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21st Century Cures: Revolutionizing the launch of new therapies

At a House Energy & Commerce Committee 21st Century Cures roundtable, Dr. Francis Collins, Director of the National Institutes of Health, remarked, “We are at risk of losing something which has been one of America’s greatest glories: our success in biomedical research.” While tremendous progress has been made to advance our understanding of disease, there are currently treatments for only 500 of the 7,000 rare diseases. Dr. Collins shared his vision for how the US can harness advances in science and technology, including supporting the next generation of talented investigators. He mentioned how energized he was by advances in medical technologies and the promise of personalized medicine. He concluded his remarks by saying, “We can fix this, but it will take the full power of all of you and the recognition that this needs to be […] a high priority for our nation.”

In reflecting on the work of 21st Century Cures to date, I think about how we as a nation can unite the ecosystem, enhance translational sciences, and revolutionize the launch of new therapies. How can we continue to build on opportunities and keep our footing as a global competitor in the area of life sciences innovation? The Energy & Commerce Committee’s 21st Century Cures draft discussion document released in January presented an early vision of a bold blueprint for modernizing the process around discovery, development and delivery of safe, effective therapies (see the February 3 Health Care Current).

The cost of getting new therapies to market has never been higher; the risk of failure for those who take on the risk is great. 21st Century Cures seeks to break through the silos within the research and development chain and throughout the approval and post-marketing stages. Its provisions aim to:

  • Incorporate patient perspectives into the regulatory process and help address their unmet medical needs
  • Build the foundation for 21st century medicine
  • Streamline clinical trials
  • Support continued innovation at our federal public health agencies
  • Modernize medical product regulation

It has been just over a decade since the US Food and Drug Administration (FDA) launched the Critical Path Initiative. This effort was a call to action to modernize the tools necessary to evaluate and make predictions around the safety, effectiveness and manufacturability of medical products. Results from the Orphan Drug Act of 1983 emphasize that legislative changes can also create a huge impact to the ability to get new cures to those who need them most. In 2014, the FDA Office of Orphan Products Development approved more orphan drugs than any year before, according to analysis done by the FDA Law Blog. To me, 21st Century Cures represents a similar opportunity that could spur collaboration and biomedical innovation. Ultimately, some of these priorities could improve health care quality and outcomes for Americans.

The E&C Committee members traveled around the country, hosting eight hearings and several roundtables, calling for public comments and listening to patients, providers, industry, researchers, government agencies and policymakers. The multi-stakeholder approach the Committee has taken to inform the 21st Century Cures draft discussion document is testament to the many good ideas and talented individuals focused on the five areas that 21st Century Cures addresses. These voices are captured in the draft discussion document.

In my opinion, one of the most exciting and promising aspects of 21st Century Cures is the precision medicine initiative. Precision medicine involves tailoring medical treatment to the individual characteristics of each patient. It results in an individualized approach that takes into account variability in genes, environment and lifestyle.

The human genome project that began more than two decades ago was a critical breakthrough in medicine. In recent years, it has led to game-changing technological advances, such as the ability to sequence genomes more effectively, advances in imaging and cognitive computing and the ability to aggregate large volumes of data to inform clinical decision making. These have all contributed to more personalized approaches to treatment.

But, the health care industry’s quest to harness genomics and analytics to support researchers and providers so they can discover new ways to get the right treatments to the right patients at the right time is not over. There are still many unknowns about the underlying causes of many serious diseases, what treatments work best for what individuals and why individuals with the same disease can progress at different rates. A focused precision medicine initiative could get us one step closer to finding those answers.

The draft discussion document is the culmination of almost a year of stakeholder engagement, but the task at hand is not over. With so many voices invested in the initiative, the Committee has a challenging road ahead. But, with the demand for value-based care growing, and the U.S. health care system transformation underway, the US appears ready to take on such a challenge. The road ahead involves incorporating the reactions and feedback the Committee receives into the next phase of the legislation, and for some of the provisions and placeholders, incorporating more detailed policies into the final version. Continued discussions on funding sources and federal budget impact will also be forthcoming before the legislation reaches the President’s desk. At this early stage, it is difficult to gauge the financial impact that these proposed ideas may have on government and the health care industry. A key question moving forward is, “What will the return on investment (ROI) be from these potential recommendations?”

I share the optimism of Dr. Collins that the U.S. can build on its past successes and prioritize biomedical innovation. I think the U.S. is ready to build upon the power of data, analytics, genomics and advances in care and collaborative research. Much of the groundwork has been laid through collaboration and consensus building to help meet the goal of bolstering global competitiveness and achieving improved health care quality and outcomes for Americans.

Read the entire Health Care Current here and subscribe to receive weekly updates.


Author bio

Dr. Cooper is the Global Health Care Sector Leader and the US National Inclusion Leader for Deloitte. She is a Deloitte Consulting LLP principal and most recently served as the Federal Health Sector leader for Deloitte in the United States. Previously, Terri served as lead client service partner for the National Institutes of Health (NIH) and the U.S. Life Sciences R&D practice leader. She has more than two decades of experience in the life sciences and health care industry and has provided a broad range of strategic advisory services. She holds a Joint Honors BS Degree in Chemistry/Pharmacology and a PhD in Pharmacology from the University of London.