into each coverage rate category; excludes 4 drugs not covered by any plan.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

half of the PDPs (i.e., medium and high use of PA). Ten drugs are given a PA requirement by at least 90 percent of the PDPs, and 5 of these are given a PA requirement by 99 percent of the plans (somatropin (R-DNA), somatropin for injection, somatropin, immune globulin (human), and tacrolimus).

The PA use rate and tier 4 placement rate are highly correlated (correlation coefficient = .75). That is, drugs that are more likely to be placed on tier 4 are also more likely to have PA requirements.

Table C-A2 shows coverage rate, tier placement, and utilization management rates by drug. See Tables C-A3 and C-A4 for a list of orphan drugs with only a pediatric orphan indication (N = 27) and the coverage rate, tier placement, and utilization management rates by drug.

DISCUSSION

Medicare beneficiaries’ access to orphan drugs is jointly determined by the following three factors: whether or not the plan covers the drug, the formulary tier the drug is placed on (and the cost sharing requirements associated with each tier), and whether there are any utilization management requirements for the drug.

In terms of the percentage of plans that cover the drugs, Medicare prescription drug plan coverage of orphan drugs is relatively extensive. The majority of drugs have complete coverage (100 percent) or high rates of coverage (>75 percent) among PDPs. The fact that many of these drugs are in protected classes (for either the orphan indication or another approved indication) may explain the high coverage rates of these drugs.

Nonetheless, it is important to emphasize that 26 orphan drugs are covered by less than 75 percent of PDPs, and 4 of these are not covered by any PDP. Also, only 4 of these 26 drugs are available in generic form. If a drug is not covered by a PDP, beneficiaries in that PDP must pay out-of-pocket for the full cost of a brand-name drug unless a lower-cost generic alternative is available.

There is also variation in orphan coverage between types of PDPs—notably, there is much higher coverage in national than nonnational plans. There is also slightly higher coverage among nonbenchmark than benchmark plans.

However, plan coverage alone does not guarantee access—tier placement and utilization management requirements may limit access of covered drugs by imposing financial barriers (e.g., high cost sharing on specialty tiers) or administrative barriers (e.g., paperwork required for PA). We found that PDPs often place covered orphan drugs on a high cost sharing tier and/or require prior authorization. However, we found minimal use of quantity limits or step therapy, the latter of which was expected since there are often few, if any, therapeutic substitutes for these orphan drugs.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Our findings are similar to a recent analysis of tier placement and use of utilization management by national PDPs for 10 common specialty drugs (KFF, 2009b).The authors found that almost all of the PDPs covered the 10 drugs and that 7 of the drugs were placed on a specialty tier by more than 75 percent of the plans. The authors also found high rates of utilization management for these drugs. Four of the 10 drugs analyzed are on our list of orphan drugs—Sensipar, Copaxone, Thalomid, and Tracleer; the last 3 are placed on tier 4 or above by approximately four out of five PDPs in 2010.

Non-low-income subsidy PDP enrollees typically face high levels of out-of-pocket spending for drugs on a specialty tier. In 2009, the national PDP average monthly specialty tier cost sharing amount for these three orphan drugs was $602, $1,512, and $1,916 (for Copaxone, Tracleer, and Thalomid, respectively) (KFF, 2009b). Patients taking these drugs typically reached the catastrophic out-of-pocket payment limit, which was $4,350 in 2009, in less than 3 months for both Thalomid and Tracleer and in 7 months for Copaxone—this is assuming no deductible and no doughnut hole, the latter of which will be partially eliminated with the recent health care reform (KFF, 2010a). After reaching the limit, these patients were then responsible for paying 5 percent of the full cost of the drug—these monthly out-of-pocket payments were an average of $99, $247, and $314 (for Copaxone, Tracleer, and Thalomid, respectively), calculated using data in reference (KFF, 2009b). For beneficiaries in the majority of PDPs, these financial barriers to access were compounded by utilization management requirements, predominantly PA.

When used appropriately, formulary management techniques such as tier placement and PA can improve the appropriate use of drugs and save costs. However, orphan diseases, by definition, have limited treatment options and there may not be a lower-cost therapy available to patients. Although most orphan drugs are covered by PDPs, patients who require drugs that are placed on high cost sharing tiers are forced to either pay large out-of-pocket costs or forgo treatment. The cost-related and utilization management-related nonadherence for orphan drugs among the Medicare population is not known. Also, although the financial barriers are largely removed for those receiving low-income subsidies, it is not known how utilization management requirements affect these beneficiaries’ access to orphan drugs.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

ADDENDUM

TABLE C-A1 Orphan Drugs Relevant to Medicare Population (1983-2008 approvals) (99 drugs)

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

10/3/84

Cromolyn sodium 4% ophthalmic solutionb

Opticrom 4% ophthalmic solution

Treatment of vernal keratoconjunctivitis

11/30/84

Naltrexone HClb

Revia

For blockade of the pharmacological effects of exogenously administered opioids as an adjunct to the maintenance of the opioid-free state in detoxified formerly opioid-dependent individuals

8/30/85

Potassium citrateb

Urocit-K

(1) Prevention of calcium renal stones in patients with hypocitraturia. (2) Prevention of uric acid nephrolithiasis. (3) For avoidance of the complication of calcium stone formation in patients with uric lithiasis.

