Regulatory Rundown: Week of May 21-25
Brittany Blocker, M.S., MBA
Regulatory Policy & Intelligence, Sr. Manager at Vertex Pharmaceuticals
Senate Committee Advances Bill Boosting FDA’s FY2019 Budget by $159M (RAPS, 5/24)
On Thursday, the Senate Appropriations Committee signed off on a $159 million increase in discretionary funding for the Food and Drug Administration (FDA). The increase is $150 million less than the increase proposed in the House. The additional funds will be used to support domestic manufacturing, expand the FDA’s engagement with outsourcing facilities, and medical device manufacturing.
Gottlieb: FDA Prepping for Push of New Gene and Cell Therapies (RAPS, 5/22)
The FDA is planning to release new draft guidance documents on gene therapy-related issues and a guidance on the clinical development of gene and cell therapies. The first area the agency will focus on in terms of offering potential accelerated approval endpoints for certain gene therapy products is hemophilia. The guidance will explain nuances and general differences between traditional drug or biologic development and cell and gene therapy development.
‘Right to try’ legislation heads to the White House (Washington Post, 5/22)
On Tuesday, the House passed the “right to try” legislation with a vote of 250 to 169. The bill would allow people with life-threatening illnesses to bypass the FDA to obtain experimental medications. Under the new legislation, the FDA would not oversee the right-to-try process. Drug manufacturers would be required to report adverse events only once a year. Additionally, the agency would be restricted in how it uses adverse event information when considering the experimental treatments for approval. President Trump is expected to quickly sign the measure.
FDA looking at ways to include price info in drug ads, Gottlieb says, as questions loom (STAT, 5/18)
The Food and Drug Administration (FDA)’s proposal to have pharmaceutical companies include drug prices in advertisements has prompted a series of questions. FDA Commissioner Scott Gottlieb shared that the agency has formed a working group to answer questions like how consumers would benefit and whether the government can legally require disclosures. The inclusion of drug prices in direct-to-consumers ads was one of two actions that Health and Human Services (HHS) Secretary Alex Azar focused on during his remarks on the administration’s drug pricing plan.