By James E. Valentine & Alexander J. Varond – FDA recently issued draft guidance, entitled “Special Protocol Assessment,” which, when finalized, will replace FDA’s 2002 final guidance by the same title. At 23-pages long, the draft guidance is much more thorough than its predecessor (only 11 …
By Jay W. Cormier – The National Institutes of Health (NIH) recently announced that it would be suspending drug manufacturing at two of its facilities after preliminary findings by two outside consulting groups found that certain sterile products were not manufactured in compliance with GMP quality …
By Kurt R. Karst – Ever since FDA Commissioner Dr. Robert M. Califf's February 2016 call for a sweeping review of FDA policies concerning opioids (in response to growing pressure from Congress), the Agency has been on a tear to meet that call with new policies and requirements. There's the recent announcement …
By Alexander J. Varond – FDA’s breakthrough therapy designation (BTD) program has been, by all accounts, a popular program. Sponsors, looking to gain extra support from the Agency, shorten review times, and signal to investors that FDA views their preliminary clinical evidence favorably, have submitted over …
By James E. Valentine – Last month, the U.S. Government Accountability Office (GAO), the independent, nonpartisan agency that provides auditing, evaluation, and investigative services to Congress, publicly released the findings of a report on FDA’s handling of drug postmarket safety oversight. As reflected in the title of the …
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”) …
