New CMS Guidance for Biotech Industry On Coverage With Evidence Development: It’s All About The Outcomes

This is a guest post from Robert E. Wanerman, a member of Epstein Becker Green’s Health Care and Life Sciences practice. Do you have a response to Robert’s post? Respond in the comments section below.

Wanerman_Robert_4x5For many biotech manufacturers, obtaining Medicare coverage is a significant milestone in commercializing an item or service. Although Medicare coverage for specific items or services that use biotechnology methods for their production, design or delivery can vary in different parts of the United States, a small number of them are guaranteed nationwide coverage if the Centers for Medicare and Medicaid Services (“CMS”) has issued a National Coverage Determination (“NCD”).  Obtaining a NCD is a difficult process for companies in the biotech industry, and depends on factors such as the quantity and quality of clinical data, including outcomes data, and a consensus of professional opinions or practice guidelines.

Since 2006, CMS has carved out a middle ground for covering items and services when it believes that the clinical data is insufficient to justify issuing a NCD, but believes that the item or service shows promise for improving outcomes among Medicare beneficiaries.  In these situations, CMS has the discretion to cover an item or service with conditions under its Coverage with Evidence Development (“CED”) authority.  If CMS agrees that CED coverage is appropriate, the sponsor then has to design a study that CMS will approve.  How CMS makes that second decision whether or not to approve a study design is often poorly understood.  On November 20, 2014, CMS released its latest guidance on CED coverage, which adds some additional clarification for interested biotech parties.

The new CMS guidance builds on its past CED approvals and typically contains several requirements for an approvable CED study:

  • Medicare beneficiaries must be enrolled in a clinical trial approved by CMS;
  • The study design must answer the questions framed by CMS in its approval;
  • The study must comply with all human subject protection regulations;
  • The study must be registered on before any enrollment can occur (registry studies must also be listed in the Agency for Healthcare Research & Quality’s (AHRQ) Registry of Patient Registries (RoPR));
  • The study protocol must specify the method and timing of public release in either a peer-reviewed journal or in a publicly-available registry of all identified outcomes to be measured, including release of outcomes if outcomes are negative or the study is terminated early, which must be made within 12 months of the study’s primary completion date (even if the trial does not achieve its primary aim);
  • The study protocol must explicitly discuss beneficiary subpopulations affected by the item or service under investigation, particularly traditionally underrepresented groups in clinical studies, how the inclusion and exclusion criteria affect enrollment of these populations, and a plan for the retention and reporting of the subpopulations; and
  • The study protocol must explicitly discuss how the results are or are not expected to be generalizable to affected beneficiary subpopulations.

What is new about this guidance is CMS’s focus on outcome measurements.  Two new elements stand out: first, it strongly suggests that in order to approve a CED study, there must be a comparator in a control group in order to minimize potential biases and to give CMS a better grounding to evaluate the effectiveness of the new item or service at the end of the study period.  The comparator in the control arm of the study may be a placebo or standard of care treatment.  Although CMS does refer to blinding as a technique to minimize the placebo effect, the Guidance does not address evaluating (1) the risks to Medicare beneficiaries of sham procedures, such as a spine procedure to relieve back pain as discussed in the Guidance, or (2) how to offset the disincentives for a Medicare beneficiary to enroll in a clinical trial if they know that there is a chance of receiving a sham procedure instead of treatment.

The second new element in the Guidance is CMS’s suggestion that the CED study design include interim analyses that would be shared with CMS.  Although this could potentially expedite a final NCD decision if the results are strongly positive, it may increase the burdens on the trial sponsor and could become problematic for the sponsor if the interim data is not promising and CMS in response changes its demands for data in order to make a final determination.

Although the Guidance is something less than a roadmap for biotech manufacturers and other stakeholders seeking Medicare coverage, it does provide a clearer idea of CMS’s expectations when reviewing a request for CED protocol. As in the past, interested parties are encouraged to meet with CMS’s Coverage and Analysis Group and to maintain a dialogue with the agency throughout the process, to exchange ideas and to fine-tune the proposed clinical trial in order to reach a consensus with CMS.

About the author:

Robert E. Wanerman is a member of Epstein Becker Green’s Health Care and Life Sciences practice. His practice concentrates on regulatory, reimbursement, and compliance matters affecting biotech and health care manufacturers, service providers, and investors in biotech and health care organizations. Robert has extensive experience counseling clients in matters arising under the Medicare and Medicaid programs. He can be reached at [email protected].

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