11/8/85

Trientine HCl

Syprine

Treatment of patients with Wilson’s disease who are intolerant of, or inadequately responsive to, penicillamine

4/10/86

Levocarnitineb

Carnitor

Treatment of genetic carnitine deficiency

12/15/86

Clofazimine

Lamprene

Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum

12/30/86

Tranexamic acidc

Cyklokapron

Treatment of patients with congenital coagulopathies who are undergoing surgical procedures (e.g., dental extractions)

3/19/87

Zidovudineb

Retrovir

(1) Treatment of AIDS-related complex. (2) Treatment of AIDS.

8/11/88

Tiopronin

Thiola

Prevention of cystine nephrolithiasis in patients with homozygous cystinuria

11/22/88

Metronidazole (topical)b

Metrogel

Treatment of acne rosacea

5/2/89

Mefloquine HClb

Lariam

(1) Prophylaxis for Plasmodium falciparum malaria that is resistant to other available drugs. (2) Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

5/25/89

Rifampinb,c

Rifadin I.V.

For antituberculosis treatment where use of the oral form of the drug is not feasible

6/5/89

Selegiline HClb

Eldepryl

As an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson’s disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism

12/22/89

Cromolyn sodium

Gastrocrom

Treatment of mastocytosis

10/2/90

Citric acid, glucono-delta-lactone and magnesium carbonatec

Renacidin irrigation

Treatment of renal and bladder calculi of the apatite or struvite variety

12/10/90

Calcium acetateb

Phos-lo

Treatment of hyperphosphatemia in end-stage renal failure

12/26/90

Altretamine

Hexalen

Palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy

10/30/92

Sotalol HClb

Betapace

Treatment of life-threatening ventricular tachyarrhythmias

11/25/92

Atovaquone

Mepron

For the acute oral treatment of mild to moderate Pneumocystis carinii pneumonia in patients who are intolerant to trimethoprim-sulfamethoxazole

12/23/92

Rifabutin

Mycobutin

Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection

7/23/93

Interferon beta-1bc,d

Betaseron

In ambulatory patients with relapsing-remitting multiple sclerosis to reduce the frequency of clinical exacerbations

9/10/93

Megestrol acetateb

Megace

Treatment of anorexia, cachexia, or an unexplained significant weight loss in patients with a diagnosis of acquired immune deficiency syndrome

9/23/93

Lodoxamide tromethamine

Alomide ophthalmic solution

Treatment of ocular disorders referred to by the terms vernal keratoconjunctivitis, vernal conjunctivitis, vernal keratitis

3/7/94

Desmopressin acetateb

N/A

Treatment of patients with hemophilia A or von Willebrand’s disease (type I) whose factor VIII coagulant activity level is greater than 5%

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

3/22/94

Pilocarpineb,d

Salagen

Treatment of symptoms of xerostomia from salivary gland hypofunction caused by radiotherapy for cancer of the head and neck

5/31/94

Rifampin, isoniazid, pyrazinamide

Rifater

For the short-course treatment of tuberculosis

6/30/94

Aminosalicylic acid

Paser granules

Treatment of tuberculosis infections

7/29/94

Sulfadiazineb

N/A

Toxoplasmosis, as adjunctive with pyrimethamine

8/15/94

Cysteamine

Cystagon

Treatment of nephropathic cystinosis in adults and children

8/3/95

Amiodarone HClb

Cordarone

For initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy

11/22/95

Tretinoinb,d

Vesanoid

Induction of remission in patients with acute promyelocytic leukemia who are refractory to or unable to tolerate anthracycline-based cytotoxic chemotherapeutic regimens

12/12/95

Riluzoleb,d

Rilutek

Treatment of patients with amyotrophic lateral sclerosis

4/30/96

Sodium phenylbutyrated

Buphenyl

Adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase

5/17/96

Interferon beta-1ac,e

Avonex

Treatment of relapsing forms of multiple sclerosis to slow the accumulation of physical disability and decrease the frequency of clinical exacerbations

5/17/96

Allopurinol sodiumb,c

Aloprim for injection

Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy that causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy

5/22/96

Ofloxacinb

Ocuflox ophthalmic solution

Treatment of bacterial corneal ulcers

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

6/11/96

Albendazole

Albenza

(1) Treatment of cystic hydatid disease of the liver, lung, and peritoneum, caused by the larval form of the dog tapeworm, Echinococcus granulosus. (2) Treatment of parenchymal neurocysticercosis due to active lesions caused by larval forms of the pork tapeworm, Taenia solium.

