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.

Overhaul to help FDA become more specialized

Alexander Gaffney of raps.org provides a very interesting article on FDA’s move to have a more specialized program to deal with scientific advances and regulatory complexity. Gaffney introduces the article by discussing the cryptic message from FDA saying there was a need for the agency to realign the programs. When FDA announced the Program Alignment Group (PAG) they sent out a memo outlining some of the areas the PAG will focus on.

  • specialization across FDA’s inspection and compliance functions based on specialization within FDA’s regulated industries and the demands of new legislation
  • training that is developed collaboratively by ORA and the Centers and leads to the development of competency and training requirements to enhance and maintain FDA’s workforce 

These are just a few of the focus areas provided in the article. Gaffney goes on to talk about how the PAG will impact FDA centers all over the country whether it be in the specialization of some employees or trying to “de-layer” to allow FDA to take timely and appropriate action. To determine whether this new program helps or hurts FDA, we will have to wait and see. To read the full article click here.

Diabetes Testing Devices-FDA Releases New Guidance Documents


Alexander Gaffney of raps.org details the Food and Drug Administration’s (FDA) push for new development approaches for diabetes testing systems.  FDA has typically not distinguished between prescription and non-prescription diabetes testing devices.  In review of the guidance documents, it is clear that FDA wishes to address concerns regarding the potential sharing of devices, as well as cleanliness. To view Gaffney’s article, click here.  To help address these concerns, FDA released two new separate draft guidances on January 6, 2014.  To see both guidance documents, see the links below:




All comments are due April 6, 2014.  Need help with your diabetes testing device or diagnostic, contact us at contact@pearlpathways.com.

Could new work group streamline FDA’s efforts?

Alexander Gaffney of raps.org details that the US Food and Drug Administration (FDA) is ready to launch a new work group, the Program Alignment Group (PAG).  The goal in creating PAG is to streamline efforts from various organizations and centers of FDA to avoid duplication, reduce complexity, and increase globalization efforts. There are six core initial areas that the PAG will begin to focus on that deal with specialization, training, new work planning, compliance policies, lab optimization, and center/ ORA practices. FDA commissioner, Margaret Hamburg says it will take time and resources for the PAG to become a fully functioning group, but it has her full support. To read the full article, click here.