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Centers for Medicare & Medicaid Services

View Public Comments for CED Public Solicitation

Burkholder, Randy

Coverage and Analysis Group
Centers for Medicare and Medicaid Services
Mailstop: C1-12-28
7500 Security Boulevard
Baltimore, MD 21244

Re: CED Public Solicitation

Dear Sir or Madam;

The Pharmaceutical Research and Manufacturers of America (PhRMA) appreciates this opportunity to respond to CMS’ November 7 request for public input on the Medicare coverage with evidence development (CED) policy. PhRMA is a voluntary nonprofit organization representing the country’s leading research-based pharmaceutical and biotechnology companies, which are devoted to developing medicines that allow patients to lead longer, healthier, and more productive lives.

PhRMA appreciates CMS’ willingness to examine ways to improve the CED policy as part of its commitment to improving health outcomes for Medicare beneficiaries. In particular, the agency sought input on: implementation of CED through the national coverage determination (NCD) process or other avenues under Part A and Part B; potential impact of CED on the Medicare program and its beneficiaries; and suggested approaches to CED to maximize benefit to Medicare beneficiaries.

In response to CMS’ general request for input, and the specific topics identified above, PhRMA recommends that the agency:

  • ensure that beneficiaries have access to innovative, evidence-based items and services outside the context of CED policies;
  • maintain implementation of CED through the national coverage determination process;
  • clarify the statutory basis for the CED policy;
  • continue to apply CED only ”infrequently,” and in those areas where it is most appropriate and likely to be effective;
  • describe the factors CMS will consider in determining whether CED is warranted;
  • avoid placing an undue administrative burden on providers; and
  • define open, transparent procedures and ensure predictable CMS decision-making.

We provide more details on each of these recommendations in the comments below.

1) Support patient access to medically appropriate, evidence-based items and services under Medicare

PhRMA supports CMS’ statement in the notice linking CED to the goal of accelerated beneficiary access to medically appropriate items and services. CMS’ revised CED policy should give patients and providers assurance that the policy will result in true expansion of Medicare coverage, and will not have the effect of restricting the individual patient’s access to medically appropriate care that otherwise would be covered.

This is particularly important in light of the comments CMS received on the prior CED guidance document from patient and provider organizations, many of which stressed the importance of ensuring that the policy does not inadvertently create barriers to patient access to medically appropriate care, including access to drugs for medically appropriate off-label uses.

PhRMA recommends inclusion of two provisions in CMS’ CED policy to address these concerns. Given the importance of local coverage to beneficiaries’ access to needed services, the CED policy should include language providing assurance that access to items and services that would otherwise be available via local or regional Medicare coverage will not be restricted by CED. This would be consistent with the initial CMS draft guidance on CED, and with the approach the agency took in its CED-type policy for off-label uses of four colorectal cancer drugs in 2005. As in our October 2006 comments on CMS’ prior CED guidance document, we ask the agency to preserve this important patient safeguard by including the following language:

“CED will not reduce the importance or frequency of local coverage determinations as a pathway by which treatments and technologies are made available to Medicare beneficiaries. For items and services covered by CED, contractors will continue to exercise discretion in deciding whether to cover these items and services for beneficiaries not participating in the CED protocol.”

In addition, Medicare statute and coverage policy support the important role that off-label use of medicines plays in providing beneficiaries with access to needed care. This reflects the broad recognition of the importance of off-label use of treatments in the optimal care of many patients. Consistent with our prior comments, CMS should include in its revised CED policy a statement that CED will not be applied to the types of off-label uses of medicines for which Medicare coverage already is provided under existing Medicare statute and coverage policy. For example, CED should not apply in cases where an off-label use is listed in a recognized drug compendium or supported by peer-reviewed literature from an appropriate medical journal.

CMS’ current statutory framework and agency policy on coverage of drugs and biologicals under Medicare Part B provides a sound framework for ensuring timely beneficiary access to medically appropriate, evidence-based treatments. Within this coverage context, use of treatments typically evolves rapidly over time, as does the underlying evidence base. The extensive changes that occur in compendia listings and labeled indications for drugs and biologicals after they are initially approved indicate the considerable extent to which evidence development already occurs. Seeking to apply CED policy in this context therefore is unnecessary, and may have the unintended effect of constraining evidence development that would have otherwise occurred. In addition, experience to date in CMS implementation of a CED-like policy for four anticancer drugs for certain off-label uses illustrates some of the inherent challenges in applying CED to drugs and biologicals. CMS’ approach of not applying CED to drugs since 2005 is appropriate and reflects the fact that the current statute and Medicare policy provide a sound framework for beneficiary access to needed therapy and ongoing evidence development, as well as the practical challenges of applying CED to Part B drugs and biologicals.

For these reasons, PhRMA requests that CMS revise the Guidance to include the following text:

“CED will not be applied to an unlabeled use of an FDA-approved drug or biological that may be considered medically appropriate by a local or regional Medicare contractor based on its support in major drug compendia, peer-review literature and/or accepted standards of medical practice. CED will also not be applied to a drug if its use is supported in a recognized drug compendium or in published peer-reviewed medical literature, or is otherwise determined by a contractor, as provided for in the Medicare Benefit Policy Manual, to be reasonable and necessary.”

