Despite tremendous progress and knowledge gained from post-approval R&D, some in Congress still fail to recognize innovation doesn’t stop at U.S. Food and Drug Administration (FDA) approval. After approval, scientists and physicians continue to generate information on a new medicine or vaccine, as we’re seeing with COVID-19 vaccines today. This is done both for approved indications and to develop the medicine potentially for new, supplemental indications treating important unmet medical needs.
The latest drug pricing plan gives the government a new authority to set prices for medicines in Medicare after a product has been on the market for 7 (small molecule drug) or 11 (biologics) years. They call it “negotiation,” but it isn’t that at all. The latest plan not only includes some of the same price setting policies as H.R. 3 which threatens access to medicines, but it also guts the critical incentives necessary to support further investment in R&D after medicines are approved.
Today, we’re setting the record straight on why post-approval advances are so important and should be encouraged, not discouraged.
1. R&D continues long after a medicine is approved and first reaches patients.
Medical advances for patients often don’t end with initial FDA approval. Rather, researchers apply the knowledge they’ve gained to inform additional research on FDA-approved medicines. This research is time and resource intensive and scientifically challenging, often requiring additional clinical trials and significant data collection before the FDA grants any additional approvals or product label updates.
Many of these advances that occur following initial FDA approval have resulted in increased survival rates, improved patient outcomes and enhanced quality of life for patients with cancer, autoimmune diseases and rare diseases, among others. Whether discovering a new medicine or finding new ways to manufacture, administer and advance previously approved treatments, biopharmaceutical companies are committed to finding treatments and cures to help patients live longer, healthier lives.
2. Post-approval research often leads to lifechanging improvements for patients.
Once a medicine is approved, researchers take their work even further to assess whether there may be additional benefits for patients. In some cases, additional research determines that the medicine can also be used to treat different states of the same disease, such as earlier stages of cancer. Additional research may also demonstrate the medicine can be used to treat completely different conditions including different forms of cancer, or different diseases altogether. And in other cases, additional research may lead to increased safety or effectiveness or new dosage forms or forms of administration of a medicine that can improve patient adherence or convenience, leading to better patient outcomes. These are significant improvements that simply would not exist without post-approval research.
3. Congress has long recognized the importance of post-approval research to address unmet patient need.
To help stimulate the development of treatments for additional unmet medical needs and encourage companies to conduct the significant and challenging additional research required, Congress has implemented policies that help support post-approval biomedical R&D. Policies such as the Best Pharmaceuticals for Children Act (BPCA) and the Orphan Drug Act (ODA) were passed by Congress to encourage investment in areas of high scientific uncertainty and significant unmet patient needs and have resulted in important benefits for patients.
The BPCA incentivizes companies to conduct studies for children beyond studies related to the adult indications for the medicine by providing an additional 6 months to existing exclusivity for the medicine once pediatric studies have been conducted and evaluated by FDA. The incentives in the BPCA encourage sponsors to invest in pediatric research despite the challenges of conducting pediatric research including smaller disease populations compared to adults, selection of appropriate dose levels, blood volume and tissue sampling restrictions, impact on school and family life and obtaining informed consent. Since 2012 over 68 products have been studied under BPCA.
Since the enactment of ODA in 1983, the FDA has approved more than 600 medicines for rare diseases, in contrast to fewer than 10 medicines for rare diseases in the decade prior to its enactment. Many of these indications were developed for medicines that were previously approved for use in other populations or in other diseases. Despite these recent treatment advances, rare and pediatric diseases remain an area of high unmet need. Currently more than 90% of rare diseases, including those that impact children, have no available treatment option.
Given the importance of post-approval advances to so many patients, it is beyond disappointing some in Congress are pushing a price setting policy that threatens it, ultimately leading to fewer innovations which so many patients are depending on. Now is not the time for Congress to undermine scientific innovation.