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Margaret Anderson
Executive Director, FasterCures
Bioscience and Health and Medical Research and Regulation and Science
Margaret Anderson is executive director of FasterCures/The Center for Accelerating Medical Solutions, defining the organization's strategic priorities and positions on key issues, developing its programmatic portfolio, and managing its operations. Prior to her appointment as executive director, she was FasterCures' chief operating officer for five years.
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Rx for Innovation: It is Time to Get to Work for Biomedical Research

By: Margaret Anderson
November 11, 2016
   
   

We are in an exciting time for biomedical innovation, but there is still much to be done to realize science’s promise. President-elect Donald Trump will need to work to ensure an environment where innovation and ingenuity here in the United States continue to thrive. And we have some ideas about where to start.

Through our Rx for Innovation project, FasterCures interviewed more than 150 key stakeholders from more than 130 organizations across all sectors of the R&D ecosystem. We asked interviewees to identify opportunities for the next president to advance biomedical innovation.

The ideas we heard were cogent, thoughtful and direct. Interviewees conveyed a sense of excitement about the possibilities and potential that lie ahead, as well as the urgency to take action that ensures we build on recent progress. Most importantly, they called for a movement to encompass all of us and create a #HealthCitizenship enterprise where individuals engage more actively in the innovation system, resulting in a system which more effectively embraces the needs of its citizenry.

Our months of conversation were analyzed, synthesized and ultimately digested into 26 discrete recommendations. While this provides a hefty to-do list for President-elect Trump and his Administration, we think that each of these steps is critical to ensuring an environment that nurtures and delivers patient-centered innovation.

We broke the recommendations into these categories:
1. System-level action
2. Patient centricity
3. Regulatory resources
4. Translational science
5. Clinical trials
6. Data
7. Access to innovation

“You want change? You have to look at who are the drivers and bring all of them to the table and look at all the intended and unintended consequences.” - Regulatory expert in a Rx for Innovation interview

Biomedical innovation is vital to America’s health and economic well-being. Indeed, the life sciences promise to be as transformative to the early 21st century U.S. economy and society as the Internet was to the late 20th century.

While our diverse interviewees expressed equally diverse views, one theme that emerged was the need to make the system work as a system. This system would fund strong basic, translational and clinical research, while also acknowledging where there are gaps and roadblocks that need attention (e.g., clinical trials). This system would create tools and policies that enable data to be gathered and analyzed in the most efficient way, while providing a robust budget and workforce for regulatory decision-making. Most importantly, this system would look at how we are going to provide the best health outcomes for our citizens while maximizing access to innovation and value to individuals and society.

Such a system requires us to keep our eye on the whole enterprise, end our piecemeal approach and think about this system as a whole. The next Administration can bring the idea of this interdependent system to the fore; doing so is essential to making the serious headway needed to ensure we are prepared for the challenges to the research enterprise that lie ahead.

“Let’s get into the weeds about how patient engagement could be harnessed as a force for innovation.” - Health policy expert in a Rx for Innovation interview

Patients need to be involved in the entire research process by, for example, identifying the unmet needs of medical product development and risk assessments, and weighing in on how we define value for the innovations of the present and the future. To achieve such patient- centricity, we must invest in developing and advancing the science of patient input to ensure all stakeholders can benefit from and realize these opportunities of patient engagement.

We propose the White House take the lead by hosting a “Health Citizenship Summit” by the end of 2017, which would bring together thought leaders from all relevant agencies, including representatives from the National Institutes of Health (NIH), Food and Drug Administration (FDA), Centers for Disease Control and Prevention, Centers for Medicare and Medicaid Services, Health Resources & Services Administration, Agency for Healthcare Research and Quality and the Department of Health and Human Services (HHS) headquarters. External stakeholders representing each sector of the biomedical ecosystem, including patients, payers, providers, academia and industry would also be invited to participate. The summit would be a venue for stakeholders to roll up their sleeves and hash out solutions to the challenges we face as a citizenry. In fact, many of our report’s recommendations across all seven themes reflect this spirit of #HealthCitizenship.

“There’s still a resource issue [at FDA] - it’s difficult for them to get ahead, [there is] not enough in their budget.” - Investor in a Rx for Innovation interview

Future innovation requires a strong regulatory system prepared to address new scientific and technical challenges. As one interviewee noted, “People need to let go of preconceptions and learn about what the FDA is and how it works.” Strong, uninterrupted leadership from a confirmed FDA commissioner is critical to ensuring such strength.

At FasterCures, we believe that the current commissioner, Robert Califf, has precisely the leadership skills and technical expertise that the agency needs, and we would support his continuation in this mission-critical role. Regardless, we encourage timely nomination of an FDA commissioner within the first 100 days.

FDA leadership will also need President Trump to focus on increasing the agency’s budget and strengthening its workforce. Without resources (financial and human), it will be difficult for the agency to stay ahead of the innovation curve.