8/23/96

Somatropin for injectionc,e

Serostim

Treatment of AIDS wasting or cachexia

9/6/96

Midodrine HClb

Amatine

Treatment of symptomatic orthostatic hypotension

9/26/96

Pentosan polysulfate sodium

Elmiron

Relief of bladder pain or discomfort associated with interstitial cystitis

10/25/96

Betaine

Cystadane

Treatment of homocystinuria

11/27/96

Tizanidine HClb

Zanaflex

Treatment of spasticity associated with multiple sclerosis and spinal cord injury

12/20/96

Glatiramer acetatec

Copaxone

For reduction of the frequency of relapses in patients with relapsing-remitting multiple sclerosis

1/28/97

Zinc acetate

Galzin

For maintenance treatment of patients with Wilson’s disease who have been initially treated with a chelating agent

3/14/97

Anagrelideb

Agrylin

Treatment of patients with essential thrombocythemia

5/29/97

Toremifened

Fareston

Treatment of metastatic breast cancer in postmenopausal women with estrogen positive or receptor unknown tumors

12/10/97

Ursodiolb

Urso

Treatment of patients with primary biliary cirrhosis

2/25/98

Hydroxyureab

Droxia

To reduce the frequency of painful crises and to reduce the need for blood transfusions in adult patients with sickle cell anemia with recurrent moderate to severe painful crises

4/9/98

Sacrosidase

Sucraid

Oral replacement therapy of the genetically determined sucrase deficiency

6/5/98

Mafenide acetate solution

Sulfamylon solution

For use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

6/22/98

Rifapentine

Priftin

Treatment of pulmonary tuberculosis

7/16/98

Thalidomide

Thalomid

Acute treatment of the cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL) and as maintenance therapy for prevention and suppression of the cutaneous manifestations of ENL recurrences

8/24/98

Lamotrigineb

Lamictal

Adjunctive treatment of Lennox-Gastaut syndrome in pediatric and adult patients

8/24/98

Infliximabc,e

Remicade

Treatment of moderately to severely active Crohn’s disease for the reduction of signs and symptoms, in patients who have an inadequate response to conventional therapy; and treatment of patients with fistulizing Crohn’s disease for reduction in the number of draining enterocutaneous fistula(s)

12/24/98

Modafinil

Provigil

Improve wakefulness in patients with excessive daytime sleepiness associated with narcolepsy

2/2/99

Alitretinoin

Panretin

Topical treatment of cutaneous lesions in patients with AIDS-related Kaposi’s sarcoma

3/19/99

Lidocaine patch 5%

Lidoderm patch

For relief of allodynia (painful hypersensitivity) and chronic pain in postherpetic neuralgia

6/28/99

Doxorubicin liposomec,d

Doxil

Treatment of metastatic carcinoma of the ovary in patients with disease that is refractory to both paclitaxel-and platinium-based chemotherapy regimens

10/21/99

Exemestaned

Aromasin

Treatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy

12/29/99

Bexarotene

Targretin

Treatment of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy

5/10/01

Imatinib mesylated

Gleevec

Treatment of patients with chronic myeloid leukemia (CML) in blast crisis, accelerated phase, or in chronic phase after failure of interferon-alpha therapy

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

8/28/01

Topiramated

Topamax

As adjunctive therapy in patients 2 years and older with seizures associated with Lennox-Gastaut syndrome

11/20/01

Bosentan

Tracleer

Treatment of pulmonary arterial hypertension

1/18/02

Nitisinone

Orfadin

Adjunctive therapy to dietary restriction of tyrosine and phenylalanine in treatment of hereditary tyrosinemia type 1

7/17/02

Oxybate

Xyrem

Treatment of cataplexy associated with narcolepsy

10/8/02

Buprenorphine in combination with naloxone

Suboxone

Treatment of opioid dependence in patients 16 years of age or older

10/8/02

Buprenorphine hydrochlorided

Subutex

Treatment of opioid dependence in patients 16 years of age or older

11/22/02

Nitazoxanide

Alinia

Treatment of diarrhea caused by Cryptosporidium parvum and Giardia lamblia

3/25/03

Pegvisomantc

Somavert

Treatment of acromegaly in patients who have had an inadequate response to surgery and/or radiation therapy and/or other medical therapies, or for whom these therapies are not appropriate

7/31/03

Miglustatd

Zavesca

Treatment of mild to moderate Type I Gaucher disease in adults for whom enzyme replacement therapy is not a therapeutic option

12/1/03

Somatropin (r-DNA)c,e

Zorbtive

Treatment of short bowel syndrome in patients receiving specialized nutritional support

3/8/04

Cinacalcetd

Sensipar

Treatment of hypercalcemia in patients with parathyroid carcinoma

4/20/04

Apomorphine HClc

Apokyn

For the acute, intermittent treatment of hypomobility, “off” episodes, associated with advanced Parkinson’s disease

5/17/04

Tinidazole

Tindamax

(1) Treatment of giardiasis caused by G. duodenalis (also termed G. lamblia). (2) Treatment of intestinal amebiasis and amebic liver abcess caused by E. histolytica.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

