Day 173: The Federal Policy Landscape, and Why It Matters to You
The uncertainty in the regulatory environment is a roadblock to faster cures, according to Mark Alles, CEO of Celgene. Not knowing what endpoints will be acceptable to regulators over time prevents industry from accelerating what works.
Involving Patients in Determining Value
Cynthia Rice, senior vice president of advocacy and policy at JDRF, believes that patients can ease uncertainty by helping define the measures that should be covered by insurance and the outcomes that the U.S. Food and Drug Administration (FDA) should measure. "We need the government to play a partnership role with patient organizations, the private sector, and more," Rice said. Marc Boutin, CEO of National Health Council, agreed that the voice of the patient must be represented in drug development and in the delivery of care.
Former FDA Commissioner Robert Califf recommended "good, outcomes-based" clinical trials as a way to shape a value-based system. He went on to caution the panel about granting early access too broadly. He expressed concern that the right mechanisms are not in place to ensure treatments given early access are appropriately monitored and studied to prove their long-term efficacy.
"Continuous fabric of data across time is what you need to make conclusions about health care over a lifetime," Califf said. He noted that people spend less than one percent of their lives in a clinic or hospital. It's important for the system to gather, access, and use data from time spent outside of a health-care center in a meaningful way.
Boutin took this idea a step further, asking, "How do we capture data in a meaningful way to create a health-care system where value is not just used in the short term, but in the long term?" He suggested that evaluating the value proposition over a lifetime would lead to a system focused more on prevention.
Alles pointed out that the heterogeneity among disease groups makes it very difficult to have one definition of value. He recommended a regulatory framework with some flexibility to define value by disease area.
The Future Surrounding Biomedical Innovation Policy
Alles believes that a value-based system is the future. "In five years, this conversation will be totally different," he said.
Califf echoed this sentiment that change is on the horizon, positing that the tech industry will be a key driver of innovation. "You're going to see an investment in health that dwarfs anything that's ever been seen before," Califf said, citing the FAANG (Facebook, Amazon, Apple, Netflix, and Google) companies as examples.
Califf suggested that voice recognition and automation, like Alexa from Amazon, will allow care providers and patients to spend more meaningful time speaking with each other, while other technologies will enable more clinicians to participate in hands-on health care.
Tirrell concluded the panel by asking all participants to share one wish for the federal policy system.
Alles wished for a bipartisan way to look at health care. Rice wished for continued, strong, bipartisan support for funding the National Institutes of Health. Califf wished for a health system that brings people together instead of dividing them. Boutin wished for a multi-stakeholder consensus of what value is in the long-term.
With the increasing pace of biomedical innovation and the rapid rise of technology, the P4C audience will not have to wait long to see if those wishes come to fruition.
CEO, National Health Council
Vice Chancellor for Health Data Science and Director of the Center for Integrated Health Data Science, Duke Health; Donald F. Fortin, MD Professor of Cardiology, Duke University School of Medicine
Senior Vice President, Advocacy & Policy, JDRF