Raqiyyah Pippins and Katie Riley from fooddruglaw.com wrote an informative article on FDA’s hearing on Over-The-Counter (OTC) drugs. The hearing comes at a period where there is a lot of frustration with the monograph process and FDA’s position on novel modes of delivery for certain ingredients FDA is requesting that people communicate the strengths and weaknesses of the existing OTC review process and is accepting comments until May 12th and will release a transcript of the hearing on April 25th. To read the full article click here.
Check out Louise Zornoza’s recent coverage of a new guidance for Health Canada on raps.org . The new guidance released on April 4, 2014 covers new design and format requirements on monographs. The changes are intended to emphasize clinical relevance and make information easier to access. It will also provide format and information consistency from the various kinds of drugs and drug classes. To read the full article click here.
We are very excited to announce that Gretchen Bowker will be presenting at St. Vincent Research Brown Bag Lunch Series. The series is a bi-monthly event to provide education and networking opportunities to our research professionals. Gretchen Bowker will be speaking on “Practical Tips on Hosting an FDA Audit. The presentation will be on 4/10/2014 from 12:00-1:00 at the Indiana Neuro-Science Conference Room on the St. Vincent Campus.
This is a private invite only event.
On January 4th, 1983 President Ronald Reagan signed into effect the Orphan Drug Act. For over 30 years, orphan drug development has grown in volume, and has had a major impact on the biopharma and diagnostics industries. With advancement in technology including increased development of novel companion diagnostics as well as the mapping of the human genome, orphan drug approvals by FDA have continued to rise. New data has been released showing that 2013 was a big year for orphan drug approvals. In 2011, FDA approved 26 orphan drugs. The number dropped slightly in 2012 g to 25, but rebounded in 2013 to a record 31. Orphan drug designations were also high in 2013 reaching a record breaking 260.
It is estimated by PHRMA in a 2013 Medicines in Development report entitled “Rare Diseases: A Report on Orphan Drugs in the Pipeline,” that 30 million Americans have one of the 7000 diseases deemed rare. Rare disease is defined as one affecting under 200,000 people in the United States. Further, this report estimated that there were 452 medicines and vaccines in development for rare disease in 2013. To review this report, click here. To review a recent article in medcitynews.com covering the surge in orphan drug development and approvals, click here.
Pearl Pathways has the pleasure of working with several of our clients on developing the appropriate regulatory strategy, executing global regulatory filings, and assisting with clinical trials for orphan drugs. Contact us if you need assistance with your orphan drug or device.
We anticipate that this focus on orphan drug development will continue among both large pharm and small biotechs. Navigating these diseases in small patient populations is complex and can be daunting, but new technologies and the regulatory landscape have made it a bit easier to impact rare and debilitating diseases.
Zachary Brennan from in-pharmatechnologist.com covered FDA’s plan to increase foreign facility inspections. FDA has made inspecting high-risk firms a top priority not just in the United States but also overseas. This is not new for FDA as they planned on visiting 750 high-risk firms in 2013, but they were actually able to visit 808. Out of those 808 visited, 43 warning letters were issued. To read the full article click here.
Zachary Brennan from in-pharmatechnologist.com wrote an interesting article on the European Medicines Agency’s (EMA) move for pharmaceutical companies to produce a risk-management plan (RMP) for the public. The document will contain the known and unknown issues and safety concerns that go along with the medicine and what measures should be taken to prevent injury or illness. The EMA’s plan is to first focus on the creation of RMPs for new products in 2014, with the potential to look at products created in previous years. The RMP is just another addition to the agency’s summaries called the European public assessment report. To read the full article click here.
John Carroll from fiercebiotech.com just published an intriguing article on recent efforts by parents of children with severe illnesses including Duchene Muscular Dystrophy (DMD) in the wake of a recent FDA denial for compassionate use. Public relations efforts including flash mob demonstrations and petitions to FDA for pilot studies have been used. As John Carroll shares, “There are no easy answers here. Just hard questions.” To read the complete article click here.
PR Newswire on thestreet.com just produced an interesting article on the Michael J. Fox Foundations’ (MJFF) efforts to change the way people are selected for clinical research. The Michael J. Fox foundation is a non-profit organization focused on finding a cure for Parkinson’s disease. The foundation is heavily invested in certain positions in the Parkinson’s research environment and using their influence to publish a paper on how to help improve recruitment for clinical testing. Awareness building efforts, patient education and providing actionable steps can help improve participation in testing. To read the full article click here.