But frankly it reads like a publication edited, rewritten and vetted by the FDA's political minders and not the work of scientists. The glossy document spends half it's pages extolling the virtues of activities that have nothing to do with accelerating the development of new products through cheap and easy to use tools and metrics. Rather, we get a roll call of Obama administration initiatives such as Startup! America, the FDA Small Business Liaison Program, Young Entrepreneurs, Partnering with the Small Business Administration and programs with one time funding from the stimulus bill. Taken together they are a Potemkin Village to divert attention to the increasing regulatory uncertainty flowing from comparative effectiveness research requirements, projected cuts in reimbursements and other features of the new health care law that undermine innovation.
When the report discusses activity that the FDA will undertake to 'drive innovation' it largely restates work already underway. To be fair, efforts to integrate the use of biomarkers, adaptive trial designs, etc., that could accelerate development of drugs and devices takes time. Often companies, fearing it could actually doom a product by using new approaches, simply load up on old ones. But by the same token when you have the FDA demanding more safety data on the back and front end of the regulatory process and certain divisions change endpoints, revoke approvals and raise the bar so high as to make approval unlikely -- think drugs for obesity and diabetes and Avastin -- a 37 page brochure celebrating 'regulatory science' and 'partnerships' should make anyone who follows the FDA highly skeptical of real progress. Which means that specific initiatives to accelerate targeted treatments and combination products -- initiatives that were launched in 2004 under the Critical Path Initiative -- will need it's own targeted effort to be adopted.
And even here driving innovation is undermined by the stealth effort to turn the FDA into an accomplice of those seeking to use comparative effectiveness research as rationing tool:
"FDA has launched the Partnerships in Comparative Effectiveness Science (PACES) program to support the development of new mathematical methods to support patient-centered outcomes research. PACES provides funds to pilot out the technical, infrastructure, scientific and legal constructs that will be used as foundations for science computing communities involving FDA scientists and data. These activities will support scientifically sound assessments of medical interventions consistent with FDA’s public health responsibilities."
The PACES program is nothing but the mathematical manipulation of claims data -- data that tells you nothing about differences in patients -- to create the illusion that you are truly measuring differences in patients. The FDA's solicitation for the PACES program begins:
"CER could extend beyond the application of specific intervention methods to controlled clinical trials or within specific health care settings. For example CER could be used to better understand what interventions work best for individuals and subgroups within populations."
Let it be noted that there is already a lot of this sort of analysis included in clinical trials already. So why do we need another initiative of this nature? The solicitation explains:
"Both pre- and post-approval data collected and housed at the FDA can be combined with other datasets on long term health outcomes that reside in other agencies or the private sector. As personalized medicine develops, FDA expects an increase in the need for comparisons across products (and associated delivery mechanisms) to define how these products should be used in combination for individualized health care before and after FDA approval."
Does this sound like an requirement that will speed up product development. Why should clinical trials to compare response across products (CER) be a requirement for FDA approval? Why should it be a requirement after FDA approval? The additional cost and time of such trials will be hugely expensive. At least $100-$200 million per product. And by requiring the use of algorithms derived from claims data, the FDA guarantees that detection of no differences when in fact they exist in the real world.
Worse, the FDA's Janus project, an effort to standardized the submission of patient-level data to expedite biomarker development and promote smaller trial designs is being hijacked for CER purposes (from the PACES solicitation):
• Component 1- The Contractor shall plan and organize the CER project with FDA stakeholders, including participation in annual FDA-hosted planning workshops (three workshops, two days each) to define candidate CER questions and priorities, analyses strategies and datasets to be utilized.
• Component 2- The Contractor shall provide training on methodologies developed to FDA staff during two of the FDA-hosted planning workshops.
• Component 3 - The Contractor shall identify or develop appropriate analytic methodologies for CER and apply them to FDA and/or other data; develop new scientific computing strategies and hone existing computational strategies to perform these CER analyses. The Contractor shall prepare reports and manuscripts of the results suitable for publication in scientific journals.
• Component 4- The Contractor shall develop innovative clinical trial design strategies for prospective CER clinical trials and analyses of healthcare data including providing formal recommendations for best practices for submission of studies to the FDA when they involve product comparisons. These strategies and recommendations shall be documented in reports and manuscripts suitable for publication in scientific journals.
Even if all the happy talk about driving innovation was real, it could all be undermined by this effort to use FDA's most significant data program into a tool for rationing. It will drive innovation all right, off a cliff.
I believe that senior leadership at the FDA from Commissioner Hamburg on down have the best of intentions. And I know for a fact that Janus and other activities undertaken in response to the Critical Path were to be used to bring new and better products to market with greater speed and at less expense. It's those who want to slow down the development and adoption of innovation and who have politicized the FDA with CER claptrap that are at fault. They think they know better and their monstrous certainty will kill innovation for decades to come.
