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Opportunities for the Next Administration to Advance Biomedical Innovation

12/08/2016


Biomedical innovation is vital to America’s health and economic well-being. President-elect Donald Trump has the opportunity to lead in this area and construct policies to maintain the system’s strength and productivity. FasterCures engaged our unique network of stakeholders across the R&D ecosystem to interview more than 150 thought leaders across eight sectors. We synthesized these insights into the “Rx for Innovation” report, which presents 26 recommendations for how the next administration can translate the ideas we heard into meaningful action.

Join FasterCures for a free Webinar to discuss “Opportunities for the Next Administration to Advance Biomedical Innovation,” on Thursday, Dec. 8, 2016, from 1 to 2 p.m. Eastern.

Speakers include:
Patrick White, President, Act for NIH
Mary Dwight, Senior Vice President for Policy and Patient Programs, Cystic Fibrosis Foundation
Margaret Anderson, Executive Director, FasterCures, a center of the Milken Institute (moderator)

Summary

The U.S. government plays key roles across the entire biomedical innovation system, from research to regulatory review to reimbursement. As a new president and administration prepare to take office in January, FasterCures convened leaders from the research and patient communities on Dec. 8 for a Webinar to discuss “Opportunities for the Next Administration to Advance Biomedical Innovation.” Executive Director Margaret Anderson was joined by Patrick White, president of ACT for NIH, and Mary Dwight, senior vice president for policy and patient programs at the Cystic Fibrosis Foundation (CFF).

 Rx for Innovation: Recommendations for the New Administration

Anderson began the discussion by introducing FasterCuresRx for Innovation project, which developed a set of recommendations for the incoming administration to improve the biomedical innovation system. Interviews of 152 thought leaders from across the system were synthesized into a report, released a few days after the 2016 election, making 26 recommendations across seven domains:

Innovation Recommendations VERT cropped

Next, Anderson turned the discussion over to White, who focused on opportunities for the National Institutes of Health (NIH).

NIH: Engine of Discovery

With a $32 billion annual budget, NIH is the world’s single largest funder of biomedical research. However, flat budgets from fiscal year 2003 to fiscal year 2015 resulted in NIH losing 22 percent of its purchasing power. A $2 billion increase in 2016 has begun to turn the tide, and ACT for NIH’s White talked about the critical need for steady, sustained growth in NIH’s budget and recommended 5 percent plus inflation as an appropriate annual increase. Because additional funding is not an end in itself, White argued that a stable, upward trajectory will enable NIH, and the scientists it supports, to both plan for long-term research activities and carry out higher-risk research – which are both critical to making the breakthroughs that will lead to new therapies.

White also spoke about the potential benefits of a more independent NIH that can more easily organize itself to reflect the scientific opportunities that lay ahead, in contrast with the current set of institutes and centers that are largely organized based on organ system or disease type. Part of that independence would be a stronger NIH director, with enhanced authorities to start innovative, cross-cutting initiatives using a larger Common Fund, and possibly a six-year term for the director, which would provide steady leadership.

All three speakers engaged in a discussion about how NIH can promote diverse types of research. There is a perennial debate on how NIH should divide its budget between basic and applied research. Approximately 52 percent of NIH’s research budget is directed at basic science, and the ratio of basic to applied science has stayed constant in recent years. Some researchers have called for an increase in the proportion of basic science funding, saying that fundamental breakthroughs in our understanding of biology are what lead to medical advances. However, White pointed out that Congress appropriates funding to NIH to accelerate the path toward new disease treatments, and that we need both basic science and more applied work to get where we all want to go. CFF’s Dwight offered that developments in the care and treatment of cystic fibrosis (CF) patients is an ideal case study: Years of fundamental research on the CF gene, lung and protein function, when combined with applied research on new therapies, have led to remarkable advances in the fight against CF in recent years.

Maintaining Momentum on Patient Centricity

A major topic of discussion was on how to continue integrating the patient perspective into biomedical research, product development and regulatory decision-making. All agreed that a bedrock principle of patient centricity is the development of safe and effective medical products. “Policies can help us reinforce the need for patients in these activities,” noted Dwight, who went on to discuss the need for building the science and methodologies for moving from disconnected anecdotes about patient experience into data that accurately reflect the holistic picture.

One avenue for achieving this goal is the maturing field of real-world evidence. While not intended to replace randomized controlled clinical trials, real-world evidence can provide a picture of how a new treatment is working (or if there are any unexpected side effects) in the broader population of people who receive the product during the course of clinical care. As part of the 21st Century Cures Act (H.R. 34, section 3022), FDA will initiate a study of how real-world evidence can be used to evaluate new indications for approved products, or satisfy post-approval study requirements. Other policies that promote data sharing and interoperability will also be needed for real-world evidence  to reach this potential by connecting data sets across diverse providers, payers, biopharmaceutical companies and researchers.

In addition to regulatory decision-making, patient centricity can also be an important tool for assessing the value of a medical product, and Dwight highlighted how the collection of post-approval real-world evidence has shown very positive outcomes for new CF therapies and has helped to increase access for patients. In a sentiment that all can support, Dwight noted, “We’re not done until everyone can benefit from innovation.” This is a reminder that, while there has been great progress on policies intended to promote biomedical innovation and patient centricity, there is still much work for all of us to do.