Recent NHC Comments Submitted to FDA & CMS


By Eric Gascho, NHC Vice President of Policy and Government Affairs

The National Health Council frequently submits letters and comments on the administration’s rules and actions when its subjects are relevant to our policy work and the work of our patient organization members and when the result could affect patients with chronic diseases and disabilities. Over the past several weeks, the NHC has submitted comments to the Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS) on the following:

  • FDA discussion documents on forthcoming guidance on patient engagement guidance in drug development;
  • FDA discussion document on patient engagement in medical device development;
  • CMS guidance on “State Relief and Empowerment Waivers;”
  • CMS Advance Notice of Proposed Rulemaking entitled “International Price Index Model for Medicare Part B Drugs;” and
  • CMS proposed rule entitled “Modernizing Part D and Medicare Advantage to Lower Drug Prices and Reduce Out of Pocket Expenses.”

Comment Letters to the FDA

December 14, 2018: We commented on the FDA’s patient-focused drug development (PFDD) discussion documents related to Guidance 2: “Methods to Identify What is Important to Patients” and Guidance 3: “Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcomes Assessments (COAs).” The NHC provided the following overarching comments:

Consideration of patients as partners: The NHC believes that PFDD happens at two levels: (1) Patients engaged as partners informing the drug-development process, and (2) patients participating as study subjects, such as those that provide data on the patient experience and in clinical trials that have been designed in a more patient-centered way. In general, the referenced discussion documents focus heavily on the latter and not enough on the former. We believe the focus should be both.

Patient centered versus patient reported: FDA should reiterate and clarify that not all patient-reported outcomes (PROs) are patient centered and not all patient-centered outcomes are reported by patients, as confusion appears to be highly prevalent among many stakeholders.

Utility of patient experience data is not limited to COAs: The NHC supports the FDA’s goal of developing guidance that provides structure for incorporating patient perspectives as a standard practice. We recommend that the Agency clarifies other means by which patient-experience data can be used throughout the product lifecycle.

Patient engagement is an evolving field, and flexibility is needed as methods and data sources evolve and advance: As this evolving and emerging science advances forward – it would be helpful for the Agency to provide stakeholders with ample patient-engagement and patient-experience data examples.

January 8, 2019: We commented on the Center for Devices and Radiological Health (CDRH) Patient Engagement Advisory Committee (PEAC)’s Discussion Document from its November 15, 2018 meeting. The NHC provided comments on five different, overarching sections:

Definition of Patient Engagement: The NHC agrees with the FDA that a common definition of “patient engagement” is necessary to establish a shared understanding of key concepts and to ensure that work progresses in a cohesive, consistent manner. However, the CDRH’s proposed definition is not completely aligned with existing FDA work, which will present challenges for stakeholders in the future. The NHC lists a few recommendations for the CDRH to adopt in order to promote efficiency and demonstrate alignment across the entire FDA.

Value and Impact: We agree with the benefits of patient engagement outlined in this section and encourage CDRH to further emphasize the benefits and impacts that patient involvement and the patient perspective can bring beyond simply the operational aspects of clinical trials, particularly around quality and relevance of data.

Challenges: We believe the document does not capture the full range of challenges that may affect the success of patient engagement in clinical trials. The NHC has explored many of these challenges to patient engagement in research, development, and review of medical products through multi-stakeholder forums; we encourage CDRH to leverage these findings in its guidance development process.

Approaches: We strongly encourage CDRH to align with other stakeholders who are also doing work in this area, including the Center for Drug Evaluation and Research (CDER).

Conclusion: The NHC recommends CDRH create opportunities to more systematically gather stakeholder input to inform the framework for the planned guidance.

Comment Letters to CMS

December 24, 2018: We commented on CMS guidance on “State Relief and Empowerment Waivers.” The NHC requested the withdrawal of the proposed expansion of these waivers, as we do not believe that the financial benefits of lower premiums for healthy individuals contemplated under the Guidance will outweigh the likelihood or magnitude of harms to individuals with chronic diseases and disabilities. Specifically, we are concerned that:

The policies contained in the Guidance could adversely impact the risk pool and erode the Affordable Care Act’s protections for individuals with pre-existing conditions.

State waivers that promote purchase of non-compliant coverage could confuse consumers and leave them susceptible to being significantly under-insured.

December 31, 2018: We commented on the CMS Advance Notice of Proposed Rulemaking entitled “International Price Index Model for Medicare Part B Drugs” Although we support CMS in testing new models of care that align payment incentives with value and quality, our response to the proposed rule fully depends on how CMS addresses the missing elements and whether the proposal will restrict access to needed therapies for people with chronic diseases and disabilities.

General recommendations:

  • CMS must develop broad patient safeguards for all Medicare policies, including quality measures, and clearly articulate its process for monitoring and addressing access issues. We urge CMS to implement sufficient quality measures and other patient protections, and to articulate specific plans to monitor care delivery and quickly address patient access issues
  • Any projected or realized cost savings should be shared with Medicare beneficiaries to reduce their out-of-pocket costs. We strongly urge that CMS apply any projected and realized cost savings from proposals to reduce health care costs to beneficiary cost-sharing requirements to reduce out-of-pocket costs for our nation’s seniors.
  • Medicare demonstrations should be small and targeted so that evaluation of access challenges and cost savings can be done successfully. CMS should consider smaller demonstrations that allow for easier assessment of the alignment between the policy change and subsequent outcomes.

Specific concerns:

  • CMS must serve an active role in overseeing relationships between those providing and facilitating care to Medicare beneficiaries and create guardrails to ensure access. Thus, we strongly urge CMS to take an active regulatory and oversight role in the agreements between manufacturers, distributors, and providers, specifying obligations to ensure access and safety, managing conflicts of interest, and developing appropriate guardrails.
  • CMS must carefully consider its methodology for determining provider payments to ensure any new proposal does not have the unintended consequence of incentivizing selection of low-value treatments.  Without detail on how add-on fees will be calculated, we are concerned that this attempt to remove incentives to prescribe high-cost/low-value care may create the opposite impact by incentivizing low-value care if it is less expensive for providers to administer.

January 25, 2019: We commented on the CMS proposed rule entitled “Modernizing Part D and Medicare Advantage to Lower Drug Prices and Reduce Out of Pocket Expenses.” While we support efforts to improve competition, promote better negotiation, lower list prices, and reduce out-of-pocket costs, we have significant concerns with the proposal. Specifically:

The NHC is concerned that the proposed additional exceptions to plan requirements for covering drugs within the protected classes will have a disproportionate impact on individuals with chronic diseases and disabilities.

Proposed allowance of utilization management and cost-containment tools for Part B drugs within Medicare Advantage will impede patient access and create a coverage disparity between MA plans and fee-for-service Medicare.

We did, however, support some of the provisions of the proposed rule. Specifically, the NHC supported CMS’ proposals to:

  • Enhance the information plans provide to patients and providers;
  • Prohibit plans from implementing “gag clauses;” and
  • Reduce cost sharing by re-defining “negotiated price.”

To read these and other previous letters and comments in greater detail, visit