Pharma, BioPharma

FDA: More basic science research needed to accelerate cures

Robert Califf, a deputy commissioner at the FDA, says that in order to accelerate medical treatment options for diseases like Alzheimer’s, we must improve our basic understanding of disease processes.

Drug discovery is moving slowly for many diseases because of a “lack of understanding of the biology of disease,” a top Food and Drug Administration official recently wrote.

Robert Califf, a deputy commissioner at the Food and Drug Administration, calls out the “paucity of reliable biomarkers in some diseases”  in a recent blog post – citing it as a sign that a lot of disease research isn’t progressing as it should.

“It will take a collaborative effort to improve our nation’s understanding of certain diseases and to translate any resulting scientific discoveries into cures,” Califf wrote.

The FDA just put out a new report that compares disease where there is a robust pipeline of drug therapies in development – and the ones where much is left to be desired. The regulatory process has been improved in recent years, and in terms of drug approval the FDA calls itself the fastest system in the world:

Yet, despite these successes at speeding up drug approval, as well as recent leaps forward in our understanding of human biology, too many diseases are still awaiting treatments and cures. Serious public health needs, such as treatments for Alzheimer’s disease, are not being met. And rising research and development (R&D) expenditures are not being matched by a proportionate discovery of innovative medicines.

Alzheimer’s disease, for instance, is still a poorly understood disease – which is hindering significant progress in drug development and approval, the FDA says. We still don’t have any consistent understanding of Alzheimer’s biomarkers, which in turn is preventing us from creating any meaningful clinical improvement.

The FDA report also cites diabetes, HIV/AIDS and Hepatitis C as case study diseases – in what’s been effective, and what has not. Hep C, for instance, is a shining example of the way deep scientific understanding of disease processes can lead to cures.

A broader understanding of Hep C only came, after all, when HIV/AIDS research in the 1990s helped unveil the molecular mechanisms of other viruses. From there, drug development was able to accelerate, leading to the wildly successful Sovaldi.

“When research does not offer answers to important scientific questions, cures cannot be developed,” Califf writes.


Califf, in his blog post, says that once we get a deeper basic science understanding of diseases like Alzheimer’s – using tools like biomarkers and surrogate endpoints – we can reduce the length and cost of clinical trials. He writes:

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.