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 was hung up in the Food and Drug Administration’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 ravaging our nation.
That same accelerated approval pathway has saved countless lives over the last 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 unthinkable.
Despite this, the pathway is back in the news — and for the wrong reasons. Last December, Congress passed legislation that significantly changed the accelerated approval pathway. The latest reforms could substantially weaken the path and its potential to bring future treatments down the pike.
The end game of these reforms — and who stands to benefit from them — remains unclear. People living with serious illnesses certainly won’t benefit, nor will their doctors who are constantly looking for ways to better care for their patients.
Political leaders won’t benefit either. 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 has been an unsung hero of American medicine. It’s a narrowly tailored exemption to the FDA’s traditional approval process. Accelerated approval is reserved for treatments that address serious or life-threatening conditions without adequate therapies.
Under traditional 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.
Patients and their families managing the effects of cancer and other illnesses should have the right to try cutting-edge medicines. The tests required for traditional FDA approval can take years. That’s time many patients don’t have.
For patients living with chronic, progressive or irreversible conditions, accelerated approval provides great hope. In fact, for seriously ill and suffering patients with no other options, compassion demands accelerated approval.
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 — 76.5 percent of accelerated approvals between 1992 and 2016 — confirmatory testing has led to conversion to traditional approval.
Compromising the accelerated approval pathway would lead to fewer new treatments, period.
Emerging biotech companies are responsible for 80 percent of all experimental medicines in the drug-development pipeline. Yet, these firms often lack the funds to see a drug from creation to confirmatory testing. With accelerated approval, they can begin selling their medication sooner, enabling them to financially sustain the final rounds of testing themselves.
Unfortunately, that might not be a workable business model for much longer. Last fall, Richard Pazdur, the FDA’s top oncology regulator, said he intends to withhold 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. For example, the Centers for Medicaid & Medicare Services has disclosed plans to pay less for accelerated approval drugs that haven’t completed confirmatory studies. Similarly, Massachusetts, Tennessee and Oregon have sought to limit the access of Medicaid patients to accelerated approval drugs.
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. The dangerous precedent this sets for patients will be felt far beyond just the 6 million people with Alzheimer’s disease.
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.
President Biden needs to decide whether he wants to try to make good on his Moonshot’s ambitious goal of reducing the cancer death rate by 50 percent over 25 years — or whether it’s the Moonshot that’s going to die of a thousand bureaucratic cuts.