On the last day of the World Orphan Drug Congress, John Crowley, Chairman and CEO, Amicus Therapeutics provided an inspiring overview of how the Fifth Prescription Drug User Fee Act (PDUFA V) and new government policies may affect the orphan drug industry. From a historical perspective, Mr. Crowley noted that the introduction of the Orphan Drug Act thirty years ago was significant, but it was not until the late 1980s and early 1990s that the Orphan Drug Act really began to have an impact.

That impact was largely based on the work of the HIV advocacy groups spearheading the idea that biomarkers could be used as outcome measures and that coincided with improvements in biotechnology. Following those developments, the number of orphan drugs studied significantly increased.

On the other hand, Mr. Crowley said there were many factors that impeded orphan drug development. The financial crises in 2004 and in 2008 led investors to be very hesitate to invest in the biotech industry. That hesitancy was largely due, in Mr. Crowley’s opinion, to the different mindsets that inhabit investors compared to people in the biotech industry. Investors want results quickly. That is how the stock market works.

In contrast, biotech personnel are used to learning from their failures as they move towards their long term goals. That is how science works. Another factor that has impeded orphan drug development was the FDA’s focus on safety issues following the discovery of the Vioxx safety problems in the early 2000s. The FDA increased concern with safety data made it difficult for analysts (and investors and scientists) to predict what drugs the FDA would approve. That uncertainty in what the FDA would approve led to further hesitancy by investors.

In 2010, many people understood that this mindset needed to change and that all of these financial and regulatory factors that were impeding the clinical scientific breakthroughs were going to destroy the biotech industry involved in orphan drug development. Mr. Crowley recalled that during board meetings involved in PDUFA, he stated that “the ship is going down and if we don’t go for substantial changes, we will all sink.” Others agreed with this statement and as a result, patient advocates, industry executives, venture capitalist etc joined forces to stress the need for PFUFA to undergo significant changes. Changes that would make it easier for investors, patients, and business executives to know the FDA is more open and supportive of the needs of the orphan drug industry.

Fortunately those patient, investor, and scientific voices were heard during congressional hearings on PDUFA and in July 2012, the PDUFA V, that was part of The Food and Drug Administration Safety and Innovation Act (FDASIA), was signed into law. PDUFA V with its inclusion of the Ultrafast Act, Breakthrough Therapies Act, Patient Participation in Medical Product Discussions provision, etc. made it more feasible for biotech companies (and big pharmaceutical companies) to invest in orphan drug development.

Read more at Rare Disease Report.

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