The powers that be in Washington are renewing their campaign to gain greater control over Americans’ ability to access life-saving drugs.
Lawmakers and regulators alike have decided to wage war on one of the few components of our health care system that works well — the Food and Drug Administration’s accelerated approval process. This regulatory pathway has saved countless lives by speeding groundbreaking medicines to market. Obstructing that pathway would be folly.
The FDA created the accelerated approval pathway in 1992 in response to the HIV/AIDS crisis. Patient advocates were demanding earlier access to promising treatments for HIV/AIDS and other life-threatening conditions.
Under accelerated approval, the FDA allows patient access to treatments for serious diseases before they’ve completed all the clinical trials required for traditional approval, which can take years. For example, the FDA can grant accelerated approval to a cancer drug the agency expects to demonstrate “clinical benefits,” such as improved long-term survival rates, based on indications that it will do so — like shrunken tumors in patients in clinical trials.
Via the traditional pathway, FDA approval for novel drugs takes an average of eight years. That’s too long for many patients. Non-small-cell lung cancer — the most common type — has a five-year survival rate of just 26%.
Accelerated approval can shave up to five years off the approval process, which helps get patients what they deserve — access to treatments as quickly as possible.
The pathway has been a resounding success. In the 30 years since its inception, over 250 drugs have received accelerated approval. Oncology treatments make up 65% of those therapies; about 42% treat rare diseases.
One study found that between 1992 and 2016, 76.5% of drugs available to patients on the accelerated approval pathway for at least five years went on to garner traditional approval. Only 10.3% were withdrawn following additional trials, with traditional approval pending on the remainder.
And data show that drugs approved via expedited review are typically more effective than medicines cleared via the traditional pathway.
New polling shows that people overwhelmingly favor accelerated approval. More than two-thirds of voters in Arizona, Georgia and Nevada “somewhat” or “strongly” support it. Seven in ten do so in New Hampshire.
So why are some lawmakers and bureaucrats dismissing these decades of positive results and attacking accelerated approval instead?
Earlier this year, Rep. Frank Pallone, D-N.J., introduced a bill that would effectively halt accelerated approval. Among other things, his proposed legislation would require a drug on the accelerated pathway to secure traditional approval within five years — or face removal from the market.
The FDA already has protocols in place to rescind a drug’s approval if it turns out to be ineffective or develops an adverse side-effect profile relative to benefits. This legislation could arbitrarily deprive patients of drugs that are working for them.
Pallone’s attack on accelerated approval is not the only one currently under way. In April, the Centers for Medicare and Medicaid Services announced they’d dramatically limit coverage of Aduhelm, an Alzheimer’s treatment that received accelerated approval from the FDA, to Medicare patients participating in additional clinical trials. CMS said it wanted more clinical data on the drug’s effectiveness.
Medicare almost always covers FDA-approved drugs. So the Aduhelm decision represents a dangerous precedent for patients’ ability to access breakthrough treatments. It undermines the legitimacy of the FDA and situates CMS as an additional drug review agency — a gatekeeper role for which the agency is ill-suited.
The accelerated approval pathway stands in stark contrast to the red tape and bureaucratic backups that typically characterize the FDA. Lawmakers and other officials shouldn’t get in the way of such rare success.