“How do we modernize the research infrastructure?... At the end of the day, infrastructure is not sexy, but it is critically important.” - Research policy expert in a Rx for Innovation interview

Many of the people we spoke with cited the need to support robust science funding - of both basic and translational biomedical research - and to support efforts to enhance its underlying infrastructure. Infrastructure is likely to play a prominent role in President Trump’s agenda, and we urge the Administration to think of the biomedical innovation infrastructure in the same category as bridges and roads. Without strong infrastructure to conduct and support the science, we will not be able to deliver on all the promise and potential of our country’s researchers.

Systems don’t happen overnight; they require ongoing cultivation, funding and focus for the biomedical research system to deliver on its tremendous promise. We advocated for the creation of NIH’s National Center for Advancing Translational Sciences and encourage strong support for this entity and other initiatives aimed at bridging the translation gap. Beyond increased research dollars, the next Administration can support NIH by becoming adept at communicating the many benefits - to the country and the world - derived from every taxpayer dollar invested in NIH.

“With clinical trials research, we need a better infrastructure, more trained people... I would start by convening a blue ribbon panel... You really want the best.” - Regulatory expert in a Rx for Innovation interview

Our recommendations address the need to streamline clinical research. The next Administration can’t punt on this challenge.

We need to create a revolution in clinical trials through a focused effort to leverage new technologies and accelerate existing efforts across the public and private sectors. To that end, we propose a “Clinical Trial Task Force,” chaired by the director of the White House Office of Science and Technology Policy, which would focus federal activity on advancing the tools, technologies and methodologies that underpin this essential piece of infrastructure. It would also provide a platform for the government, academia and industry to facilitate collaboration across sectors. There is certainly precedent for this high-level engagement of the White House: Cancer Moonshot, meet Clinical Trials Task Force.

“Data is the new raw material that is being hoarded.” - Patient advocate in a Rx for Innovation interview

Patient and disease foundation stakeholders see data as ripe for disruption, and several pointed to Uber and Amazon as real-world examples of disruptors showing how fast an industry can change.

The system needs to reflect on how we incentivize data sharing in academia and industry. Now that electronic health records adoption is widespread, the government must ensure that meaningful interoperability follows. The Office of the National Coordinator for Health IT (ONC) has obtained “interoperability pledges” from major health systems and health IT vendors. ONC’s Sync for Science and Blue Button efforts also promote interoperability and movement of data. However, government needs tools to effectively enforce these promises.

FasterCures feels strongly that now is the time for 21st Century Cures to become law. However, if it is not enacted before Inauguration Day, President Trump should support legislation similar to the 21st Century Cures provisions that would prevent health data blocking and empower HHS to investigate and seek civil monetary penalties from offenders (H.R. 6 and S. 2511 in the 114th Congress).

“Because of technology and where science is, we are capable of doing all kinds of great things... We have not figured out how to pay for it. That may be the most important thing [the next] Administration has to address.” - Research policy expert in a Rx for Innovation interview

We heard from those who felt it won’t matter whether we continue to bring forth innovation if we cannot pay for it. The national dialogue about the cost of health care and drug pricing is reaching a deafening level, and concerns and ideas about how to address these issues surfaced in numerous interviews.

We need both innovation and affordability, and payers, providers, industry and patients need to be part of an adult conversation about how we can take the health-care dollars currently being spent in our system and provide better care for all. We all need to examine the factors contributing to America’s health-care spending and get engaged. That means looking at better health outcomes, asking patients what matters to them and factoring that input into decision-making.

To date, patients have not been meaningfully brought into these decisions. There are enough vested interests in the system to go around, so finger pointing is wasting time. Everyone needs to analyze how they are evolving the discussions on plans, pricing, product and outcomes.

“If you were designing government from scratch, would it look as it does today?” - Patient advocate in a Rx for Innovation interview

This is a tough question to answer, but one interviewee thought it was important to have the next Administration reflect on this. The key thing we gathered from the more than 150 interviewees was the need for bold and purposeful movement forward and not being afraid to tackle big, audacious challenges.

The enterprise is ready for systems-level thinking about solutions and acting on those proposals. Every stakeholder is ready to raise his or her hand, brainstorm strategies and solutions and execute on the best ideas. Finding better solutions for the patients of today and those to come tomorrow should be a non-partisan area of agreement. FasterCures is ready; we’ve started by putting forward these 26 recommendations. Onward.

Sources:

Office of the National Coordinator for Health Information Technology, Interoperability Pledge, https://www.healthit.gov/commitment (accessed October 19, 2016).

Josephine Briggs and Josh Mandel, “NIH and ONC Launch the Sync for Science (S4S) Pilot: Enabling Individual Health Data Access and Donation,” HealthITBuzz, March 21, 2016, https://www.healthit.gov/buzz-blog/health-innovation/nih-and-onc-launch-the-sync-for-science-pilot/ (accessed October 19, 2016).

Office of the National Coordinator for Health Information Technology, https://www.healthit.gov/patients-families/your-health-data, accessed October 19, 2016.

https://www.congress.gov/bill/114th-congress/house-bill/6, accessed October 19, 2016.

https://www.congress.gov/bill/114th-congress/senate-bill/2511, accessed October 19, 2016


This blog post first appeared in The Huffington Post.