Drugs

Boost Faith in Accelerated Approvals: Researchers

Yale University researchers say that as FDA broadens its use of accelerated approvals, it should be taking steps to increase public trust in that approval pathway. Writing in the New England Journal of Medicine, the researchers say the agency’s empowered stance on using accelerated approvals resonates with those who think it should prioritize timely access to innovative medical products, but raises serious concerns for clinicians, patients, and caregivers.

Previously, they write, FDA approval signaled that the drugs were safe and effective for use and that their benefits outweighed their risks. “But with more aggressive adoption of the accelerated approval pathway, particularly in areas other than oncology,” they write, “clinicians, patients, and caregivers are being asked to make individual decisions about whether ‘reasonably likely’ to predict benefit outweighs personal risk, despite substantial residual uncertainty. Their decisions are complicated by the considerable financial costs of these new medicines, the effort needed to navigate health insurance plans, the time and money spent traveling to receive treatment, and the products’ potential harms.”

The article notes that the recently passed Food and Drug Omnibus Reform Act included FDA-supported modifications to the accelerated approval program that give the agency the power to request that companies start confirmatory triaks before they receive accelerated approval. It also streamlines the process for withdrawing drugs when postmarket studies are negative or delayed, and requires companies to provide confirmatory trial status reports to FDA every six months.

In addition to those changes, the researchers suggest that to better support clinicians, patients, and caregivers, the agency consider:

determining whether the pathway is appropriate for common diseases associated with substantial morbidity and mortality, even if other program conditions are met;
establishing opportunities for public input whenever new surrogate markers are being considered “reasonably likely” to predict clinical benefit and establishing standardized reevaluation periods, such as every five years, for all surrogate markers to ensure that scientific evidence continues to support their use;
asking external advisory committees to weigh in on all potential accelerated approvals to foster transparency and build public trust;
preparing informational manuals to support patients and clinicians that leverage validated decision-support tools to communicate anticipated benefit and potential harm; and
shortening the period allowed for fulfilling postmarketing requirements.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome