America’s biopharmaceutical companies are at the heart of a robust research and development (R&D) ecosystem that develops more innovative medicines than any other country in the world. In recent years, rapid advances in scientific discovery have ushered in a new era of medicine, transforming our ability to treat, and in some cases cure, some of the most challenging diseases, including cancer, rare diseases and autoimmune conditions. These advancements are due to the productivity of the United States’ biomedical research ecosystem, which is sustained by a policy framework that is designed to support and advance America’s leadership in the innovation of new medicines.
Central to the success of this ecosystem is the leadership that comes from America’s biopharmaceutical research companies.
Some critics have claimed that this success is because the National Institutes of Health (NIH) use public funds to discover new therapies which are then just handed off to biopharmaceutical companies to be manufactured, packaged and monetized. This fundamental misunderstanding of the way drug development works has led to policy proposals that could seriously harm the U.S. research ecosystem and jeopardize its longstanding success. Now more than ever, it is critical that both public and private assets can be brought to bear in addressing critical diseases such as COVID-19.
Here are five things to know about the development of innovative medicines:
1) Basic science research is conducted by both the public and private sectors and lays the foundation for our understanding about how the human body functions. The goal of basic science research is to understand the function of newly discovered molecular compounds and cells, strange phenomena in the body or little-understood disease processes. Academic, government and private industry scientists all contribute to the vast body of basic science research, and that knowledge is shared and expanded upon by scientists through peer-reviewed publications, scientific meetings and licensing of intellectual property (IP).
2) Because the NIH conducts limited research specifically related to drug development, without the investment of the biopharmaceutical industry the knowledge resulting from basic science research supported by NIH would generate many ideas for potential drugs and drug targets – but very few new medicines. The biopharmaceutical industry’s unique role in the research ecosystem is to utilize its scientific and industrial expertise to recognize the best scientific opportunities and take the necessary risks to build upon and further advance basic science research into safe and effective treatments that can be made available to patients. The federal government cannot effectively research, develop and manufacture vaccines and new treatments without the resources, scientific expertise, R&D, manufacturing and technological platforms from private sector biopharmaceutical companies.
3) Much of the success of the U.S. research ecosystem is due to the positive impact of the Bayh-Dole Act on public-private research collaboration. Congress passed the Bayh-Dole Act in 1980 with bipartisan support to incentivize the private sector to make the substantial and risky investments needed to transform discoveries from government-funded basic research into useful products. Bayh-Dole has helped lay the foundation for the robust and entrepreneurial U.S. R&D ecosystem. Prior to enactment of the Bayh-Dole Act, the government retained the patents on federally-sponsored inventions – and only 5% of those patents were ever used in the private sector. The reason the U.S. is the global leader of biopharmaceutical innovation is because the IP system promotes competition by ensuring each player excels at their role and is incentivized to take risks and share information throughout the process.
4) It is not solely a question of dollars invested. NIH has a critical public health mission to uncover new knowledge that will lead to better health for everyone – and we should keep it that way. Imagine the loss for the advancement of public health if the NIH was solely focused on developing new therapeutics. Through the research grants NIH provides, not only is basic science research advanced, these funds also have a critical role to play in training of future scientists, developing and supporting medical libraries, training medical librarians and other health information specialists, and educating on the importance of prevention for maintaining good health. NIH-funded studies are critical for understanding the natural history of diseases, identifying critical biomarkers, and establishing clinical guidelines for best standard of care. Private sector companies regularly collaborate with NIH by providing funding and drug supplies, contracting with clinical trial networks to run industry-sponsored clinical trials, and providing scientific expertise to those networks through advisory committees. And, similar to the way NIH cannot fulfill all of the responsibilities of the industry, the industry cannot fulfill all of NIH’s responsibilities. Each member of the biopharmaceutical ecosystem plays a unique and vital role.
5) Policies that reduce incentives for the private sector to invest and take risks could have serious unintended consequences for future innovation.
In the face of the COVID-19 crisis, PhRMA member companies have committed to collaborating with a wide range of partners including working with various governments to ensure that when new treatments and vaccines are approved, they will be available and affordable for patients. Concerns that the biopharmaceutical industry will inappropriately price future vaccines or treatments are unfounded. During public health emergencies such as pandemics, the biopharmaceutical industry has a track record of responsible pricing and actively partnering with the government to ensure availability and affordability. Dr. Anthony Fauci has said he is not aware of any situation where companies priced vaccines out of reach in pandemic situations.
Rather than harming the highly successful U.S. biopharmaceutical research ecosystem and the patients who need innovative treatments, we should look to policies that will support patient access and affordability without undermining the development of tomorrow’s life-saving medicines. Through thoughtful, market-based approaches we can continue to support a thriving biomedical research ecosystem and allow the biopharmaceutical sector to continue to partner with the public sector to deliver innovative medicines and improve the lives of patients in unprecedented ways.
Explore more about the innovative biopharmaceutical ecosystem here.
Jocelyn Ulrich Jocelyn Ulrich, MPH, is Deputy Vice President, Policy and Research, at the Pharmaceutical Research and Manufacturers of America (PhRMA). At PhRMA, she is responsible for developing legislative and policy analysis and research studies on a range of issues impacting innovative biopharmaceutical companies including intellectual property issues, FDA policy issues, the R&D process, the value of innovation, and other issue areas impacting the environment for innovation. In addition to her experience at PhRMA she has over 15 years of experience in the pharmaceutical industry at Pfizer, Human Genome Sciences, and EMD Serono in roles in clinical research management, investigator-initiated and collaborative research, and global policy and corporate affairs. Jocelyn holds an MPH in global health policy and management from New York University.