The bipartisan 21st Century Cures initiative, launched by Republican Energy and Commerce Committee Chairman Fed Upton (R, MI) and Congresswoman Diana DeGette (D, CO), is turning into a high-tech town hall on biomedical innovation, with tech companies, patients’ groups, drug companies, and hospital systems all reflecting on barriers to innovation—and how to overcome them.
One persistent theme, so far, from the Energy and Commerce (E&C) hearings is that innovation is a team sport. Private industry can only reach its full potential with a steady flow of new scientific discoveries that it can translate into new technologies. Support for government investment increases when basic research delivers tangible benefits in the form of better health, longer lives, and high paying jobs generated by a robust biotechnology industry.
And both industry and academic researchers benefit from an FDA that can promote public health by embracing new tools that ensure that safe, effective new products are swiftly delivered to patients.
The E&C hearings have already generated a wealth of ideas for advancing “team-based” innovation – here are a few of the biggest ones:
- Update FDA regulations to harness the full potential of modern technologies to advance targeted treatments to patients. The current framework for FDA approval of new medicines was created in 1962. Today, according to the Tufts Center for the Study of Drug Development, it costs an average of $1.3 billion and as much as 15 years to develop a single new FDA-approved medicine. Only about 1 in 3 medicines that make it to market even recoup their R&D costs. The enormous regulatory uncertainty and sunk costs facing innovators translates into lost opportunities to use cutting-edge science to advance state-of-the-art care, while resulting in higher prices for FDA approved medicines. While the agency has moved admirably to speed up development for drugs for AIDS, cancer, and some orphan diseases, many other indications lag behind.
We need a new development paradigm that allows us to deliver customized cures to patients much faster and less expensively than is currently possible. One approach is to expand the FDA’s current accelerated approval pathway, where drugs are approved earlier in the development process, based on surrogate endpoints or “biomarkers” that give an early read on whether the drugs are reasonably likely to deliver clinical benefits later. Congress could require FDA to pilot an expanded system like this for every disease with serious unmet medical need, particularly in targeted groups of patients. In fact, this exact approach was recommended by President Obama’s Council of Advisors on Science and Technology in 2012.
- Make patients true partners in advancing medical research. Today, far too much data that could be used to advance personalized medicine is lost because health care systems can’t effectively share data, while providers are reluctant to pool data out of fear they will run afoul of federal regulations, such as HIPAA. Another research protection, the Common Rule, restricts researchers’ ability to go back and reanalyze data or tissue samples using new tools or diagnostics if they weren’t spelled out in the initial patient’s informed consent form. Patients must own and control their own health data, with appropriate privacy protections—but we should also allow broader informed consent forms that facilitate the pooling and ongoing analysis of de-identified clinical trial, outcomes data, and even data from the emerging field of wearable diagnostics. Enhanced data security and better research use of data can go hand in hand—ensuring patients directly benefit from their participation in medical research—helping to save lives now, as well as in the future.
- Ensure a stable, predictable source of funding for basic medical research. If you look at America’s most successful biotech hubs—from Boston to San Francisco—you see that NIH driven medical research at hospitals, universities, and academic medical centers creates the knowledge base that private industry uses to launch their search for new cures. Basic science, applied technology, and the profit motive work together to drive a virtuous cycle of medical innovation.
To sustain the industry’s pipeline of new products, NIH needs a predictable, sustainable source of funding, particularly to help young researchers launch their careers. Doubling the NIH’s budget again may not be in the cards (or even wise), but a predictable stream of funding would be immensely helpful. Pegging NIH funding to GDP growth would be one way of insuring steady funding, perhaps in tandem with additional reforms to ensure that NIH funded projects are, in fact, generating reproducible science.
- Develop robust incentives for innovators working on hard to treat diseases like Alzheimer’s. In addition to modernizing the FDA’s regulatory toolkit and supporting NIH’s critical research, policymakers must ensure that there are adequate market incentives to drive large, ongoing private investments in disease areas where the scientific hurdles are especially complex (like central nervous system disorders). For instance, proving that a drug or vaccine prevents Alzheimer’s might require clinical trials lasting decades—exhausting any product’s effective patent life. One simple solution would be to guarantee 12 or 15 years of patent life for any product that passed FDA approval for Alzheimer’s, schizophrenia, or Parkinson’s. This would help encourage companies that have exited the CNS space—like AstraZeneca and Novartis—to get back into the fight. Innovators need to know that if they embrace the financial and scientific risks inherent in developing new treatments, their investments will be able to reap significant financial rewards when they finally do reach patients.
There is no single “home run” policy reform that would fix the challenges facing innovators overnight. But Congress can build a framework of smart, interlocking policies that can accelerate patient access to better treatments; enable a rapid learning, data-driven health care system; and incentivize companies to tackle the most complex diseases.
Taken together, these policies could enable a quantum leap in personalized medicine and ensure U.S leadership of the field for decades to come.