Drugs

Broader FDA Interpretations Needed for Review Vouchers: Hill

A bipartisan Congressional effort is being made to broaden FDA interpretations when deciding on priority review vouchers to increase treatments for sickle cell disease (SCD). In a 10/25 letter to FDA commissioner Robert Califf, senator Tim Scott (R-SC) and rep. Danny Davis (D-IL) have raised concerns about how the agency interprets “active ingredient” when awarding priority review vouchers (PRVs), which could negatively impact drug development for rare diseases like sickle cell disease.

“Specifically, we understand that FDA has made an initial decision to narrowly interpret the definition of ‘active ingredient’ in the PRV statute, resulting in the unexpected denial of at least one pediatric PRV,” they wrote in the letter. “However, the decision whether to narrowly or broadly interpret the definition of ‘active ingredient’ for purposes of awarding a PRV is not so much a question of science as it is one of policy.

“Congress has not provided guidance,” the lawmakers continued, “as to how FDA should determine if two active ingredients in an ex-vivo gene therapy are the same or different, but the text of the statute and the legislative history make clear that Congress wanted to create an incentive to treat patient populations that might not warrant investment if left simply to ordinary market forces. By interpreting this statutory requirement in a narrower way than the statute requires — and one that flies directly against Congressional intent — we risk disrupting this delicate ecosystem, jeopardizing not only the availability of SCD treatments today, but also investments in complex and life-saving innovation for the future.”

The letter also says that the lawmakers have become aware that FDA may be in the process of reconsidering its interpretation. “This decision is critical for the future of rare disease drug development and rare disease patients, and we are grateful for the FDA’s close attention to this matter,” they said, urging the agency to keep their considerations in mind while it conducts its review.

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