6/10/04

Glutamine

Nutrestore

Treatment of short bowel syndrome in patients receiving specialized nutritional support when used in conjunction with a recombinant human growth hormone that is approved for this indication

8/12/05

Quinine sulfate

Qualaquin

Treatment of uncomplicated Plasmodium falciparum malaria

10/28/05

Nelarabinec,d

Arranon

Treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens

11/2/05

Deferasirox

Exjade

Treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age or older

12/20/05

Sorafenib

Nexavar

Treatment of patients with advanced renal cell carcinoma

12/27/05

Lenalidomide

Revlimid

Treatment of patients with transfusion dependant anemia due to low or intermediate-1 risk myelodysplastic syndromes associated with a deletion 5 q cytogenetic abnormality with or without additional cytogenetic abnormalities

3/1/06

Cetuximabc,d,e

Erbitux

For use in combination with radiation therapy, for the treatment of locally or regionally advanced squamous cell carcinoma of the head and neck (SCCHN) and for use as a single agent for the treatment of patients with recurrent or metastatic SCCHN for whom prior platinum-based therapy has failed

3/29/06

Tacrolimusb,c

Prograf

Prophylaxis of organ rejection in patients receiving allogenic heart transplants

4/28/06

Recombinant human acid alpha-glucosidasec,e

Myozyme

For use in patients with Pompe disease (GAA deficiency)

5/2/06

Decitabinec

Dacogen

For treatment of patients with myelodysplastic syndromes

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

6/28/06

Dasatinibd

Sprycel

(1) Treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy. (2) Treatment of adults with CML with resistance or intolerance to prior therapy including imatinib.

7/24/06

Idursulfasec,d,e

Elaprase

Indicated for patients with Hunter syndrome (mucopolysaccharidosis II, MPS II)

10/6/06

Vorinostat

Zolinza

Treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma (CTCL) who have progressive, persistent, or recurrent disease on or following two systemic therapies

5/30/07

Temsirolimusc

Torisel

Treatment of advanced renal cell carcinoma

5/31/07

Somatropinc,e

Norditropin

Treatment of short stature in patients with Noonan’s syndrome

6/15/07

Ambrisentan

Letairis

Treatment of pulmonary arterial hypertension (WHO group I) in patients with WHO class II or III symptoms to improve exercise capacity and delay clinical worsening

8/30/07

Lanreotidec

Somatuline depot

Long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy

9/13/07

Raloxifene

Evista

Reduction in risk of invasive breast cancer in postmenopausal women with osteoporosis and reduction in risk of invasive breast cancer in postmenopausal women at high risk for invasive breast cancer

10/29/07

Nilotinibd

Tasigna

For the use for chronic phase (CP) and accelerated phase (AP) Philadelphia chromosome positive chronic myelogenous leukemia (CML) in adult patients resistant to or intolerant to prior therapy that included imatinib

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approvala

12/13/07

Sapropterin

Kuvan

Indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive phenylketonuria (PKU)

2/27/08

Rilonaceptc,e

Arcalyst

Treatment of cryopyrin-assisted periodic syndromes (CAPS)

8/15/08

Tetrabenazine

Xenazine

Treatment of chorea associated with Huntington’s disease

9/12/08

Immune globulin (human)c,e

Gamunex

Treatment of chronic inflammatory demyelinating polyneuropathy

11/14/08

Rufinamide

Banzel

Adjunctive therapy of seizures associated with Lennox-Gastaut syndrome

11/20/08

Eltrombopag

Promacta

Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy

NOTE: CIAS1 = cold-induced autoinflammatory syndrome.

a We excluded drugs that are covered under Medicare Part B or that are not relevant for the Medicare population (i.e., removed orphan approvals for a pediatric indication). The drugs are sorted by the exclusivity date (i.e., date of approval of orphan indication) for first orphan approval with a relevant indication. Drugs with multiple orphan designations often have exclusivity different dates associated with each approved indication. The text for some indications has been abbreviated.

b These drugs are available in generic form.

c These drugs have one of the following routes of administration: injection, intravenous, intramuscular, irrigation, or subcutaneous.

d These drugs have one or more indications on the Social Security Compassionate Allowances List.

e These are biologics, as opposed to chemical entities.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-A2 Medicare Stand-Alone PDP Coverage of Orphan Drugs: Inclusion on Formulary (i.e., Plan Coverage), Tier Placement, and Utilization Management (99 drugs)

Generic Name

Trade Name

% Plans That Cover Drug

Citric acid, glucono-delta-lactone, and magnesium carbonate

Renacidin irrigation

0.0

Clofazimine

Lamprene

0.0

Glutamine

Nutrestore

0.0

Zinc acetate

Galzin

0.0

Lodoxamide tromethamine

Alomide ophthalmic solution

45.6

Tinidazole

Tindamax

47.4

Metronidazole (topical)

Metrogel

49.3

Somatropin for injection

Serostim

50.4

Recombinant human acid alpha-glucosidase

Myozyme

51.8

Rifampin, isoniazid, pyrazinamide

Rifater

52.7

Doxorubicin liposome

Doxil

53.0

Nelarabine

Arranon

53.6

Cetuximab

Erbitux

53.6

Somatropin (r-DNA)

Zorbtive

53.6

Temsirolimus

Torisel

54.1

Allopurinol sodium

Aloprim for injection

56.6

Decitabine

Dacogen

57.8

Mafenide acetate solution

Sulfamylon solution

59.1

Rilonacept

Arcalyst

61.2

Lanreotide

Somatuline depot

64.1

Rifampin

Rifadin I.V.

