The Latest in Regulatory and Reimbursement

Biocom submitted comments urging CMS to rescind its proposed Most-Favored Nation pricing models. The CDC announced a leadership change. The FDA introduced its new plausible mechanism framework via draft guidance, the Quality Management System Regulation for medical devices took effect last month, and the agency announced a major change in clinical trial standards. HRSA published a request for information regarding the 340B Rebate Model Pilot Program.

Biocom Opposes MFN Price Control Models

Biocom submitted comments urging CMS to rescind its proposed most-favored nation (MFN) pricing models, GLOBE and GUARD, which target certain drugs covered under Medicare Parts B and D, respectively.

NIH Director Serving as New Acting CDC Director

NIH Director, Dr. Jay Bhattacharya, will temporarily serve as the acting CDC director while continuing to lead the NIH. Former CDC Acting Director, Jim O’Neill, will be nominated to lead the National Science Foundation.

FDA Announces New Plausible Mechanism Framework

The FDA announced its new plausible mechanism framework to support the development of individualized therapies for ultra-rare diseases and published a draft guidance outlining this framework for genome editing and RNA-based therapies. Biocom is soliciting comments on the guidance in the following section. 

FDA Pivoting Towards Single Trial Standard

In a recent NEJM article, FDA Commissioner Makary and CBER Director Prasad explain that the FDA will require, by default, one adequate and well-controlled clinical study, plus confirmatory evidence, to demonstrate a product’s safety and effectiveness. Historically, the FDA has required two pivotal trials as the standard.  

Quality Management System Regulation Takes Effect

On February 2, the Quality Management System Regulation rule took effect. The FDA will no longer conduct inspections of device facilities under the previous Quality System Inspection Technique and the FDA published a new Compliance Program Manual.

Request for Information: 340B Rebate Model Pilot Program

Last month, the Health Resource and Services Administration (HRSA) published a request for information seeking input on whether to adopt a rebate model in the 340B program and, if implemented, how the operation of the rebate framework. Comments are due April 20. 

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Featured Members

Congratulations to Ascendis Pharma on receiving FDA approval for YUVIWEL, a once-weekly treatment for children with achondroplasia. Achondroplasia is a rare genetic condition causing skeletal dysplasia and affected individuals may have an increased risk of muscular, neurological and cardiorespiratory complications.