Global Health and the Future Role of the United States (2017) / Chapter Skim
Currently Skimming:
7 Catalyzing Innovation
7 Catalyzing Innovation
Pages 205-240
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 205... ...
This includes addressing multiple steps along the value chain, such as enabling innovative trial designs, streamlining regulation, ensuring both the supply and demand through manufacturing capacity and market incentives, and finally building research and development (R&D) capacity in LMICs.
|
From page 206... ...
These include enabling innovative trial design approaches; streamlining regulation; ensuring supply; creating market incentives; and building capacity for medical products manufacturing in partner countries to foster global health security and better support local needs. Enabling Innovative Trial Design Approaches Traditional clinical trials, though the mainstay of clinical research, often have shortcomings because of their rigidity or poor adaptability of results in real-life circumstances.
|
From page 207... ...
. Adaptive Trial Designs Clinical trials are often hindered by the rigidity of the protocols.
|
From page 208... ...
. While FDA has issued draft guidance on the use of adaptive clinical trials (FDA, 2010)
|
From page 209... ...
. And with the passing of the 21st Century Cures Act, FDA is now required to develop a framework on evaluating real world evidence for drug regulation to guide its use in clinical trials (Hills and Zegarelli, 2017)
|
From page 210... ...
. Thus, while biomarkers may improve the efficiency of clinical trials and their use has been encouraged by FDA (FDA, 2004)
|
From page 211... ...
, and only 9.6 percent of all new drugs successfully progress from Phase 1 clinical trials to FDA approval (Thomas et al., 2015)
|
From page 212... ...
Breakthrough Therapy The breakthrough therapy mechanism expedites the development and re view of new drugs that target serious conditions and suggest, based on clinical evidence, an improvement over available therapy. A manufacturer can request designation for a product no later than phase 2 clinical trials.
|
From page 213... ...
Orphan Drug Act The Orphan Drug Act of 1983 was introduced to incentivize drug development for rare diseases11 by providing pharmaceutical companies a 7-year market exclusivity, a tax credit that covers 50 percent of clinical trial costs, R&D grants, fast-track approval, and a waiver of the user fee associated with the application (Franco, 2013)
|
From page 214... ...
Priority Review Voucher Program The PRV program was established in 2007 and designed to reward innovators for developing novel treatments for diseases that would otherwise not attract development interest. While initially intended for neglected diseases, additional PRV programs have since been created to include rare pediatric diseases in 2012 (Gaffney et al., 2016)
|
From page 215... ...
Generating Antibiotic Incentives Now Act In contrast with the Orphan Drug Act and the PRV program, the GAIN Act does not address a rare or neglected disease, but still fills an unmet need. As discussed in Chapter 3, the dire health and economic challenges that antimicrobial resistance poses, in addition to the dwindling pipeline for new antimicrobials, makes it essential to spur development.
|
From page 216... ...
. Acknowledging the need for a long-term commitment and outlook when developing and manufacturing medical products, government contracts with CIADMs can be renewed for up to 25 years.
|
From page 217... ...
government to implement because of the extremely high costs of drug development; they would require a large infusion of money up front in order to incentivize involvement. In 2014 the President's Council 17 Push mechanisms incentivize industry by reducing the cost of R&D.
|
From page 218... ...
small biotechnology companies) , and the product or process being targeted (i.e., emerging infectious diseases versus classic drug development)
|
From page 219... ...
Yet, building local capacity to conduct clinical trials in countries where these diseases are endemic is paramount. As the world faces new health threats that can more easily cross national borders, investing in global R&D will require expansion of local capabilities to anticipate new threats and augment opportunities for the development of novel vaccines and therapeutics.
|
From page 220... ...
Although these types of partnerships can be costly to U.S.-based academic institutions, additional funding can be leveraged through the Fogarty International Center at NIH and USAID,21 which ensures sustainability of 19 Personal communication with Daniel Bausch, Tulane University, November 9, 2016. 20 Personal communication with Robert Einterz, AMPATH, December 5, 2016.
|
From page 221... ...
. Through another NIHsponsored grant, Vanderbilt University is working with Tulane University and the Kenema Government Hospital in Sierra Leone to build capacity and training programs for researchers in the recently Ebola-affected countries so that they can soon write grant proposals and conduct their own clinical trials.
|
From page 222... ...