65.9

Somatropin

Norditropin

69.1

Buprenorphine hydrochloride

Subutex

71.1

Pentosan polysulfate sodium

Elmiron

72.0

Buprenorphine in combination with naloxone

Suboxone

73.6

Immune globulin (human)

Gamunex

73.8

Albendazole

Albenza

75.9

Idursulfase

Elaprase

76.0

Nitazoxanide

Alinia

78.1

Quinine sulfate

Qualaquin

83.1

Cromolyn sodium

Gastrocrom

83.9

Sapropterin

Kuvan

84.0

Levocarnitine

Carnitor

84.4

Interferon

Avonex

84.5

beta-1a

 

 

Apomorphine HCl

Apokyn

85.4

Tetrabenazine

Xenazine

86.4

Oxybate

Xyrem

87.1

Atovaquone

Mepron

88.8

Rifapentine

Priftin

89.0

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

% Plans That Place on Tier 3

% Plans That Place on Tier 4

% Plans That Have ST

% Plans That Have QL

% Plans That Have PA

65.2

32.8

0.0

0.5

0.0

46.4

30.7

0.0

0.3

0.0

39.5

4.6

0.0

0.0

0.1

12.5

82.9

0.0

21.1

99.6

13.5

81.8

0.0

0.0

37.8

45.8

20.2

0.0

0.0

0.0

17.4

74.3

0.0

0.0

56.7

30.8

65.0

0.0

0.0

28.1

26.0

69.6

0.0

0.0

74.6

14.0

81.8

0.0

19.8

99.5

9.3

86.0

0.0

0.0

69.5

8.4

24.2

0.0

0.0

1.1

9.2

82.1

0.0

0.0

71.7

38.5

14.3

0.0

7.1

0.0

7.9

91.7

0.0

18.2

63.2

7.3

92.2

9.8

33.4

80.4

14.2

16.0

0.0

0.0

7.4

15.6

80.9

0.0

15.4

99.7

45.9

15.3

0.0

28.2

51.9

46.2

12.3

0.0

14.7

0.0

39.1

22.7

0.0

11.5

34.5

18.4

80.9

0.0

0.0

99.8

34.7

16.3

0.0

0.0

0.0

9.4

84.6

0.0

0.0

45.9

49.3

18.7

0.0

33.9

1.3

41.0

18.1

0.0

30.7

52.1

32.0

17.0

0.0

0.0

0.0

13.8

83.5

0.0

0.3

44.7

5.0

0.0

0.0

0.0

7.9

12.8

82.0

0.1

51.9

91.1

11.6

70.5

0.0

8.6

56.5

14.8

82.0

0.0

22.7

69.8

12.4

58.5

0.0

27.6

37.9

17.7

74.5

12.0

19.7

13.6

45.6

28.7

0.0

0.0

0.0

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Generic Name

Trade Name

% Plans That Cover Drug

Eltrombopag

Promacta

90.5

Cysteamine

Cystagon

91.9

Midodrine HCl

Amatine

92.3

Modafinil

Provigil

93.5

Trientine HCl

Syprine

94.0

Anagrelide

Agrylin

95.6

Riluzole

Rilutek

95.7

Mefloquine HCl

Lariam

95.8

Ambrisentan

Letairis

98.3

Tiopronin

Thiola

99.4

Betaine

Cystadane

99.8

Pegvisomant

Somavert

99.9

Tizanidine HCl

Zanaflex

99.9

Cromolyn sodium 4% ophthalmic solution

Opticrom 4% ophthalmic solution

99.9

Deferasirox

Exjade

99.9

Ofloxacin

Ocuflox ophthalmic solution

99.9

Alitretinoin

Panretin

100.0

Altretamine

Hexalen

100.0

Aminosalicylic acid

Paser granules

100.0

Amiodarone HCl

Cordarone

100.0

Bexarotene

Targretin

100.0

Bosentan

Tracleer

100.0

Calcium acetate

Phos-lo

100.0

Cinacalcet

Sensipar

100.0

Dasatinib

Sprycel

100.0

Desmopressin acetate

N/A

100.0

Exemestane

Aromasin

100.0

Glatiramer acetate

Copaxone

100.0

Hydroxyurea

Droxia

100.0

Imatinib mesylate

Gleevec

100.0

Infliximab

Remicade

100.0

Interferon beta-1b

Betaseron

100.0

Lamotrigine

Lamictal

100.0

Lenalidomide

Revlimid

100.0

Lidocaine patch 5

Lidoderm patch

100.0

Megestrol acetate

Megace

100.0

Miglustat

Zavesca

100.0

Naltrexone HCl

Revia

100.0

Nilotinib

Tasigna

100.0

Nitisinone

Orfadin

100.0

Pilocarpine

Salagen

100.0

Potassium citrate

Urocit-K

100.0

Raloxifene

Evista

100.0

Rifabutin

Mycobutin

100.0

Rufinamide

Banzel

100.0

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

% Plans That Place on Tier 3

% Plans That Place on Tier 4

% Plans That Have ST

% Plans That Have QL

% Plans That Have PA

19.3

78.0

0.0

46.0

