This article is part one of a two-part series covering the highlights of the Galien Forum. Part 2 can be accessed here.
Last week I attended the Galien Forum, held on October 27 at the Alexandria Center for Life Science in New York City, which brought together top industry executives and FDA regulators to discuss a number of hot topics and future-oriented issues effecting the development and commercialization of medicines globally. The following summary of this day-long event attempts to capture some of the highlights and provocations issued from panel participants, as well as comments from FDA Commissioner Robert Califf taken from his keynote interview with Michael Rosenblatt, co-chair of the Galien Forum and senior partner at Flagship Pioneering.
Are Drug Prices Too High?
In a panel titled “Are prescription drug prices too high?” Rosenblatt suggested rephrasing the question to: “Are people paying too much out of pocket?” before describing current drug coverage as being less like insurance, and more like cost-sharing plans detached from care and outcomes. He disputed the idea of “consumerism” in healthcare, and gave a hypothetical example of an employer-insured individual with cancer receiving a drug – one prescribed without alternative choices – with a coinsurance amount that could easily lead to bankruptcy. “In this system of cost-sharing, the patient is the only party not at the table,” said Rosenblatt. “Deductibles and copays have gone up.”
Harold Paz, EVP health sciences, Stony Brook University and CEO of Stony Brook University of Medicine, pointed to a recent JAMA study which found that new drug launch prices rose from a median price of $2,115 in 2008, to $180,007 in 2021. Value-based arrangements tied to patient outcomes can be useful in driving drug adherence, avoiding downstream costs, and improving treatment results, said Paz, but only 11% to 12% of current drug contracting is value-based; the rest continues to be paid through a fee-for-service model, which “we have to move away from,” said Paz.
The fee-for-service model isn’t working, agreed Stanley Crooke, founder and CEO of the n-Lorem Foundation because patients aren’t involved in making drug value determinations. Regarding price increases on existing drugs, Crooke said there is “no way to put enough lipstick on that to make it anything but a pig,” adding that too much money is being spent on wrongheaded marketing and advertising. Crooke suggested that the FDA’s Risk Evaluation and Mitigation Strategies (REMS) model could be a starting place for designing new ways to direct resources toward patient outcomes.
Better Tools For Clinical Trial Diversity And Inclusion
During a panel on diversity and health equity, Maha Radhakrishnan, group SVP and chief medical officer at Biogen, described her company’s partnership with the National Minority Quality Forum (NMQF) to create an “index” that uses Medicare and Medicaid claims data and billing information to “find patients and enroll them in trials.” Real world evidence is an opportunity to supplement randomized clinical trials with real heterogeneous populations, said Radhakrishnan, (A report on real-world evidence generation in Alzheimer’s disease, and Biogen’s partnership with NMQF, is available here.)
Tracking disease incidence rates can be a simple and effective way to improve clinical trial diversity, according to panelists. Prevalence should be equal to participation, or in other words, disease incidence rates should determine clinical trial inclusion numbers regarding diverse populations, said Ariel Katz, cofounder and CEO at H1, a healthcare data platform company. Eliav Barr, head, global clinical development and chief medical officer at Merck Research Laboratories, noted the important role of data monitoring groups in helping to make sure that trial enrollment at Merck is sufficiently diverse.
During the Q&A session, a healthcare executive and audience member pointed out to the panel that lingering fears among African Americans about clinical trial participation due to historic wrongs such as the Tuskegee syphilis experiments, are vastly overstated by the media, a fact that has been confirmed elsewhere in survey data. “Black people don’t participate in trials because they aren’t asked. People who do participate say: ‘My doctor recommended me for it,’ said the audience member. The panel’s moderator, Katrina Armstrong, dean and EVP, Vagelos College and health sciences, at Columbia University, added that after adjusting for direct experiences of racism, the population least trusting of the healthcare system is white suburban women.
Califf On Accelerated Approval
The FDA’s Accelerated Approval program, which has been criticized post Aduhelm, will “come up” during Congress’s lame duck session, said Robert Califf, FDA Commissioner. The lame duck session begins following the November 8 election results and extends until January 3, 2023, when the 118th U.S. Congress will convene. As for Califf’s opinion of the program, he is “not in favor of backing off at all.” But he is also not in favor of unvalidated biomarkers. “‘Reasonably likely’ is not the same as proven to be true,” said Califf. “Drug companies haven’t done enough post-approval.” More evidence generation is needed post-market, and that evidence should be based on what was done pre-market, he said.
Asked about lessons learned regarding speed versus accuracy in vaccine approvals, Califf said there is a “difference between cutting corners and taking risks … the Twitterati doesn’t get that. If you wait for all of the evidence to come in, everyone will be dead.” Califf went on to praise his FDA colleagues — Jeffrey Shuren, Janet Woodcock, and Peter Marks, specifically — and assured audience members that Americans are in “great hands” regarding drug and device safety. However, he lamented the fact that Americans are, generally speaking, in terrible health. “In the league of high-income nations, we are in last place,” said Califf. “We’re going in the wrong direction on chronic disease,” adding that America needs a more integrated system that combines care with medical interventions. Not so much whether a treatment will work for a given patient, but getting the right treatment to the right person, which is “not FDA’s lane,” Califf added.