Conversely, the importance of adequate laboratory capacity is illustrated by the African Center of Excellence for Genomics of Infectious Diseases in Nigeria, which was able to accurately diagnose the index case of the Nigerian Ebola outbreak and enable the government to contain the spread before the outbreak became out of control (NASEM, 2017a)
|
From page 223... ...
. The committee agrees with the statement made by the Committee on Clinical Trials During the 2014–2015 Ebola Outbreak, that "What seems certain to us is that the actual options are to pay now and prepare in advance, or to pay later when an outbreak occurs, with the likelihood that the cost will be multiple times greater in the latter case" (NASEM, 2017a, p.
|
From page 224... ...
Conclusion: Creating the capacity for low- and middle-income countries to conduct clinical trials where the burden of disease is highest, using their own workforces and facilities, is both more efficient and more cost-effective than relying on donor nations for these efforts. Creating this capacity will require investing in labo ratory capacity, and an appropriately trained research-competent workforce.
|
From page 225... ...
BARDA should assess ex panding its list of priority products for codevelopment with industry, taking into account global health priorities. • Streamlining regulation: FDA should receive adequate re sources to improve the tropical disease priority review voucher program and should assess the application of the provisions outlined in the Generating Antibiotic Incentives Now Act to neglected tropical diseases beyond those on the qualified pathogen list.
|
From page 226... ...
Though the applications of digital health technology are expansive, the committee has categorized them into three broad application areas for discussion: data systems creation, health care service optimization, and 23 The Mobile Alliance for Maternal Action (MAMA) was launched in 2011 as a public– private partnership between USAID, Johnson & Johnson, the UN Foundation, and BabyCenter with the goal of catalyzing a global community to deliver vital health information to new and expectant mothers and their families through mobile phones.
|
From page 227... ...
. Though long-term, complete solutions to both of these issues requires the scale-up of human resources and creation of adequate amounts and types of clinics, digital health tools have been able to provide effective interim solutions.
|
From page 228... ...
The model of eCompliance has the potential to be expanded beyond TB, to areas such as consistent antenatal care, childhood vaccination documentation, or prevention of mother-to-child HIV transmission. Research Efficiency: Clinical Ink Clinical Ink is a technology company that offers a mobile solution to stream line clinical trials, called SureSource, which is currently the only platform that meets all U.S.
|
From page 229... ...
See Box 7-2 for an example of a digital health solution for clinical trials. A Need for a Paradigm Shift The tremendous value and corresponding excitement that digital health provides has come at a cost, as the proliferation of interest and the variety of stakeholders involved has created a fragmented approach to the use of digital health tools in many countries.
|
From page 230... ...
While such a platform would be useful for day-to-day health care delivery and operations, it would also revolutionize how surveillance and response for public health threats can be managed within and between countries. The committee sees a digital health platform revolutionizing the three broad areas of digital health: data systems, health care service optimization, and research efficiency, as described in the following sections.
|
From page 231... ...
A digital health platform can improve on the efficiencies already gained through facilitation of data-sharing among investigators, potentially allowing for novel research inquiry. For example, if two similar clinical trials are occurring simultaneously, a digital health platform could enable controlled sharing of data (within the scope of research ethics)
|
From page 232... ...
. The Global Digital Health Index is further described in Box 7-3.
|
From page 233... ...
The committee sees a strong opportunity for the United States to build on this goal and rethink its global health strategies to join the global momentum toward harmonization of digital health approaches. SUMMARY AND RECOMMENDATION Decreases in the costs and increased demand of mobile technology have allowed for the integration of successful digital health efforts in the United States and around the globe (Mehl and Labrique, 2014; Steinhubl et al., 2013)
|
From page 234... ...
and the U.S. Department of State should incentivize and support countries in building interoperable digital health platforms that can efficiently collect and use health data and analytic insights to enable the delivery of integrated services within a country.
|
From page 235... ...
2015. The Food and Drug Administration and pragmatic clinical trials of marketed medical products.
|
From page 236... ...
n.d.-b. Global Digital Health Index.
|
From page 237... ...
2016. Unlocking the potential of digital health.
|
From page 238... ...
2016. Efficiencies of platform clinical trials: A vision of the future.
|
From page 239... ...
2015. The economics of orphan drugs: The effectiveness of Priority Review Vouchers on the development of drugs to combat neglected tropical diseases.
|
From page 240... ...
Contemporary Clinical Trials 31(1)
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.