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
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—
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;
-
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
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Tier 3: nonpreferred brand-name drugs
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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). |
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
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.
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
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. |
TABLE C-2 Orphan Drug Coverage by Type of Medicare Prescription Drug Plan (99 drugs)
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
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. |
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
TABLE C-4 Orphan Drugs by Rate of Tier 4 Placement and Type of Medicare Prescription Drug Plan (95 drugs)
TABLE C-5 Orphan Drugs by Rate of Prior Authorization Use and Type of Medicare Prescription Drug Plan (95 drugs)
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.
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.
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. |
Exclusivity Start Date |
Generic Name |
Trade Name |
Indication for Original Approvala |
5/25/89 |
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 |
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% |
Exclusivity Start Date |
Generic Name |
Trade Name |
Indication for Original Approvala |
3/22/94 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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 |
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. |
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 |
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 |
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 |
Prograf |
Prophylaxis of organ rejection in patients receiving allogenic heart transplants |
|
4/28/06 |
Myozyme |
For use in patients with Pompe disease (GAA deficiency) |
|
5/2/06 |
Decitabinec |
Dacogen |
For treatment of patients with myelodysplastic syndromes |
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 |
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 |
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 |
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 |
Arcalyst |
Treatment of cryopyrin-assisted periodic syndromes (CAPS) |
|
8/15/08 |
Tetrabenazine |
Xenazine |
Treatment of chorea associated with Huntington’s disease |
9/12/08 |
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. |
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 |
% 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 |
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 |
% 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 |
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 |
% 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 |
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 |
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 |
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 |
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 |
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).