Is a "Disease-Free" World Within Reach?
Stronger partnerships between the public and private sector would help ensure patients have access to new medicines as a result of new discoveries. In a keynote speech at Research!America’s National Health Research Forum on September 7 in Washington, D.C., Mikael Dolsten, M.D., president of worldwide research & development, Pfizer, said the U.S. is a leader in worldwide R&D but sustained funding levels for the National Institutes of Health (NIH) and a regulatory system built for the 21st century are critical to build on our success.
The program featured panel discussions with top leaders in government, industry, patient advocacy and academia. Inspired by the Cancer Moonshot, Dolsten said he believes that the initiative’s goal of achieving more than a decade’s worth of progress in cancer research in less than five years should be applied to other disease areas in an effort to move towards a disease-free world.
The first panel, moderated by Marilyn Serafini, health care policy consultant, former award-winning journalist, addressed escalating health threats, such as the opioid epidemic.
“Clinicians like me were taught that you can’t get addicted to these medicines if you’re really in pain, and we thought they were safe and effective,” said Anne Schuchat, M.D., principal deputy director, Centers for Disease Control and Prevention. “It turns out that for a lot of types of pain, for chronic long-term pain, opioids are not safe and effective. As clinicians we have to do better. There's a huge culture of medical practice that needs to change.”
Gopal Khanna, director, Agency for Healthcare Research and Quality, said data will also play a big role in reversing the opioid epidemic. “It was the data we collected from the states that helped us recognize the opioid epidemic years ago. So data is really the underlying structure on which research will be conducted going forward,” he said.
“You can’t think about this just in terms of treatment and recovery,” said Governor Charlie Baker of Massachusetts. “You have to think about it in terms of prevention and education.” He added that medical, dental, nursing and pharmacy school students are required to take a course in opioid addiction therapy and pain management in Massachusetts which could be a model for other states.
“Across the board there’s just a real lack of sufficient education for clinicians and patients and everybody else in the ecosystem,” said Lucinda Maine, Ph.D., R.Ph., executive vice president and CEO, American Association of Colleges of Pharmacy. “On the research side, it goes everywhere from understanding pain better as a neurological phenomenon to finding some non-addictive strategies for addressing pain more effectively. We have to have more data. We have to have greater insights into genetics to understand when genetics begins to influence the use of these products and their addictive qualities.”
Former Congressman Patrick Kennedy said there’s also a lack of interest among medical professions to provide medication-assisted treatment, such as Suboxone, for opioid addiction. “Right now less than 10% of the providers in this country write for medication-assisted treatment,” Kennedy said. He agreed that the system of medical education needs to “change top to bottom” to better address the epidemic.
Seth Ginsberg, co-founder and president, Global Healthy Living Foundation, said he hopes the important research being done to address the opioid epidemic doesn’t come at the expense of millions of Americans who are experiencing “genuine and incredible” pain. “We can help people in pain and we can prevent people from getting addicted,” Ginsberg said. “And I believe that patient-reported outcomes could technically sit as a fulcrum between those sides.”
In his remarks, Scott Gottlieb, M.D., commissioner, Food and Drug Administration (FDA), also addressed the opioid epidemic and said FDA is exploring abuse-deterrent drug formulations, medically assisted therapies, and non-addictive pain products. “I think the biggest role that FDA can play in trying to address this crisis is in reducing overall exposure and helping solve the new addiction crisis,” he said. In order to reduce addiction, we need to reduce exposure. And the ways we’re going to do that is to make sure fewer prescriptions are written.”
Gottlieb also announced that the agency is modernizing the way it reviews new drug applications particularly those involving gene and cell therapy. “We need to be able to make sure that our policies match the complexity of the novel science that’s being harnessed to attack disease.” He added that the FDA plans to make patient participation in the drug development process more transparent and more systematic,
The second panel, moderated by Byron Pitts, anchor & chief national correspondent, ABC News, focused on efforts to speed medical progress utilizing new technologies and tackling health disparities among other approaches. Victor Dzau, M.D., president, National Academy of Medicine, said clinical research struggles to keep pace with innovation such as CRISPR/Cas 9 gene editing.
“I think technology, in terms of science discovery, is moving really quickly but what’s falling behind is adoption and practice. That’s where I think we need a lot of push, a lot of change,” Dzau said. Researchers should consider different clinical trial designs, such as adaptive trials, to help accelerate the pace of clinical trials, and find ways to integrate databases with clinical research to streamline the patient enrollment process, he added.
Mark McClellan, M.D., Ph.D., MPA, director, Duke-Robert J. Margolis, MD, Center for Health Policy, Duke University, said more is research is needed to ensure that new therapies are meeting individual patients’ needs. “A patient is not just someone with pain or someone who gets a procedure but [rather] a whole constellation of genetic pre-dispositions, behavioral and environmental influences, and preferences that influence what their treatment should involve,” he said.
Nancy Brown, chief executive officer, American Heart Association, stressed the importance of research to address the disproportionate impact of certain diseases, such as hypertension, on minority populations.
Others on the panel included, Joe Selby, M.D., MPH, Executive Director, Patient Centered Outcomes Research Institute, Joel W. Beetsch, Ph.D., Vice President of Patient Advocacy, Celgene, and Scott Whitaker, President and CEO, AdvaMed.
The third panel, moderated by Jeanne Cummings, deputy Washington bureau chief, The Wall Street Journal, tackled the question “is a disease-free world possible”?
William N. Hait, M.D., Ph.D., global head, Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson & Johnson, said halting the progression of disease is a pathway towards creating a world without disease.
“The earlier you can push back the diagnoses [of diseases], the better the outcomes will be.”
Gary Reedy, chief executive officer, American Cancer Society, said he is very optimistic that in the next 15 to 20 years, common cancers, such as breast or prostate cancer, will be either cured or managed as a chronic disease with further advances in precision medicine.
But Reedy added that researchers must study social determinants of health that impact medical progress. “If we can address some of these socioeconomic issues, education issues, access issues, that’s going to help a lot,” he added.
“If you want to reduce illness, it’s better to have it not happen in the first place,” said Francis Collins, M.D., NIH director. “Coming up with a universal influenza vaccine is going to be critical if we are going to avoid what could be the greatest threat to human life on the planet in the next five or 10 years. If we’re going to talk about trying to save lives, that’s a great place to put our investments.”
Collins also highlighted the All of Us Research Program, a precision medicine initiative that seeks to collect health data from 1 million Americans from diverse ethnic, socioeconomic and gender backgrounds. “It is going to give us a database of unprecedented depth in terms of environmental exposures, genetic factors, health behaviors,” he said. “This will be transformative.”
Others on the panel included Iris Loew-Friedrich, M.D., Chief Medical Officer, UCB, Mikael Dolsten, M.D., Ph.D., president of worldwide research & development, Pfizer, Ann Cary, RN, MPH, Ph.D., FNAP, dean, University of Missouri-Kansas City School of Nursing and Health Studies, and David Neal, CBE, FMedSci FRCS, Senior VP of Global Academic Research, Elsevier.