Re: Request for Comments Regarding Evidentiary Characteristics of Coverage with Evidence Development
Dear Ms. Ellis:
The Biotechnology Industry Organization (BIO) appreciates this opportunity to respond to the Medicare Evidence Development & Coverage Advisory Committee’s (MEDCAC) questions regarding the evidentiary characteristics of coverage with evidence development (CED). BIO is the largest trade organization to serve and represent the biotechnology industry in the United States and around the globe. BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products.
As a representative of members dedicated to developing new therapies and ensuring access to them, BIO supports the use of CED when it is used to help patients gain access to otherwise unavailable drugs, biologicals and other therapies. Our members invest billions of dollars each year in clinical research to develop and disseminate evidence to help guide the effective use of their therapies. This investment generally continues after the Food and Drug Administration’s (FDA’s) stringent drug and biological approval requirements are met and often includes post-marketing studies required by FDA. We also support the dissemination of this evidence, as appropriate, to further clinical knowledge and enhance and improve the clinical decision-making process.
Before responding to MEDCAC's panel voting questions, we believe it is important to address more broadly the applicability of CED to drugs and biologicals. The Centers for Medicare and Medicaid Services (CMS) should not second-guess FDA’s drug approval decisions by requiring additional post-approval studies of a drug or biological for “medically accepted indications,” as defined by statute or pursuant to longstanding Medicare guidance, as FDA has already determined that the products are safe and effective. CED is not necessary or appropriate for FDA-approved uses of drugs and biologicals that are approved for use in the Medicare population, including disabled and patients older than age 65. Moreover, the Social Security Act’s definition of “drugs or biologicals” requires that each drug or biological be included or approved for inclusion in the United States Pharmacopoeia or be “approved by the pharmacy and drug therapeutics committee (or equivalent committee) of the medical staff of the hospital furnishing such drugs and biologicals for use in such hospital.” These requirements, combined with FDA approval, provide additional assurance that the therapy has been thoroughly reviewed by independent experts prior to coverage.
In addition, CED should not be applied to drugs and biologicals that are used for “medically accepted indications” of drugs or biologicals used in anti-cancer chemotherapeutic regimens, which include the FDA-approved uses as well as uses that are listed in certain compendia or are supported by peer-reviewed literature. Medicare also has long granted its contractors authority to determine that unlabeled uses of other drugs are “medically accepted” based on “the major drug compendia, authoritative medical literature and/or accepted standards of medical practice.” By using authoritative compendia and medical literature to define “medically accepted indications,” the statute and Medicare’s guidance protect beneficiaries’ timely access to drugs and biologicals while also ensuring that Medicare’s coverage policies are truly evidence-based.
Finally, FDA and CMS each have separate and distinct mandates that CMS must adhere to in any redesign of the CED policies. While BIO supports the two agencies working together to ensure patients have access to needed therapies, it is critical that the unique missions of these two agencies remain distinct, and not be comingled or compromised in the course of CED application. Congress deliberately bestowed FDA and CMS with distinct authorities and standards for approval and coverage decisions respectively, consistent with the different missions and constituencies of the agencies. FDA has the appropriate combination of expertise and resources to review and approve study design and results of clinical trials needed to demonstrate that drugs and biologicals are safe and effective. CMS should not attempt to use its limited resources to duplicate these capabilities. The industry should not be put into a "double jeopardy" situation (i.e., CMS should not apply CED to those products that are already subject to REMs or other post-market studies required by the FDA).
BIO appreciates CMS’ attempt to solicit stakeholders’ input regarding its CED policies and understands that CMS leadership is seeking comments to help develop new guidance on CED. We urge CMS to make any change to CED in response to, or as a result of, the MEDCAC meeting, subject to an additional notice and comment period so that stakeholders have the opportunity to comment on specific changes being proposed.
Medicare Benefit Policy Manual, ch. 15, § 50.4.2.