Over the past two weeks, we have heard from patients, patient representatives, and clinicians through the Centers for Medicare and Medicaid Services’ (CMS) Patient-Focused Listening Sessions on the first 10 drugs selected for the Drug Price Negotiation Program (DPNP). NPC has previously weighed in on the importance of rigorously incorporating patient perspectives on the value of medicines, identifying numerous areas where the patient voice should be used to inform the Agency’s drug evaluation, including identifying unmet needs, patient preferences for treatment attributes and effects, and clinical benefits. We watched all 10 listening sessions, collecting data to assess the breadth of patient input and experiences the Agency heard to inform their evaluations. Patients and patient representatives shared compelling stories, effectively communicating the burden of their conditions, the therapeutic benefit of selected drugs, and experiences with barriers to access. We appreciate CMS’s addition of the patient-focused listening sessions in response to stakeholder feedback. However, the CMS’s approach to the sessions was not without limitations. Here, we summarize three takeaways from CMS’s listening sessions that should inform the Agency’s approach to future patient engagement.
1) The passive listening format CMS instituted for the patient-focused listening sessions, while an important first step in patient engagement, provided a limited window for one-way communication with patients and their representatives. In their revised Guidance on the implementation of the DPNP, CMS instituted patient-focused listening sessions as part of the Agency’s approach to identifying relevant outcomes and considering applicable evidence on clinical benefit and patient experience. In each session, patients and patient advocates articulated the burden of their conditions and highlighted the impact of the innovative products selected for the DPNP.
However, consistent with others’ concerns expressed at the start of the sessions, we noted that the passive listening format of the sessions has limited the Agency’s ability to obtain structured patient input on critical aspects of their evaluation. CMS received important but limited feedback on how Medicare patients prioritize clinical outcomes and indirect treatment benefits for the selected drugs, the diversity of patient preferences for treatments, and the heterogeneity of treatment effects. The lack of structured input into these critical dimensions of drug evaluation amplifies questions about how CMS can better provide structured opportunities for continuous patient engagement that meaningfully impacts their decision-making. We hope CMS will continue to learn from established processes for two-way patient listening sessions, including those used by the Food and Drug Administration, as well as best practices for incorporating the patient voice in comparative clinical effectiveness. We further recommend CMS improve transparency about what aspects of the patient experience the Agency considers important in their decision-making, to inform ongoing dialogue between patients, their representatives, and CMS.
2) The patient-focused listening sessions have fallen short of capturing diverse patient and caregiver voices. Systematically and rigorously incorporating patient perspectives on the selected drugs is essential to ensure that patients have a voice in decisions that affect their health and wellbeing. Any type of drug assessment that lacks representation from patients and patient organizations fail to capture the full range of perspectives on value. For the DPNP specifically, people with Medicare and subgroups of Medicare beneficiaries may have different priorities, preferences, and clinical effects. Despite stating that approximately 20 speakers would be selected per session, CMS heard from an average of only about 11 participants per drug. It is unclear whether this gap is because of limited patient response or a decision by CMS to further limit the number of participants; if the former, the limited notice offered by CMS and lack of clarity around how CMS would use patient input may have impacted engagement with the patient community. The majority of participants in the listening sessions included patients speaking exclusively as an individual (35%), patients and advocates speaking on behalf of an advocacy organization (54%), and clinicians (8%). Where possible, we categorized patients speaking exclusively as an individual by Medicare eligibility, finding that roughly 49% of those patient participants were Medicare beneficiaries. The group of patients speaking exclusively as an individual included few, if any, Medicare beneficiaries over the age of 75, who comprise 37% of the Medicare population. A lack of gender representativeness was also clear in most sessions; two sessions lacked any female patient participants and many other sessions heard from very few male patients.
Patient voices similarly lacked race/ethnicity diversity. While patient advocates provided important information on health disparities among diverse racial and ethnic groups, the three-minute time limit for comments restricted their ability to expand upon differences in health outcomes and preferences across the diverse patient populations they represent. Beyond these important remarks about health disparities, it was unclear whether there was substantive representation from underrepresented communities in the listening sessions. The limited patient discussion around many important social determinants of health, including transportation, health literacy, and support while navigating the healthcare system, underscores the way in which the lack of representation in the sessions may mean that CMS is missing important aspects of treatment benefits during the MFP determination process.
In their comments surrounding the revised Guidance, CMS noted that the Agency’s Strategic Plan builds health equity into its core functions, including the DPNP. The sessions thus far have come up short in their ability to capture input from diverse racial and ethnic groups, caregivers, and patients above the average age of Medicare beneficiaries, suggesting that more robust patient engagement, including through surveys, advisory panels, patient experience dossiers, and other forms of direct engagement will likely be needed in future years to obtain representative patient input that informs CMS's evaluations.
3) Access matters to patients. Throughout the listening sessions, patients and advocates expressed concerns that CMS’s efforts to lower drug prices could actually make it harder for patients to access the treatments they are prescribed and may increase their out-of-pocket costs. During an initial patient listening session, a significant portion of the dialogue—nearly 20%—centered on access-related concerns. Patients discussed the potential that drugs selected for the DPNP would be subject to greater utilization management and placed on tiers with higher copayments/coinsurance. NPC continues to be concerned that the perverse incentives that remain in the ecosystem may lead to adverse tiering, formulary-driven switching, increased utilization management, or other reductions in beneficiary access to selected drugs, thwarting the intent of the program. A leading academic voice in benefit design has similarly stressed there is no guarantee that lowering the prices paid by Medicare Part D plans for selected drugs will directly benefit patients through lower out-of-pocket costs. While CMS states the agency is concerned about adverse tiering or utilization management hurdles for selected drugs, it remains to be seen whether their oversight using their existing annual formulary review process will be sufficient to deter use of these access barriers on selected drugs or non-selected drugs in the same class. The impact of the IRA on patients must remain central to discussions around the law’s potential impact and unintended consequences. CMS has stated that the agency may consider implementing new requirements for formulary coverage of selected drugs if it identifies that Part D sponsors are not providing beneficiaries with meaningful access to selected drugs. NPC and others will be closely monitoring changes to patient access as a result of the IRA and encourage the Agency to mitigate decreased access or discriminatory behavior.
We commend the participants in the listening sessions for the considerable effort they took in publicly sharing their experiences. As we move into the next phases of CMS’s evaluation process, several areas of significant and impactful uncertainty remain, including how the patient, patient advocate, and clinician input the Agency heard through the patient listening sessions will inform and influence CMS’s evaluation and price determination. It remains critical that CMS be transparent in the ways in which patient input informs and influences each stage of the evaluation process. In future sessions, we call upon CMS to continue to evolve their opportunities for patient engagement to align with evidence-based practices for obtaining purposeful and representative information.