Drugs

Improving Myelodysplastic Syndromes Trials

Cancer researchers from FDA and several academic medical centers say clinical trials for myelodysplastic syndromes (MDS) treatments should be designed from the outset to allow the practicable application of new therapies in the high-needs population, with drugs that can be administered and tolerated in community settings, and with endpoints that meaningfully improve patients’ lives over existing therapies. Writing in Clinical Cancer Research, the researchers say trials should also capitalize on an expanding understanding of the biology of these disorders to effect substantive change in patient outcomes.

The article shares the discussion at a 4/2022 panel of regulators and experts in MDS convened by FDA to improve MDS drug development. The panel reviewed challenges in MDS clinical trial design and endpoints and outlined considerations for future trial design to help drug development meaningfully meet patient needs.

Challenges identified for defining clinical benefit in MDS included cumbersome response criteria, standardized transfusion thresholds, and application and validation of patient-reported outcome instruments.

The panel said that clinical trials should reflect the biology of disease evolution, the advanced age of MDS patients, and how patients are treated in real-world settings to maximize the likelihood of identifying active drugs. In patients with lower-risk diseases, response criteria for anemic patients should be based on baseline transfusion dependency, improvement in symptoms, and quality of life.

For higher-risk MDS patients, the panelists said, trials should include guidance to prevent dose reductions or delays that could limit efficacy, specify minimal durations of treatment (in the absence of toxicity or progression), and have endpoints focused on overall survival and durable response.

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