88.7

51.0

12.0

0.0

0.0

16.1

3.5

0.0

0.0

11.5

0.2

48.8

6.9

0.2

74.3

98.5

49.2

11.5

0.0

0.0

0.0

0.1

0.0

0.0

0.1

11.5

11.5

65.9

0.0

11.1

29.1

0.0

0.0

0.0

11.2

0.2

15.5

79.8

10.8

33.6

50.7

48.2

12.5

0.0

0.0

0.0

52.0

11.0

0.0

0.0

15.0

13.7

68.9

6.3

30.2

85.9

4.3

0.0

0.0

0.1

0.1

0.0

0.0

0.0

0.9

0.2

15.3

79.3

0.0

0.0

51.2

2.1

0.0

0.0

5.3

0.2

16.2

57.6

0.0

4.2

10.8

19.3

74.6

0.0

0.0

28.6

45.5

25.6

0.0

0.0

4.4

0.0

0.0

0.0

0.0

5.1

14.0

68.0

0.0

21.5

55.2

15.0

80.0

10.6

29.8

62.4

4.3

2.2

0.0

0.0

0.2

27.9

2.2

10.5

39.8

28.6

15.2

79.7

10.7

40.9

55.4

6.3

0.0

1.4

9.3

0.1

40.4

16.9

10.7

21.1

0.0

13.0

82.4

0.0

43.8

91.7

0.0

0.0

0.0

0.0

0.2

15.2

82.3

0.0

28.8

69.0

12.6

82.5

1.1

0.0

94.4

15.3

82.4

22.6

43.0

92.1

4.2

0.0

13.8

30.6

33.5

15.0

80.2

0.0

34.6

69.9

33.6

8.4

6.3

56.4

45.7

2.1

0.0

0.0

21.3

7.4

15.9

62.4

0.0

6.7

31.1

0.0

0.0

0.0

0.0

0.2

15.2

80.0

10.7

38.7

53.2

15.6

75.3

0.0

0.0

26.4

3.1

0.0

0.0

0.0

0.2

0.0

0.0

0.0

0.0

0.2

21.1

2.2

0.0

63.8

0.0

38.5

12.4

0.0

0.0

0.0

45.7

25.6

0.0

48.3

40.9

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Generic Name

Trade Name

% Plans That Cover Drug

Sacrosidase

Sucraid

100.0

Selegiline HCl

Eldepryl

100.0

Sodium phenylbutyrate

Buphenyl

100.0

Sorafenib

Nexavar

100.0

Sotalol HCl

Betapace

100.0

Sulfadiazine

N/A

100.0

Tacrolimus

Prograf

100.0

Thalidomide

Thalomid

100.0

Topiramate

Topamax

100.0

Toremifene

Fareston

100.0

Tranexamic acid

Cyklokapron

100.0

Tretinoin

Vesanoid

100.0

Ursodiol

Urso

100.0

Vorinostat

Zolinza

100.0

Zidovudine

Retrovir

100.0

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

% Plans That Place on Tier 3

% Plans That Place on Tier 4

% Plans That Have ST

% Plans That Have QL

% Plans That Have PA

20.5

66.6

0.0

0.0

21.9

0.0

0.0

0.3

0.1

0.2

16.1

61.7

0.0

0.0

19.3

12.9

79.9

0.0

41.2

83.1

0.0

0.0

0.0

0.0

0.2

1.4

4.3

0.0

0.0

0.0

39.9

18.6

0.0

7.6

99.8

10.2

79.8

0.0

33.5

71.4

11.6

2.1

0.1

38.8

13.7

48.3

16.6

0.0

16.9

4.1

17.0

11.8

0.0

0.0

24.0

8.2

50.5

0.0

0.0

31.9

2.1

0.0

0.0

0.0

0.2

15.3

80.0

0.0

34.5

64.1

2.1

0.0

0.0

0.2

0.0

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-A3 Drugs with a Pediatric Orphan Indication (1983-2008 Approvals) (27 drugs)

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approval

10/17/85

Somatropin

Nutropin

For use in the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion

10/17/85

Somatrem for injection

Protropin

For long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion

3/8/87

Somatropin for injection

Humatrope

For the long-term treatment of children who have growth failure due to inadequate secretion of normal endogenous growth hormone

8/2/90

Colfosceril palmitate, cetyl alcohol, tyloxapol

Exosurf neonatal for intratracheal suspension

Treatment of established hyaline membrane disease at all gestational ages

1/30/91

Succimer

Chemet capsules

Treatment of lead poisoning in children

7/1/91

Beractant

Survanta intratracheal suspension

(1) Prevention of RDS (hyaline membrane disease) in premature infants less than 1250 grams birth weight or with evidence of surfactant deficiency. (2) Treatment of (“rescue”) premature infants with RDS confirmed by x-ray and requiring mechanical ventilation.

