We live in a time where the pace of biomedical discovery has never been faster and the opportunity for patients’ access to transformative medicines has never been greater. We are in the era of precision medicine, attacking disease with genetic therapies. This is an exhilarating time for patients, many of whom previously faced a future with little hope of getting better.
It’s also an exciting time for U.S.-based biopharma innovators, as we lead the world into the new frontier of medicine in an industry that employs 1.7 million Americans. Continuing to foster this culture of innovation will be key to ensuring that important new therapies reach patients in need.
As President Trump prepares to address the nation in his first State of the Union address, I’d encourage him to foster this momentum in several ways:
Support a productive, consistent, and transparent FDA. The Food and Drug Administration ended 2017 with more than 50 approvals, including the first gene and chimeric antigen receptor T-cell (CAR-T) therapies that could transform the treatment of cancer and other life-threatening — and often rare — diseases. Thanks to remarkable scientific advances and a positive regulatory environment, many more innovations stand at the cusp of FDA approval in 2018, including the first RNA-interference-based therapeutics that my company has pioneered. The president should talk about the importance of a strong and efficient regulatory process for our industry. We have observed important progress over the past year — legislation to streamline the clinical trial process, acceleration of the approval of generic medicines, and providing patients a stronger voice in regulatory decision-making. We should continue to build upon it.
Continue to select strong leaders for HHS. The president should feel free to take a victory lap on the leadership he’s already assembled at the Department of Health and Human Services FDA Commissioner Scott Gottlieb and Centers for Medicare and Medicaid Services Administrator Seema Verma both win high marks for results to date, and are tremendous leaders who have a strong, broad understanding of FDA and CMS and its many programs that support health care for Americans. Newly appointed HHS Secretary Alex Azar is an excellent recent appointment who will extend the strong leadership already in place.
Keep discussions on drug pricing grounded in facts. The trend on drug costs is moving in the right direction. CMS reports that spending on retail drugs increased by just 1.3 percent in 2016. Outside groups have affirmed this finding; the Altarum Institute recently reported a meager 1 percent increase in drug prices over the last year. Meanwhile, administration officials have taken important steps to help make prescription drugs even more affordable. Under Gottlieb’s leadership, the FDA approved 1,027 generic drugs and 56 novel drugs and biologics in 2017 — both record highs. Moreover, the FDA has taken numerous regulatory actions to facilitate the entry of generic versions of complex drugs, such as biosimilars. A robust generic drug market that can’t be gamed is a clear and proven approach to lower drug prices that also preserves innovation.
Support targeted reforms that benefit patients. Policies written in the last century should be modernized to change how our health care system pays for prescription medicines. Under Verma’s leadership, CMS has worked to lead on new value-based approaches to drug pricing. This approach will bend the cost curve in health care and help patients access therapies they need with out-of-pocket costs they can afford. But these efforts can only take us so far. It’s time to modernize federal rules so more patients enjoy the benefits of value-based pricing. CMS has also recently made significant changes to the 340B drug pricing program rules, which lower patients’ out-of-pocket costs. Here, too, more fulsome programmatic reforms would help lower out-of-pocket costs for patients.
Acknowledge that innovation is part of solving the opioid crisis. We need to consider new plans to stimulate research into and development of innovative treatments to treat pain and opioid addiction. This means increasing resources at the FDA, streamlining the drug development and review process for non-opioid alternatives, and establishing expedited approval pathways to speed the commercialization of non-addictive painkillers. The pharmaceutical industry responded to the HIV crisis with new medicines that saved lives. We’re prepared to do the same with the opioid crisis.
When it comes to biopharmaceutical innovation, America leads the world. The president has a unique opportunity tonight to advance policies that can cement U.S. global leadership for generations to come.
John Maraganore is CEO of Boston-based Alnylam Pharmaceuticals and chairman of the Biotechnology Innovation Organization (BIO), the world’s largest biotechnology trade association.
