Laws and proposals can go a long way toward fostering biomedical innovation in the U.S. The decisions that Congress makes now will play a key role in whether or not we maintain our global leadership position in this area and maximize the economic and public health solutions that the biopharmaceutical industry has to offer.
The U.S. biotechnology industry is poised to be a major driver in an innovation-driven economy and we offer real solutions to our most pressing health care needs: curing disease, reducing costs, increasing quality, and ensuring that people enjoy not only longer lives, but better and more productive lives. Our biotech companies provide high-wage jobs at both public research institutions and in the biotech companies that typically locate near centers of academic research.
In order to fully realize these potential benefits we must have a policy environment that fosters innovation and scientific breakthroughs. There are many examples of federal policies that support our industry, and encourage the advancement of innovation.
First, Congress should be applauded for the 2011 passage and enactment of the Leahy-Smith America Invents Act, or the “patent reform bill.” Small biotechnology companies rely heavily on their patents to attract investment to fund the lengthy and expensive research and development process necessary to bring breakthrough medical therapies and other products to patients and consumers. Strong intellectual property protection is critical for these companies, and they will benefit from the improvements to our nation’s patent system made by this law. However, there continue to be attacks on intellectual property in Congress and in the Courts that could be devastating to the biotechnology industry, where intellectual property is often the only asset a company has while they spend many years researching and developing breakthrough medicines.
In March of 2010, Congress enacted the Therapeutic Discovery Project (TDP), a critical tax program designed to stimulate investment in biotechnology research and development. Under this program, small biotech companies received a much-needed infusion of capital to advance their innovative therapeutic projects while creating and sustaining high-paying, high-quality American jobs. The Therapeutic Discovery Project was a significant step in the right direction by Congress to invest in growing the U.S. biotech industry and keep pace with our global competitors. Given the imbalance between the extraordinarily high demand by small biotech companies and the limited pool of funds, we hope that Congress will extend and expand this highly popular program and assist more American companies in pursuing life-saving scientific breakthroughs and supporting American jobs.
Additionally, the Congress should be commended for reauthorizing the Small Business Innovation Research (SBIR) program last year, as this too was a monumental step forward for the development of potential breakthrough products for patients. This reauthorization reinstated eligibility for a vast majority of small biotechnology companies that had been shut out of the program for the past decade, due to a regulatory ruling that made small companies who have multiple venture capital investors ineligible. SBIR provides a critical source of funding for emerging biotechnology companies in the early development stages of medical research; the changes included in the reauthorization will enable a larger number of small companies to compete for funding, thus ensuring that the program will be able to fund small biotech companies’ projects that have the greatest potential to bring innovative medical treatments to the patients who need them.
In 1992, Congress, industry, and the FDA worked together to create the Prescription Drug User Fee Act (PDUFA). This program ensures that FDA has the ability to hire additional reviewers to expedite the drug review process by having industry pay “user fees.” PDUFA has been a tremendous success and this year, the program is set for its fifth reauthorization, which will work to get the FDA back to the basics of approving lifesaving therapies and cures. PDUFA V will enhance the drug development and review process by increasing transparency and scientific dialogue, advancing regulatory science, and strengthening post-market surveillance. Most importantly, from the standpoint of innovative companies, our hope is that PDUFA V will provide patients and doctors with earlier access to breakthrough therapies.
The Accelerated Approval pathway at the FDA has been a great success story, in part. While its use has been largely limited to certain disease areas, the pathway has benefited patients tremendously because it has sped access to meaningful therapies, and because it has stimulated an explosion of investment in innovation. The Accelerated Approval pathway must be modernized and expanded to incorporate the remarkable advances in life sciences that have been and will continue to be made, in such areas as genomics, molecular biology, and bioinformatics. Clarification of when and how these tools can be utilized in an Accelerated Approval pathway will not only increase patient access to breakthrough therapies, but it will also incentivize drug development for serious and life-threatening diseases, but will encourage the development and utilization of still more tools and methodologies. More, a properly constructed accelerated approval pathway will help the FDA remain the global gold standard for the review and approval of safe and effective medicines while speeding the delivery of critical, breakthrough medicines to the patients who so desperately need them.