FDA completes rewrites of device pre-submission guidance

Alexander Gaffney from raps.org released an interesting article on FDA’s completion of the rewrite of device pre-submission guidance. FDA issued a guidance document to outline the changes and why these meetings are so important. They believe that the meetings offer a way to identify, clarify, and solve any obvious potential issues before the process starts. Also contained in the guidance are appendices that are associated with specific types of applications. To read the full guidance click here. To read the full article click here.

FDA productivity gap an issue?

Alexander Gaffney from raps.org wrote an intriguing article on productivity gaps that are haunting FDA and the drugs that are in great need of approval. The Manhattan Institute for Policy Research (MIPR) created a report to objectively examine FDA’s speed on reviewing products. It was found that a wide variety of approval times and review cycles existed across FDA divisions.  Check out the below chart from Gaffney’s blog for a sample of the data. To read the full article click here.

Chart for blog

New proposal from FDA on fast tracking medical devices

Varun Saxena from fiercemedicaldevices.com wrote an interesting article on the proposed plan of Expedited Access PMA (EAP). The FDA talked about how EAP is not a new path to market but a collaborative approach to facilitate product development under existing FDA guidelines. It is targeted for a very specific type of high-risk medical devices and allows the company to interact early and on a regular basis with FDA.  Quoted in the article was CDRH Director Jeffrey Shuren, “We are excited to offer a proposed program for expedited access for certain high-risk medical devices.” To read the full article click here.

FDA held hearing on Over-The Counter drugs, taking comments until May 12th

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.

Orphan drug approvals surge

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.

World drug industry regulators to form a coalition

Fdanews.com just published an article on the possibility of FDA and other global regulatory bodies forming a coalition called the International Coalition of Medical Regulatory Authorities (ICMRA). The goal of the ICMRA is not to develop identical standards, but to provide the world with “high-level, strategic advocacy” on quality. This idea comes from FDA, EMA, and Health Canada sharing data on quality inspections of industry facilities. ICMRA has been an idea for years, but there are still some major issues to overcome before they start the coalition. To read the full article click here.

FDA to increase foreign facility inpections

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.

Push for FDA to allow children with severe illness access to unproven drugs

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.

New FDA guidance on promotions

Alexander Gaffney from raps.org has published an article on the newly released FDA guidance Distributing Scientific and Medical Publications on Unapproved New Uses — Recommended Practices addressing how companies can use scientific literature to promote its products. Practices using certain literature can blur the line between marketing the products and protected commercial free speech.  The guidance is a revision of the 2009 guidance that was criticized for restricting life science companies’ ability to promote public health. In the new guidance FDA will be looking at science not promotion as the emphasis. To review the guidance on FDA’s website, click here, and to read Gaffney’s full article click here.

FDA’s Center for Devices and Radiological Health look to increase effectiveness in 2014

Arzezu Sarvestani from massdevice.com wrote an intriguing article on FDA’s Center for Devices and Radiological Health’s (CDRH) outlined priorities for the upcoming 2014 year. One of their main priorities will be in streamlining its clinical trial program with hopes of becoming more efficient, consistent, and predictable. To accomplish this they will have fewer applications that require multiple cycles before approval. Going along with this view the CDRH will consider moving some of the pre-data requirements to post market. FDA will soon start looking for public remarks on the framework on which pre-data requirements can be moved to post market. To read the full article click here.