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Pink Sheet – FDA/CMS Collaboration: “Sleeper Group” Goes Public With Call For Real-World Evidence

Pink Sheet – FDA/CMS Collaboration: “Sleeper Group” Goes Public With Call For Real-World Evidence

Executive Summary

US FDA, CMS and NIH are hoping to create a “single front door” for research. Article outlining collaboration on real-world evidence suggests one path forward.


The heads of FDA and CMS are outlining a vision for collaboration on development of real-world evidence ahead of the transition to a new presidential administration.


FDA Commissioner Robert Califf, Deputy Commissioner for Medical Products Rachel Sherman, and Acting CMS Administrator Andy Slavitt published a “viewpoint” piece entitled, “Knowing When and How to Use Medical Products: A Shared Responsibility for the FDA and CMS,” in the Journal of the American Medical Association November 7.


The article notes the historical and public health reasons why FDA and CMS have different standards for evidence and play different roles in the health care system, but cites an important, shared opportunity: “changes in the organization of health care and in the larger information ecosystem should allow the FDA and CMS to move increasingly toward use of shared sources of evidence while still applying the most appropriate criteria to their decision making.”


The publication came on the eve of the presidential election and a few days after Slavitt and Califf talked about real-world evidence development as a priority during the Prevision Policy/Friends of Cancer Research Biopharma Congress. Slavitt, in particularly, highlighted his work with sister agencies as an important and satisfying area of focus as acting administrator of the Medicare and Medicaid agency during his November 3 remarks.


Slavitt noted that he, Califf and National Institutes of Health Director Francis Collins “have established tri-lateral leadership discussions among our agencies.” He jokingly referred to the monthly discussion as “a sleeper group,” but declared a broad goal: The three agencies “hope to establish critical ties that can eventually create a single front door to research safety, efficacy, coverage, and pricing – with appropriate firewalls and safeguards,” Slavitt declared.


“Together we’re making it a priority to figure out how to coordinate in ways that advance product development,” he said. He noted particularly CMS’ decision to transition its parallel review program with FDA from pilot project to permanent status.


That theme of his talk may be overlooked in light of his extensive and pointed comments about drug pricing – but is important as an indication of future directions for pro-innovation work in CMS. (Also see “Drug Pricing: CMS Seeing ‘Too Many Examples’ Of Excessive Increases” – Pink Sheet, 5 Nov, 2016.)


Indeed, with both Califf and Slavitt likely to leave office in January, the JAMA article amounts to a shared vision statement and a public call for continued collaboration on real-world evidence by the next group of health agency leaders in the US.


The article outlines three areas of focus:

  1. Clinical trial demographics. FDA and CMS are “clarifying the need for including diverse populations and measuring relevant clinical outcomes within the sphere of trials conducted for regulatory approval and to inform labeling.
  2. Analyzing electronic health data. “FDA and CMS are collaborating with other federal agencies to build functional links across a range of systems developed to capitalize on existing digital information collected in the course of health care delivery, including electronic health records, insurance claims, and data housed in clinical registries.”
  3. Collaborating with the public and private sector. “The Patient-Centered Outcomes Research Institute, which is overseen by a board representing key stakeholders (including the medical products industry), is one example of an entity designed to develop this knowledge.”

“For too long, physicians, patients, regulators, policy makers, and payers have depended on suboptimal data to guide decision making because of constraints, perceived or real, about what was affordable, practicable, or desirable,” the article concludes. “But now, thanks to rapidly accelerating scientific knowledge and new perspectives, the FDA and CMS are poised to change this approach for the better and improve outcomes for patients.”

A Recurring Theme For CMS’ Slavitt

In his appearance at the Biopharma Congress, Slavitt returned to the collaboration theme in two different contexts during Q&A.


First, regarding the appropriate regulation of and reimbursement for diagnostics used in personalized medicine, he acknowledged that it is a difficult question. “It is something that Rob and Francis and I do talk about,” he said. As a core principle, he stressed, the “way to structure and construct this is not to slow down but actually to speed up.” That said, the right approach to reimbursement “is a new area that requires some statutory work to create a new regime.” It will also require a deepening of the expertise within CMS, Slavitt said.


He specifically raised collaboration in real-world evidence in response to a question about how pharmaceutical companies can develop strong cost-effectiveness arguments. “One of the priorities I put on our team is to enable health economics and outcomes research using our data,” Slavitt said. As a result, we have more data out there for people to use and test and do health economics and outcomes research on other sorts of real world evidence.”


Slavitt also noted the importance of FDA’s policies on health economic promotion as an important topic. “The other piece of it is probably has to do with labeling because …if it’s not on the label, then the concern is then they can’t claim it, and if they can’t claim it, then we can’t create a value based model on it to say you’ve reduced hospital bed days by X amount of dollars and therefore you’d offset that.”


During his Nov. 4 discussion at the Biopharma Congress, FDA’s Califf emphasized the use of real-world evidence as an important priority – essentially a legacy issue for his tenure at FDA. He also offered an understated but humorous take on collaboration with CMS.


“With CMS, part of my fun with Andy is, sometimes I feel like I am under fire,” Califf said, “but then I just call Andy an ask, ‘How many letters did you get from Congress today?’ and I feel better.”


In a more serious vein, Califf added that “this is a time when we are getting a lot of encouragement from all sides to collaborate with CMS. It is vital to everybody that we get this worked out about how to move the payment system to something based on value, because [paying based on] the number of widgets is not going t to cut. We are not going to be able to afford to do it that way.”

CDER Director Wants To “Organize and Present” Projects

The reality that the push towards greater use of real-world evidence is a long-term, post-transition project was underscored by the heads of FDA’s two therapeutic centers, Janet Woodcock (drugs) and Peter Marks (biologics) later on Nov. 4.


Marks noted work CBER did using CMS claims data to evaluate the efficacy of high-dose influenza vaccine. (Also see “Sentinel’s “IMPACT” Beyond Safety: Anti-Coagulant Study Is Early Test” – Pink Sheet, 10 Mar, 2016.)


The study, Marks said, “validates this principle that you can in certain settings use real world evidence.” However, he stressed, it relies on having “coarse outcomes,” like death or hospitalization, and it is “hard to do for an NME.” For marketed drugs, however, “it probably is a possibility,” and anything that lowers cost “is probably a good thing,” he said.


For her part, Woodcock noted that she had just participated in a briefing on real-world evidence projects in CDER that morning, and that there are about thirty projects under way. As it stands, the many different components of developing standards for data collection and analysis seem very complex. The next step is “to organize all this and present it to the world.” However, “right now it’s a little bit of a mystery to me.”…