FDA Check-Up: Drug Development and Manufacturing Challenges

I have over 27 years of experience in the biotechnology and pharmaceutical industry. My current company, OncoMed Pharmaceuticals, is working at the cutting edge of oncology research, focused on antibodies that target a specific set of cells within tumors, known as tumor initiating cells, that drive the growth of the tumor and can differentiate into various cell types within the tumor. Currently, we have five products in clinical development in over 13 completed or ongoing trials, with more than 280 patients receiving our investigational agents. We continue to pursue the discovery of additional disruptive and novel anti-tumor initiating product candidates.

The U.S. biotechnology industry is working on treatments and therapies that have the potential to deliver new solutions to our most pressing health care needs and is a key element of an innovation-driven economy. We have come a long way in turning incurable diseases to treatable diseases, increasing the ability of patients to maintain independent lives. With the number of people over 65 increasing – improving the quality of life and ability for patients to maintain independence is a national imperative.

Hundreds of companies like mine are working on these solutions with over 400 clinical trials currently underway focused on developing the next generation of medicines for over 200 diseases.

This is also an industry poised to be a major contributor to a 21st century innovation-driven economy in the United States. However, we continue to face intense competition from other countries, as well as increasing R&D costs, regulatory challenges, and a contracted funding environment.

This year we have seen positive signs that the biotechnology industry is recovering from the economic crisis. Thirty-nine biotechs have gone public this year, including my own, marking the most active IPO market in a decade. Additionally, in 2012 the FDA approved 39 new molecular entities, the most approvals we had seen in 16 years. While this is good news, the financial and regulatory environment continues to pose significant challenges to innovative drug and biologic developers.

For example – first-time financings for new companies are the lowest we have seen since 1995. And, while we have seen an increase in the number of approvals, we have also seen a steep increase research and development costs – much of which is associated with increased requirements and costs to run clinical trials.

FDASIA contained several provisions designed in cooperation with industry and FDA specifically to provide FDA with the resources and processes that encourage the utilization of modern tools and approaches, such as adaptive clinical trial designs, and to allow for more interactive scientific dialogue between FDA, the industry, and patients. Some of the most exciting provisions in FDASIA include: an expanded Accelerated Approval pathway designed to improve on the historical success this program has had with developing game-changing medicines to treat HIV/AIDS and cancer and expand its utilization in other disease areas; and a new Breakthrough Therapy designation designed to get the most promising medicines to patients more efficiently.

However, while FDASIA included an agreement by the industry to significantly increase user fee funding for the FDA to help support FDA’s drug review activities and these new programs – sequestration has diverted a portion of these industry fees to an “escrow” account that has no practical purpose for FDA, industry, or patients. This has severely hindered FDA’s ability to fully implement critical provisions of FDASIA that would improve our ability to more effectively develop and deliver innovative medicines to patients. This is the equivalent of paying our utility bills and then being told we cannot access power, lights, or heat.

BIO would like to thank Congress for proposing a two year delay in sequestration of user fees in the proposed budget agreement but urges Congress to rectify this irrational and counter-intuitive situation by passing the FDA Safety Over Sequestration Act of 2013, sponsored by Representatives Leonard Lance and Anna Eshoo with broad bipartisan support.

While many new measures have been embraced and encouraged by Dr. Hamburg, Dr. Woodcock, and their colleagues in management at the FDA – it is yet to be seen if the regulatory flexibility afforded by FDASIA is being fully embraced at and across the FDA reviewer level to advance the development of new therapies for unmet medical needs.

Releasing user fees from sequestration, successfully implementing FDASIA, and enabling our colleagues at the FDA could significantly improve the ability of our industry to more effectively develop new medicines and get them to the patients who need them. These actions could also help stimulate investment in early-stage companies that are working on the next generation of medical discoveries and breakthroughs.

BIO is committed to working with FDA and Congress to ensure these goals are achieved.

Paul Hastings is Chairman and CEO of OncoMed Pharmaceuticals and Chairman of the BIO Emerging Companies Section Governing Board.

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