By Kenneth E. Thorpe
Thirty years ago, AIDS patients faced increasing symptoms and the risk of death while awaiting life-saving drugs that had shown enormous promise in clinical trials — but that were hung up in the FDA’s traditional approval process. In response, Congress sanctioned a new FDA “accelerated approval” pathway that gave patients earlier access to medicines. Lives were spared and our healthcare system finally had solutions to manage a disease that was ravaging our nation.
That same accelerated approval pathway has saved countless lives over the past three decades. Cancer treatments have been developed and delivered to patients more readily. Rare diseases once considered untreatable now have new standards of care that were previously completely unthinkable.
Despite this, the pathway is back in the news — and for all the wrong reasons. Congress recently passed legislation that changed the accelerated approval pathway in significant ways. The latest reforms could substantially weaken the pathway and its potential to bring future treatments down the pike. It’s puzzling that some elected officials are championing the Cancer Moonshot while at the same time undermining the pathway that’s facilitated earlier access to breakthrough cancer drugs.
Accelerated approval is reserved for treatments that address serious or life-threatening conditions for which there are currently no adequate therapies.
Under traditional FDA approval, new drugs don’t get a green light until tests demonstrate they produce a clinical benefit in patients — for example, better cancer survival rates. Under the accelerated approval pathway, however, medications are conditionally approved if they can demonstrate success at a “surrogate endpoint” — that is, a predetermined and measurable step that predicts a future clinical benefit. For example, if tests show a new cancer drug shrinks tumors, there’s good reason to think it will extend lives.
Crucially, if further required testing determines the medication has failed to deliver on its initial promise, its accelerated approval can be revoked. But in the overwhelming majority of cases — some 76.5% of accelerated approvals between 1992 and 2016 — confirmatory testing has led to what’s called conversion to traditional approval.
Compromising the accelerated approval pathway would lead to fewer new treatments, period.
Emerging biotech companies are responsible for about 80% of all experimental medicines in the drug-development pipeline. Yet these firms often lack the funds to see a drug all the way from creation to confirmatory testing. With accelerated approval, they can begin selling their medication sooner, thereby enabling them to financially sustain the final rounds of testing themselves.
Unfortunately, that might not be a workable business model much longer. The FDA’s top oncology regulator said he intends to begin withholding accelerated approval of new cancer drugs until confirmatory trials are already underway. As a result, two companies have already seen promising cancer treatments delayed because of this new policy.
Further concerns lie in the inconsistencies around access to drugs approved under the accelerated approval pathway. The Centers for Medicaid & Medicare Services (CMS), for example, has disclosed plans to pay less for accelerated approval drugs that haven’t completed confirmatory studies.
Equally worrying, CMS has issued a “national coverage determination,” that will prevent most Medicare beneficiaries from accessing an entire class of groundbreaking new Alzheimer’s treatments simply because they received accelerated approval.
All told, these misguided coverage restrictions will only delay access to FDA-approved treatments that could change the course of some of the nation’s most costly chronic conditions.
Kenneth E. Thorpe is a professor of health policy at Emory University and chairman of the Partnership to Fight Chronic Disease.