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The incoming administration faces key decisions on drug testing and access as well as vaccine distribution challenges.
The new year began with a continued surge in illness and deaths due to COVID-19, along with high hopes for combating the virus with effective vaccines and therapies. Strong efficacy data and reassuring safety reports indicate that new vaccines can prevent disease, while also shifting the focus to the monumental difficulties involved in producing, packaging, shipping, and administering millions of doses as quickly and effectively as possible. The ability of biopharmaceutical manufacturers to develop and test a whole new type of vaccine in less than a year reflects the scientific and technological strength of the industry and biomedical research community, plus the critical financial and regulatory support from governments and international organizations.
Public health officials, however, face significant challenges in effectively delivering vaccines that require two doses and ultra-cold shipping and storage. The new preventives will go first to healthcare workers and to nursing homes; many populations will have to wait for more accessible products still in the pipeline. Meanwhile, state and local health departments need additional federal support for identifying priority vaccine recipients, delivering appropriate doses safely, and tracking these operations effectively.
With limited access to COVID-19 vaccines for months ahead, the research community will continue to test hundreds of new and existing therapies for potential in treating infected individuals. FDA has authorized emergency use for a few monoclonal antibodies and antivirals found slightly effective against the virus, and investigators are testing dozens of antimalarials, immunosuppressants, and other compounds. Production is proving to be a roadblock, however, as manufacturers face high hurdles in providing sufficiently large quantities of medicines to treat more than a fraction of widening infections.
FDA’s ability to assess and communicate appropriate use of new COVID-19 vaccines and therapies will be critical for the agency to regain public trust and confidence in the objectivity and expertise of its review process. In 2020, FDA became entangled in Trump administration efforts to speed new treatments to patients based on limited and often subjective information, prompting strong push-back from respected agency officials and furthering public mistrust regarding new vaccines. It is vital for new leadership to navigate carefully between promoting public access to potentially life-saving treatments and limiting expectations for unproven therapies.
In addition to dealing with COVID-19, FDA will continue efforts to move a broad range of promising new therapies to market through guidance development, accelerated approval initiatives, and efficient review and approval programs for innovator drugs, generic drugs, and biologics. FDA has seen a surge in R&D on new cellular and gene therapies, and more new biosimilars promise to facilitate access to important biotech therapies. In addition, FDA is negotiating new agreements with industry on updated user fee programs for drugs, biologics, generic drugs, and biosimilars, which need to be sent to Congress by the end of 2021 in order to be renewed by Oct. 1, 2022 when current programs expire.
The importance of reliable and flexible manufacturing processes for drugs and vaccines has gained more attention as the pandemic has brought to light global supply chain vulnerabilities. After years of prodding by FDA officials, more streamlined and responsive production methods for medical therapies may gain traction, along with strategies for better anticipating and resolving drug supply shortages. There is bipartisan support for boosting drug manufacturing in the United States, but agreement is needed on appropriate requirements and incentives.
FDA support for more streamlined clinical testing methods during the pandemic is expected to bring lasting changes to the clinical research process. Trial sponsors and investigators increasingly utilize digital health technologies to track data from remote testing sites, support virtual patient visits, and expand use of real-world evidence to identify trial participants and compare study responses to broader patient populations. While the randomized, placebo-based clinical trial will remain the gold standard for assuring product efficacy, standard protocols, common comparators, and outcomes assessments will gain wider acceptance. An important issue is how COVID-19 vaccine manufacturers gaining emergency authorization can continue placebo-based trials to gain at least a year of important safety data, and how early results will affect trials for additional vaccines.
FDA enforcement activities also face changes following a halt in most field inspections of drug manufacturing facilities due to the pandemic. Agency officials are under pressure to resume onsite inspections to better assess quality production issues needed to approve promising therapies for market. But there also will be greater reliance on alternative sources of information on the capabilities and regulatory standing of an operation.
The emergence of more COVID-19 therapies will continue to raise questions about pricing and access to vital treatments in the US and around the world. There is bipartisan support for a range of drug price control measures, including versions of the Trump administration’s reference pricing and rebate reform initiatives. Drug industry leaders insist that too-harsh price controls will limit the broad range of innovation and drug development that has been critical for combating the pandemic. There is general support for increased import of less costly drugs from Canada, but policies will remain difficult to implement due to safety concerns.
In addition to facilitating access to new drugs and vaccines, the new administration will need to address broader economic relief and public health measures as the pandemic continues. The Biden team has mapped out a broad health policy agenda that includes extension of Affordable Care Act (ACA) provisions for healthcare coverage, which may be shaped by an upcoming Supreme Court decision. Further expansion of Medicaid to low-income populations, reduction in health disparities, and lowering Medicare eligibility to age 60 as part of a public option coverage program are main goals, as well as rejoining the World Health Organization and other international public health and research programs. Pandemic control will remain the number-one challenge, while also setting the stage for broader review of healthcare coverage and access.
Jill Wechsler is BioPharm International's Washington Editor, email@example.com.
Vol. 34, No. 1
When referring to this article, please cite it as J. Wechsler, "Pandemic Control to Shape Biopharma in 2021," BioPharm International 34 (1) 2021.