12/24/91

Histrelin acetate

Supprelin injection

Treatment of central precocious puberty

2/26/92

Nafarelin acetate

Synarel nasal solution

Treatment of central precocious puberty

7/14/92

Teniposide

Vumon for injection

Induction therapy in patients with refractory childhood acute lymphoblastic leukemia

4/16/93

Leuprolide acetate

Lupron injection

Treatment of children with central precocious puberty

7/29/93

Felbamate

Felbatol

As adjunctive therapy in the treatment of partial and generalized seizures associated with the Lennox-Gastaut syndrome in children

12/27/93

Immune globulin intravenous, human

Gamimune N

Infection prophylaxis in pediatric patients affected with the human immunodeficiency virus

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approval

1/18/96

Respiratory syncytial virus immune globulin (human)

Respigam

Prophylaxis of respiratory syncytial virus (RSV) lower respiratory tract infections in infants and young children at high risk of RSV disease

9/26/97

Sermorelin acetate

Geref

Treatment of idiopathic or organic growth hormone deficiency in children with growth failure

5/27/99

Etanercept

Enbrel

Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying antirheumatic drugs

9/21/99

Caffeine

Cafcit

Short-term treatment of apnea of prematurity in infants between 28 and less than 33 weeks gestational age

6/20/00

Somatropin (r-DNA)

Genotropin

Long-term treatment of pediatric patients who have growth failure due to Prader-Willi syndrome (PWS)

7/12/02

Rasburicase

Elitek

Treatment of malignancy-associated or chemotherapy-induced hyperuricemia

7/29/03

Ribavirin

Rebetol

Treatment of chronic hepatitis C among previously untreated pediatric patients at least 3 years of age or older

10/23/03

Botulism immune globulin

Babybig

Indicated for treatment of infant botulism caused by type A or type B Clostridium botulinum

12/28/04

Clofarabine

Clolar

Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens

8/11/05

Meloxicam

Mobic

For relief of the signs and symptoms of pauciarticular or polyarticular course juvenile rheumatoid arthritis in patients 2 years of age or older

8/30/05

Mecasermin

Increlex

Long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH

12/12/05

Mecasermin rinfabate

Iplex

Treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Exclusivity Start Date

Generic Name

Trade Name

Indication for Original Approval

4/13/06

Ibuprofen lysine

Neoprofen

For closure of a clinically significant patent ductus arteriosus in premature infants weighing between 500 and 1500 g, who are no more than 32 weeks gestational age when usual medical management (e.g., fluid restriction, diuretics, respiratory support) is ineffective

12/20/06

Balsalazide disodium

Colazal

Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older

2/21/08

Adalimumab

Humira

Treatment of juvenile rheumatoid arthritis

NOTE: This list includes drugs that received approval only for a pediatric orphan indication. Remicade, which received a pediatric orphan approval for the treatment of pediatric Crohn’s disease, is also approved for an adult orphan indication and was therefore included in a previous list of drugs. The drugs are sorted by the exclusivity date (i.e., date of approval of orphan indication) for first orphan approval with a relevant indication. Drugs with multiple orphan designations often have different exclusivity dates associated with each approved indication. The text for some indications has been abbreviated.

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

TABLE C-A4 Medicare Stand-Alone PDP Coverage for Drugs with a Pediatric Orphan Indication: Inclusion on Formulary (i.e., Plan Coverage), Tier Placement, and Utilization Management (27 drugs)

Generic Name

Trade Name

No. of Plans That Cover Drug

% Plans That Cover Drug

% - Plans That Place on Tier 3

% Plans That Place on Tier 4

% Plans That Have ST

% Plans That Have QL

% Plans That Have PA

Beractant

Survanta intratracheal suspension

0

0.0

Botulism immune globulin

Babybig

0

0.0

Caffeine

Cafcit

0

0.0

 

Colfosceril palmitate, cetyl alcohol, tyloxapol

Exosurf neonatal for intratracheal suspension

0

0.0

Histrelin acetate

Supprelin injection

0

0.0

Ibuprofen lysine

Neoprofen

0

0.0

Immune globulin intravenous, human

Gamimune n

0

0.0

Mecasermin rinfabate

Iplex

0

0.0

Respiratory syncytial virusimmune globulin (human)

Respigam

0

0.0

Sermorelin acetate

Geref

0

0.0

Somatrem for injection

Protropin

0

0.0

Succimer

Chemet capsules

0

0.0

Teniposide

Vumon for injection

0

0.0

Somatropin [r-DNA]

