President Donald J Trump’s selection of Scott Gottlieb, MD, to lead the Food and Drug Administration (FDA) has ushered in a new direction for the government agency, which is likely to alter the availability and use of pharmaceuticals in the United States.
On May 11, 2017, Scott Gottlieb, MD, was sworn in as the new Commissioner of the FDA.1 A Republican physician and cancer survivor with strong ties to pharmaceutical manufacturers, Dr Gottlieb is expected to implement policy within the FDA to promote greater access to both pharmaceutical products and information about their use. This new direction for the FDA will be supported by reent legislation such as the 21st Century Cures Act (Cures Act)2 and existing regulatory guidance such as that of Section 114 of the Food and Drug Administration Modernization Act of 1997 (FDAMA 114).3 Therefore, it will be important for stakeholders to understand these policies in order to navigate the regulatory environment as it is likely to evolve.
The 21st Century Cures Act
The Cures Act was developed with the goal of expediting the discovery, development, and delivery of new treatments and cures for disease.4 Specifically, the law calls for the following actions in order to meet these goals.
Discovery: The Cures Act designates that the National Institutes of Health (NIH) be provided with $4.8 billion in new funding that is fully offset. One of the intended beneficiaries of this funding is the Precision Medicine Initiative ($1.5 billion).4,5 Initiated by the White House under former President Barack Obama, the Precision Medicine Initiative is a research effort intended to revolutionize approaches to medical treatment by better taking into account individual differences in genes, environments, and lifestyles.
A second research project designated to receive funding through the Cures Act ($1.8 billion)4 is the Beau Biden Cancer Moonshot,6 an initiative of former Vice President Joseph Biden that is intended to accelerate cancer research, to make cancer therapies more accessible to patients, and to improve cancer prevention and detection.
Finally, funding from the Cures Act was designated toward the NIH’s Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative.4 The BRAIN Initiative is aimed at accelerating the development and application of innovative technologies in order to produce a way of visualizing the brain to show how individual cells and complex neural circuits interact in both time and space.7 This technology is expected to provide researchers with new ways to treat, cure, and even prevent brain disorders such as Alzheimer disease.
Development: A second key aim of the Cures Act is to facilitate the translation of new scientific discoveries into FDA-approved treatments. According to the House of Representative Energy and Commerce Committee, the Act will achieve this aim by4:
- Modernizing clinical trials and the means by which safety and efficacy data is accumulated and analyzed;
- Making the regulatory review process more patient-centered;
- Supporting the development, qualification, and utilization of biomarkers, which help assess how a therapy is working, and on whom, earlier in the process;
- Streamlining regulations and providing more clarity and consistency for innovators developing health software and mobile medical apps, combination products, vaccines, and regenerative medicine therapies;
- Incentivizing the development of drugs for pediatric diseases and medical countermeasures, and empowering the FDA to utilize flexible approaches in reviewing medical devices that represent breakthrough technologies; and
Providing the FDA with $500 million for regulatory modernization and to improve the agency’s ability to recruit and retain the best and brightest scientists, doctors, and engineers.
Delivery: The third aim of the Cures Act is to ensure that new drugs and devices are delivered to the right patients at the right time. This includes ensuring that electronic health record systems are interoperable, supporting the evolution to a learning health care system, and helping to facilitate seniors’ access to the latest medical technology. Additionally, the Cures Act includes provisions to improve education for health care providers in order to increase patient access to the best available treatments.4
FDAMA 114
Another renewed area of focus within the FDA falls under Section 114 of the FDAMA, dealing with the promotion and use of treatments including preapproval and off-label.3 Although this law was passed in 1997, only in January of this year did the FDA release guidance regarding the ability, or lack thereof, of manufacturers and payers to communicate in two specific areas.8 One area is in the post-approval setting, with regard to the ability of the manufacturers to share healthcare economic information with formulary decision-makers and other entities commonly referred to as payers. Another focus deals with the ability of pharmaceutical companies to be able to in a very specific and targeted way share information 12 to 18 months in advance of a product’s approval with payers.
Meaning for Clinical Pathways
The initiatives in the Cures Act suggest a trend toward increased availability of treatment options as well as more data on specific patient targets to optimize outcomes. These developments would lead to increased complexity for clinical pathway developers, with more treatment options to compare and more data to consider. At the same time, the FDA guidances regarding FDAMA 114 will likely mean that more information regarding treatments, and particularly with regard to health economics and outcomes data, will be made available to the payer community. However, the guidances released in January are only a first step toward this. Furthermore, additional clarification is needed with regard to whether clinical pathway developers are considered payers.9
Together, the direction that the FDA is expected to take under the Trump Administration will likely lead to greater complexity for clinical pathway developers. If all goes well, these complexities should result in improved clinical and financial outcomes as a result of the availability of more treatment options and with more specific information on the real-world outcomes of using these treatments in specific patient populations. The more specific “right” drug for the “right” patient should be better obtained under this new FDA. However, the execution of these changes, as well as how clinical pathway developers and users adapt to these changes, will be crucial.
References
1. About FDA—Meet Scott Gottlieb, M.D., Commissioner of Food and Drugs. FDA website. fda.gov/AboutFDA/CentersOffices/ucm557 569.htm. Updated May 18, 2017. Accessed June 4, 2017.
2. 21st Century Cures Act (Public Law 114-255). December 13, 2016. https://www.congress.gov/114/bills/hr34/BILLS-114hr34enr.pdf. Accessed June 4, 2017.
3. Food and Drug Administration Modernization Act of 1997 (Public Law 105-115). November 21, 1997. gpo.gov/fdsys/pkg/PLAW-105publ115/pdf/PLAW-105publ 115.pdf. Accessed June 4, 2017.
4. The 21st Century Cures Act: An innovation game-changer, a once-in-a-generation, transformational opportunity to change the way to treat disease. House of Representatives Committee on Energy and Commerce website. https://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/documents/114/analysis/20161128%20Cures%20Fact%20Sheet.pdf. Published November 28, 2016. Accessed June 4, 2017.
5. The White House, President Barack Obama. The Precision Medicine Initiative. https://obamawhitehouse.archives.gov/node/333101. Accessed June 4, 2017.
6. National Cancer Institute at the National Institutes of Health. Cancer Moonshot. https://www.cancer.gov/research/key-initiatives/moonshot-cancer-initiative. Accessed June 4, 2017.
7. What is the BRAIN Initiative? National Institutes of Health website. https://www.braininitiative.nih.gov/ Accessed June 4, 2017.
8. Drug and device manufacturer communications with payors, formulary committees, and similar entities – questions and answers. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM537347.pdf. Published January 2017. Accessed June 4, 2017.
9. Costill D. FDA releases draft guidance on communication between pharma, payers. First Report Managed Care website. https://www.managedhealthcareconnect.com/content/fda-release-draft-guidance-sharing-hcei-between-pharma-payers. Published January 19, 2017. Accessed June 4, 2017.
