Drugs

Suggestions to Improve Rare Disease Guidance

Three stakeholders say FDA could improve its draft guidance on a collaborative approach for drug development to be used in pediatric rare diseases that uses Gaucher disease as a model. The National Organization for Rare Diseases (NORD) says that much of the draft guidance is very specific to Gaucher disease and may be difficult to easily extrapolate to other diseases. “While FDA outlines the structure of this collaborative model within Gaucher disease,” it says, “it does not specify which characteristics of this model could also apply to other disease areas. Given the regulatory caution and restraint of many biotechnology and pharmaceutical companies, we can imagine many companies choosing not to pursue this model as they would be unsure of what translates to their specific disease or development pathway.”

NORD also says that FDA does not discuss the many hurdles to collaborative drug development that would prevent the model from gaining popularity, especially obstacles involving intellectual property, patent, and proprietary barriers. Finally, it says the agency does not address how to incorporate patient organizations, which it says are often the unifying participant in drug development.

The Biotechnology Innovation Organization (BIO) raises three main issues: (1) the guidance lacks detail on applicability across a broad range of rare diseases; (2) the draft outlines use of multi-arm, multi-company clinical studies but does not provide procedural information on how to address challenges of this approach; and (3) the guidance fails to address other possible methodologies that would help reduce the total number of children required for enrollment in clinical trials. BIO expands on these three points and supplies additional comments and line edits.

Pharmaceutical Research and Manufacturers of America (PhRMA) also raises the concern about applicability of the guidance for pediatric rare disease drug development programs other than for Gaucher disease. It recommends revising the document to provide a more general guidance and offer Gaucher disease-specific recommendations in an appendix. PhRMA also calls for further guidance on how analytical methodologies could enhance a collaborative approach to pediatric drug development in other rare diseases.

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

Read more

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