Ongoing Support of Innovation
FDA’s record in FY 2011 shows that the agency is continuing to make innovative drugs available to American patients faster than other countries, while upholding its reputation as the “gold standard” for safety and effectiveness decisions. Despite this record of high-quality, efficient drug reviews, FDA recognizes that both FDA and the pharmaceutical industry face challenges in drug development.
FDA is committed to working with the US pharmaceutical industry as it continues to develop innovative treatments for serious diseases and to help ensure that patients continue to have access to safe and effective drugs at the earliest possible time. Among other initiatives, FDA is working to make sure that user fees are sufficient in PDUFA V to:
- Increase the percentage of drugs that get through the review process in the first cycle; and
- Increase communication between FDA and sponsors throughout the drug testing and review process.
Although the NDA/BLA approval phase of drug development (the phase in which FDA plays the biggest role) is reported to have the highest success rate of any phase of drug development, it is critical to our public health mission that we work with industry and other stakeholders to take steps to reduce uncertainty and increase success in the other phases of drug development.
To promote the development of innovative new therapies, FDA has made advances in regulatory science a top priority. FDA is pursuing a number of scientific goals whose purpose is to expedite drug development. For example, FDA is working to improve the science behind certain clinical trial designs. FDA issued a draft guidance document on “adaptive trial designs” that make use of early results of a trial to modify the design, making the study more likely to detect whether the drug works. FDA is now working on a guidance document on “enrichment designs,” studies that make use of patient characteristics to identify people for whom the drug is likely to be effective. These designs allow smaller studies to be successful and target the treatment to patients who will benefit most. FDA encouraged the involvement of the scientific community in the development of these guidances, discussing them at many public meetings and putting them out for public comment. These advances can make drug testing more efficient, encouraging the development of novel products, and speeding new therapies to patients.
FDA has also built a computational science center to give reviewers advanced tools to analyze clinical data--tools that have already been used to support approval of drugs that might not otherwise have been approved. And the agency has issued an innovative “drug development tools” guidance document that will facilitate use of new, more efficient methods of establishing drug safety and effectiveness. The goal of this guidance, which has been enthusiastically received in the scientific community, is to encourage and support the development of methods that can speed the availability of new products, help identify products that might be safer or more effective than existing therapies, and give physicians and scientists better information about how the products act on the human body. FDA intends to support the development of these tools with dedicated staff and resources.
FDA is also working to:
- Support the enhanced use of pharmacogenomics and qualified biomarkers, each of which has the potential to decrease drug development time;
- Improve its capacity to assess drug benefits as measured by “patient reported outcomes,”
- Continue to support new developments in personalized medicine;
- Develop a more systematic and expansive approach to benefit-risk determinations; and
- Allocate more resources to facilitating orphan drug development, by developing guidance on drug development and reaching out to the rare disease patient community.
FDA is also exploring a range of new partnerships with the National Institutes of Health and academic institutions to develop the science needed to maximize advances in biomedical research and bring the development and assessment of promising new therapies into the 21st century. With this effort, FDA is poised to support a wave of innovation to transform medicine and save lives.