Genotropin

622

38.4

57.6

25.6

0.0

27.8

99.5

Somatropin

Nutropin

625

38.6

16.6

77.6

0.0

28.0

99.5

Somatropin for injection

Humatrope

693

42.8

15.0

79.4

0.0

25.3

99.6

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

Generic Name

Trade Name

No. of Plans That Cover Drug

% Plans That Cover Drug

% Plans That Place on Tier 3

% Plans That Place on Tier 4

% Plans That Have ST

% Plans That Have QL

% Plans That Have PA

Clofarabine

Clolar

899

55.5

25.0

66.6

0.0

0.0

19.4

Mecasermin

Increlex

1,347

83.1

21.4

73.1

0.0

0.0

92.0

Nafarelin acetate

Synarel nasal solution

1,445

89.2

30.0

61.7

0.0

0.0

31.4

Etanercept

Enbrel

1,450

89.5

11.5

83.3

1.4

45.2

93.7

Balsalazide disodium

Colazal

1,536

94.8

4.4

0.0

0.0

0.0

0.2

Meloxicam

Mobic

1,546

95.4

0.0

0.0

0.1

 

0.2

Leuprolide acetate

Lupron injection

1,554

95.9

11.1

9.8

0.0

18.1

71.1

Adalimumab

Humira

1,618

99.9

13.0

82.4

1.2

46.8

94.3

Felbamate

Felbatol

1,620

100.0

38.1

18.9

0.0

0.0

0.0

Rasburicase

Elitek

1,620

100.0

17.7

77.3

0.0

0.0

49.0

Ribavirin

Rebetol

1,620

100.0

6.9

6.4

0.0

17.4

73.6

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

REFERENCES

CMS (Centers for Medicare and Medicaid Services). 2009a. 2010 Medicare Advantage Ratebook and Prescription Drug Rate Information: 2010 Low-Income Premium Subsidy Amounts. http://www.cms.hhs.gov/MedicareAdvtgSpecRateStats/Downloads/RegionalRatesBenchmarks2010.pdf (accessed September 2, 2010).

CMS. 2009b. 2010 Medicare Part D National Stand-Alone Prescription Drug Plans. http://www.cms.hhs.gov/PrescriptionDrugCovGenIn/Downloads/NationalPDPs.pdf (accessed September 2, 2010).

CMS. 2010. CMS Announces Course of Action to Identify Protected Class of Prescription Drugs. http://www.cms.hhs.gov/apps/media/press/release.asp?Counter=3409 (accessed September 2, 2010).

KFF (Kaiser Family Foundation). 2009a. Medicare: Low-Income Assistance Under the Medicare Drug Benefit. http://www.kff.org/medicare/upload/7327-05.pdf (accessed September 2, 2010).

KFF. 2009b. Medicare Part D 2009 Data Spotlight: Specialty Tiers. http://www.kff.org/medicare/upload/7919.pdf (accessed September 2, 2010).

KFF. 2009c. Medicare Part D 2010 Spotlight: Benefit Design and Cost-Sharing. December. http://www.kff.org/medicare/upload/8033.pdf (accessed September 2, 2010).

KFF. 2009d. Medicare Part D Spotlight: Part D Plan Availability in 2010 and Key Changes since 2006. http://www.kff.org/medicare/upload/7986.pdf (accessed September 2, 2010).

KFF. 2010a. Explaining Health Care Reform: Key Changes to the Medicare Part D Drug Benefit Coverage Gap. http://www.kff.org/healthreform/upload/8059.pdf (accessed September 2, 2010).

KFF. 2010b. Medicare: A Primer. http://www.kff.org/medicare/upload/7615-03.pdf (accessed September 2, 2010).

KFF. 2010c. Medicare at a Glance. http://www.kff.org/medicare/upload/1066-12.pdf (accessed September 2, 2010).

NORD (National Organization for Rare Disorders). 2006. Letter to Mark B. McClellan, M.D., Ph.D., Administrator of CMS: Orphan Drug Coverage in Medicare Part D Formularies January. http://www.rarediseases.org/news/pdf/Final_ltr_CMS_011006_V2.pdf (accessed September 2, 2010).

OIG (Office of Inspector General, U.S. Department of Health and Human Services). 2001. The Orphan Drug Act: Implementation and Impact. http://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf (accessed September 2, 2010).

Social Security Online. 2010. Compassionate Allowances. http://www.socialsecurity.gov/compassionateallowances/ (accessed September 2, 2010).

Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×

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Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Page326
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Page327
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Page328
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Page329
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page330
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page331
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page332
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page333
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page334
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page335
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page336
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page337
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page338
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page339
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page340
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page341
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page342
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
×
Page343
Suggested Citation:"Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs." Institute of Medicine. 2010. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: The National Academies Press. doi: 10.17226/12953.
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Page344
Next: Appendix D: Glossary, Abbreviations, and Public Laws »
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Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

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