The Food and Drug Administration’s new leadership held a “listening session” on Thursday to learn more about the exciting new gene, cell and immunologic therapies being developed for rare diseases in U.S. research labs. You’ve probably seen or heard about many of them in your news feeds: Gene editing; CAR-T, targeted antibodies.
The excitement among the scientists invited to the session was palpable. We’re on the cusp of a new era, they said, made possible by new technologies developed in the wake of the Human Genome Project, the government-funded effort to map the human genome that created the possibility of developing new, targeted therapies.
The need is great. An estimated 25 to 30 million people, half of them children, suffer from what the FDA classifies as a rare disease — something that afflicts fewer than 200,000 people. But the physician-scientists invited to present their concerns to the FDA — most of whom either own or consult with start-up biotechnology firms — said over and over that the U.S. was losing out to China in the race to develop cures.
Why? Too much regulation, they said. A lack of flexibility on the part of regulators. A few alluded to the more significant problem: Funding for biotech firms developing new technologies is drying up because there is (and always has been) limited profit in bringing a new therapy to market for a small patient population. That’s why they wind up costing hundreds of thousands if not millions of dollars.
The other significant reality is that most of these new therapies are still experimental. Even those that make it through the FDA approval process do not achieve a miracle cure, but are only partially successful in improving outcomes — an incremental advance from the more traditional drug therapies that came before them.
What does this mean for the evidentiary standards at FDA? In the past, Vinay Prasad, the new head of the agency’s Center for Biologics Evaluation and Research, which oversees approvals of the new genetic-based therapies, had expressed skepticism about accelerated approvals based on surrogate markers or the lack of hard evidence in clinical trials for some rare disease drugs. But based on his comments this week, he seems to be on board with the biotech industry’s pleas for looser regulation.
What will this mean for patients and their families? What will it mean for the healthcare system and its finances? How should we be thinking about who will pay for all of these breakthroughs if and when they reach the stage when they're available to patients? And how should we be helping the families who have to deal with all of these realities?
The voices of patients and their families were mostly missing from the listening session. That’s why I am pleased to have on this week’s podcast a mother of two children, one of whom suffers from a terrible disease. Erica Olensky is struggling with all of these issues. She is a public relations executive with Finn Partners and a board certified patient advocate.
I hope you listen carefully to what she has to say. Her voice needs to be heard.
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