New Legislation Would Improve FDA Drug-Approval Process

New Legislation Would Improve FDA Drug-Approval Process

The recent controversy over Medicare’s decision to only cover the cost of the Alzheimer’s drug Aduhelm for patients enrolled in a clinical trial approved by the Center for Medicare and Medicaid Services (CMS) or supported by the National Institutes of Health (NIH) started with a decision made by the Food and Drug Administration (FDA) last June.

In that decision, the FDA approved Aduhelm using the “accelerated approval pathway,” which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments.

As a result of that FDA decision, three members of its own medical advisory committee, which had overwhelmingly voted against approving the drug, resigned in protest. One of them said the decision was wrong “because of so many different factors, starting from the fact that there’s no good evidence that the drug works.”

The committee had found that the evidence did not convincingly show that Aduhelm could slow cognitive decline in people in the early stages of the disease — and that the drug could cause potentially serious side effects of brain swelling and brain bleeding.

The “accelerated approval pathway” the FDA used in that approval has come under scrutiny and last March the Chairman of the House Energy and Commerce Committee Frank Pallone, Jr. (D-N.J.) introduced legislation that seeks to ensure that drugs that enjoy a quick trip through the regulator offer “proven clinical benefit” to patients.

The FDA’s accelerated approval pathway lets the agency base approvals on certain factors other than a demonstrated clinical benefit, like extending patients’ lives. The route helps certain drugs for diseases with unmet needs, like some types of cancers and rare diseases, reach patients sooner. But companies with these drugs are required to run trials to confirm the effectiveness of the drugs after it has received the “fast-track” approval.

If those studies do not bear fruit, the FDA has procedures in place to remove the product from the market.

This past week it was revealed that a bi-partisan group of House lawmakers are moving a little closer toward cracking down on drug-makers that dishonestly use the FDA’s accelerated approval pathway.

The House Energy and Commerce Committee announced Wednesday that its sweeping user fee authorization bill will include a revised policy from Chairman Pallone that would make it easier for the Food and Drug Administration to rescind its approval for drugs cleared through the pathway when drug makers do not complete required follow-up studies.

It has been said that a disease-controlling or preventing drug does no good if you cannot afford it. It is also true that a drug that received “fast-track” approval does no good if it does not really work.

TSCL will continue to monitor this legislation as the House of Representatives further develop it.