Senior leaders from the FDA, BIO, and the industry convened at the 14th Annual BIO CEO & Investor Conference to discuss the legislative and regulatory environment, and what is in store for the FDA in the coming months and years.
With PDUFA V reauthorization looming and the implementation of biosimilars legislation continuing, plus the recent introduction of the TREAT Act, 2012 will be a big year for the FDA.
The central theme throughout the closing plenary session was the idea that we are all in this together –FDA, NIH, industry, academia, patients, investors.
“This is a team sport that requires collaboration,” said Stephen P. Spielberg, MD, PhD, Deputy Commissioner for Medical Products & Tobacco at the FDA . “We all want the same thing, and we know that we need to partner with industry and stakeholders to move beyond these hurdles.”
Dr. Spielberg advocated for novel clinical trial designs that would save time, effort and, most importantly, show efficacy. “We are going through a sea change, and it takes time to implement new paradigms.”
Jonathan S. Leff, Managing Director of Healthcare at Warburg Pincus LLC  cautioned against finger pointing as it represents a ‘false dichotomy’ since nobody is solely at fault but it is everybody’s problem. “The cost and time it takes to get drugs to the market is ever escalating, along with the need for safety and certainty.”
Richard Pops, Chairman & CEO of Alkermes plc , expressed the challenges facing biotech companies by poignantly stating, “Getting a drug approved is an unnatural act.” Pops describes his company as a post-emerging company.
As an emerging company CEO, Paul Hastings of OncoMed Pharmaceuticals, Inc.  asserted that companies like his are affected by delays in the regulatory process the most.
“A regulatory delay for us could easily cost us up to three million dollars, and extend our timeline by 30 days or more,” said Hastings. “We need a more informal approach, with more open communication to avoid costly and timely delays that could easily be handled by a simple phone call.”
Unpredictability in the review process, suboptimal communication with sponsors, and decreased FDA performance not only hinders patient access to new treatments, but also negatively impacts the ability of biotechnology companies to raise funding to support clinical development and ongoing innovation.
In the PDUFA V agreement, industry and FDA agreed upon a set of enhancements that seek to restore FDA’s review performance and get back-to-basics for patients by strengthening scientific dialogue and transparency between FDA and the sponsor during the review of a novel drug or biologic, with the goal of minimizing review issues that can delay patient access to needed treatments.
The session moderator – Jim Greenwood, President and CEO of BIO – asserted that central to all efforts has been the question of FDA’s mission to advance public health.
“Patients who died waiting for a drug are no different than those who would die from a drug,” said Greenwood.
The BIO CEO & Investor Conference wrapped up on Tuesday, February 14 in New York City at the Waldorf=Astoria.