2) Maintain implementation of CED via the national coverage determination process

The Medicare statute defines an NCD as “a determination by the Secretary with respect to whether or not a particular item or service is covered nationally” under Medicare. As described in the Nov. 7 CMS website notice, CED would continue to be used by CMS to make decisions that affect coverage of individual items and services in the care of Medicare beneficiaries. The only means for CMS to make such decisions is through the national coverage determination process. Were it otherwise, the result would to disaggregate elements of the NCD process, severely diminishing the transparency that the agency has worked to foster and that the Congress has identified as a policy priority.

3) Clarify the statutory basis for CED.

Although CED, as currently conceptualized by the agency, clearly relates to coverage decision-making, the statutory basis for the actual policy should be more clearly described by CMS. This is consistent with the Medicare Payment Advisory Commission’s June 2010 report, which stated that lack of a clearly articulated statutory basis for CED has been a barrier to effective application of the policy.

In its final CED guidance, CMS referenced two separate statutory provisions for the two types of CED (coverage with appropriateness determination (CAD), which referenced Social Security Act section 1862(a)(1)(A), and coverage with study participation (CSP), which referenced Social Security Act section1862(a)(1)(E)). A number of comments to CMS sought more detailed descriptions of how these authorities would guide the agency’s application of CAD and CSP.

To the extent CMS relies on 1862(a)(1)(E) in its revised CED policy, the agency should describe in more detail how the requirements of this provision will guide CED decisions. In particular, 1862(a)(1)(E) essentially authorizes CMS to covers items and services “in the case of research conducted pursuant to section 1142, which is . . . reasonable and necessary to carry out the purposes of that section.” CMS’ discussion of this provision should include a description of the role of the Agency for Healthcare Research and Quality (which administers Sec. 1142) in carrying out CED, and ensure that AHRQ’s work related to CED is included in the biannual reports to Congress required under this section.

4) Describe the factors CMS will use to determine whether an item or service undergoing a national coverage analysis warrants application of coverage with evidence development.

CMS should describe the specific factors it will use to determine whether an item or service under review will be subject to CED. The agency identified some broad factors in its 2006 CED guidance (for example, stating “coverage with study participation,” one type of CED, “might be appropriate” in instances where “the evidence includes assurance of basic safety; the item or service has a high potential to provide significant benefit to Medicare beneficiaries, and there are significant barriers to conducting clinical trials.”). CMS should refine and expand these broad factors, which would improve predictability of national coverage decision-making for patients, providers and product sponsors.

5) Consistent with existing CMS policy, continue to apply CED only infrequently, and only in those areas where it is most appropriate and likely to be effective.

PhRMA supports the principles that were included by CMS in the prior guidance, including those stating that CED will be applied infrequently, will be implemented through open and transparent procedures, and will not replace or duplicate FDA authority. As described above, we believe drugs and biologics covered under Part B generally should not be subject to CED – current statute and policy provide a sound framework for coverage of these treatments for evidence-based, medically appropriate uses, while also providing for ongoing evidence development.

In contrast, CMS’ use of a CED-like policy for lung volume reduction surgery has been identified as one example of a CED “success story” that ultimately led to a change in Medicare coverage policy. This example illustrates the need to focus CED on non-drug items and services with comparatively lower levels of evidence at introduction (such as new surgical procedures like LVRS that are not subject to FDA premarket review), for which the magnitude of change in the outcome being evaluated is sufficiently large, and for which the clinical question being evaluated is relatively stable.

6) Ensure CED does not impose undue burdens on patients and care providers

Implementing CED decisions has the potential to impose considerable administrative burdens on physicians and providers. In comments on CMS’ prior CED guidance, PhRMA and many other organizations expressed concern about the need for the policy to clarify how the agency will generate the required research without creating excessive burdens for providers. For example, some hospital organizations cautioned that the agency may have underestimated the potentially significant burden that CED implementation could place on providers. As CMS revises its CED policy, it should work with providers to identify approaches that will not result in excessive administrative burdens.

7) Describe open, transparent processes and ensure predictable decision-making.

CMS should ensure that its revised CED policy clearly describes the circumstances in which CED will likely be applied, and defines an open, transparent process for finalizing and implementing CED coverage policies. The process should provide opportunities for broad input on proposed CED policies, and should reflect the important role that physicians and clinical experts and researchers will play in effectively implementing CED policy. As suggested in our prior comments, this could include: CMS posting draft proposals for coverage with evidence development prior to issuance of a draft decision memo; collaboration with stakeholders on appropriate research questions to be answered and the most appropriate, least burdensome means of answering them; a clear, independent mechanisms for evaluating and reporting results of CED studies. In addition, CMS should disclose who initiates the CED request (whether CMS internally or an external party) as part of the CED process.

PhRMA appreciates this opportunity to provide input to CMS on the topic of coverage with evidence development. As the agency works to develop a new policy, we ask that you consider the issues we have identified above. In addition, we encourage CMS to work closely with a broad range of stakeholders from the patient, provider and research communities to establish a policy that is practical, predictable, and expands beneficiary access to high quality, evidence-based medical care.


Richard I. Smith
Executive Vice President, Policy and Research

Randy Burkholder
Deputy Vice President, Policy