Life Science Compliance Update

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July 22, 2015

FDA: "More Collaboration, Research Needed to Develop Cures"

Califf

The Food and Drug Administration's Deputy Commissioner for Medical Products and Tobacco, Robert Califf, recently offered his take on what is holding back medical progress. While a common critique is that the Agency's red tape is holding up innovation, Califf and an accompanying FDA report note that a "lack of understanding of the biology of the diseases" is to blame for the lack of treatment for Alzheimer's and many rare diseases.

"In these cases, the scientific community still lacks basic information about what causes these diseases and how they can be slowed and treated," states Califf. "When research does not offer answers to important scientific questions, cures cannot be developed. And when viable cures are not in the pipeline, focusing on regulation will not improve the situation, since FDA can only approve therapies with evidence for safety and effectiveness."

Califf writes:

Once key scientific questions are answered, we can use a variety of tools to reduce the length and cost of initial clinical trials for drug approval for these disease areas, and we can provide guidance to industry including advice on how to develop additional reliable biomarkers. For instance, we’ve improved the efficiency and predictability of clinical drug development by developing tools such as biomarkers and surrogate endpoints—markers of drug effect that do not directly represent an improvement in how a patient feels or functions, but are reasonably likely to predict a clinical benefit. Thus, for example, lowering a patient’s blood pressure can be used as a surrogate for the clinical benefit of preventing heart attack. Such tools have modernized clinical trial designs and may dramatically reduce the length and cost of drug development. They also can help target drugs to specific patients who can benefit most, thereby limiting the number and size of clinical trials.

An accompanying FDA report goes into detail about the state of drug discovery and development. for a number of diseases, starting with Alzheimer's (where "scientific discovery is in its infancy"), to diabetes (which is understood better than Alzheimer's, though scientific understanding is still lacking), to Hepatitis C (which now has a targeted therapy to prevent and treat the virus in specific patients). 

"While FDA has worked to transform the landscape for the final stage of drug development, progress in the discovery and testing stages of drug development has not kept pace," the report states. "As a result, too many diseases are still awaiting treatments and cures." View the report here.

Califf adds:

These are exciting times as we experience simultaneous revolutions in the biological and information sciences. We expect that the astounding increase in knowledge of biological systems enabled by whole genome sequencing, cloud computing, social media, and wearable devices to monitor physiology will create challenges to traditional thinking. And we are confident that this increased knowledge will continue to expand the pipeline of new therapies. This report emphasizes that we are prepared to deal with the product of this scientific investment by using regulatory paradigms that match the state of the science and by supporting dissemination of the latest knowledge applied to drug development.

The Agency will "continue to work to speed patient access to therapies shown to be safe and effective through our existing programs that allow for expedited review, development, and approval of certain medical products." In conclusion, he adds: "To encourage innovation, we also will continue to work with other government agencies and the healthcare community, including members of patient groups, academia, and industry. It will take a collaborative effort to improve our nation’s understanding of certain diseases and to translate any resulting scientific discoveries into cures."

 

 

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