By James E. Valentine – On January 13, 2015, CDER’s Clinical Outcome Assessments Staff (formerly Study Endpoints and Labeling Development (SEALD)) announced the launch of Stage 1, or the pilot stage, of its Clinical Outcomes Assessment (COA) Compendium. A COA measures patients’ symptoms, overall mental state, or …
By Riëtte van Laack – On December 23, 2015, FDA issued a proposed rule amending the regulations for prescription and over-the-counter (OTC) fixed-combination products, co-packaged drugs, and combinations of active ingredients under consideration for inclusion in an OTC monograph. The 20-page proposal is intended to harmonize …
By James E. Valentine & David B. Clissold – In an annual update presented at the FDA/CMS Summit on December 14, 2015, Dr. John Jenkins, Director, Office of New Drugs, the Center for Drug Evaluation and Research (CDER or the Center), provided metrics and insights into …
By Alexander J. Varond – On December 8, 2015, FDA approved two new therapies: Vonvendi (BLA 125577) for von Willebrand disease and Kanuma (NADA 141-453) for lysosomal acid lipase (LAL) deficiency. These approvals bring the number of new molecular entities and new therapeutic biological products approved this …
By Jay W. Cormier – On December 8, 2015, FDA issued a complex set of approvals for a unique product, Kanuma (sebelipase alfa). Kanuma is indicated for the treatment of a rare disease known as lysosomal acid lipase (LAL) deficiency. The list of distinguishing descriptors for the …
By Josephine M. Torrente & Etan J. Yeshua – In September, FDA proposed a significant limitation on the way drug sponsors can appeal FDA decisions, and yesterday Hyman, Phelps & McNamara, P.C. (HP&M) formally requested that the Agency reverse course. In a draft guidance document issued …
By Kurt R. Karst – Last week we posted on one of two bills recently introduced in Congress that continues a trend to push for (or reward) new product development by offering an incentive different from the standard grants of patent and non-patent marketing exclusivities (S. 2055, the …
By James E. Valentine* – In recent years there has been an increasing focus on patient engagement, centering on new opportunities for FDA to incorporate the patient perspective into its regulatory decision making. These discussions have resulted in Congress and FDA establishing new policies and programs …
By James E. Valentine* & David C. Clissold – On September 8, the U.S. Department of Health and Human Services (HHS) and fifteen other Federal departments and agencies announced a Notice of Proposed Rulemaking (NPRM) to revise the Federal Policy for the Protection of Human Subjects, …
By Alexander J. Varond – Hardly a week goes by without news of FDA’s priority review voucher (“PRV”) programs. The last several weeks are no exception. In this blog post, we focus on five key updates related to FDA’s tropical disease and rare pediatric disease (“Pediatric”) …
By James E. Valentine* – On August 17, 2015, FDA announced the availability of an update to its 11 year old guidance on botanical drug development. Botanicals are plant materials, algae, macroscopic fungi, and combinations of those things that can be regulated as a food (including a …
By Kurt R. Karst – Earlier this month, legislation was introduced in both the U.S. Senate and U.S. House of Representatives to fund FDA for the next fiscal year: Fiscal Year 2016. Both the Senate bill and the House bill, titled “Agriculture, Rural Development, Food and …
By Alexander J. Varond – In a recent FDA Voice blog post, titled “More Collaboration, Research Needed to Develop Cures,” FDA discusses the speed of drug discovery and development. The blog post also references a white paper released the same day, titled “Targeted Drug Development: Why …
By Alexander J. Varond – On July 10, 2015, the U.S. House of Representatives passed the 21st Century Cures Act. In all, there were 344 “yes” votes and only 77 “no” votes, with half of the “yes” votes coming from Republicans and the other half coming …
By James C. Shehan – On July 9, 2015, Representatives Matt Salmon (R- AZ), Paul Gosar (R-AZ) and Marlin Stutzman (R-IN) introduced H.R. 3012, the Right to Try Act of 2015. The bill seeks to expand the access of terminally ill patients to experimental drugs in …
