Biologics

Biological Therapies Need Clinical Trials: Califf, Marks

FDA commissioner Robert Califf and CBER director Peter Marks say biological therapies should be subject to adequate randomized controlled trials (RCTs) before receiving FDA approval. Writing in Science Translational Medicine, Califf and Marks say the “reasons for requiring adequate, reliable evidence demonstrating that the benefits of a proposed intervention outweigh its risks are well established and are embedded in the foundational principles of FDA. Appropriately designed, well-controlled clinical trials are needed because the vast majority of proposed interventions fail to provide a benefit that exceeds risk, despite the good intentions and solid preclinical science required by funders, ethics committees, and regulators when human trials are initiated. In particular, when study outcomes are subjective, case studies with no control group and no blinding often lead to biased observations.”

Califf and Marks write that while they remain highly enthusiastic about the potential for biological therapies to address human disease, failing to define in adequate and well-controlled clinical trials which therapies are effective in which patients, and instead continuing to offer investigational therapies to patients at the expense of conducting adequate and well-controlled clinical trials, leads to the potential for profiteering from the hopes of patients and families.

“We hope that the clinical community … will band together to implement appropriately designed randomized clinical trials with adequate sample sizes in a timely manner to provide definitive evidence of safety and effectiveness, one way or the other,” Califf and Marks conclude. “FDA is open to considering new study designs and operational strategies that apply the principles of ‘quality by design’ to ensure that valid inferences can be drawn about treatment effects in a manner that optimizes efficiency in busy clinical environments…. Conducting appropriately designed clinical trials, ultimately combined with FDA approval, is the way to protect the rights and safety of participants in these trials. Conducting RCTs also ensures that more people are treated with beneficial therapies, enables coverage by payers, facilitates their inclusion in clinical practice guidelines, and avoids wasting money and giving people false hope based on ineffective therapies.”

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’

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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

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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