Getting Patient Input Right During the IRA Implementation

Important questions remain on how patient input will be incorporated in the IRA process as we approach the release of the list of drugs selected for negotiation.

Systematically and rigorously incorporating patient perspectives on the value of medication is essential throughout a drug evaluation. CMS’s initial Guidance on the implementation of the IRA’s Medicare Drug Price Negotiation Program offered no clear insight into the Agency’s plan to engage patients and incorporate their input in its drug price setting mechanism. Yet, an NPC analysis of publicly-available comment letters submitted by stakeholders in response to the initial Guidance revealed that neither payers nor health systems encouraged CMS to incorporate robust patient engagement into the evaluation process.

In our members-only resource analyzing key stakeholder comments to the initial Guidance, we observed that many stakeholder groups, including payers and health systems, did not advocate for robust patient engagement in their publicly available comment letters.

Those who joined NPC in urging CMS to prioritize patient and caregiver input throughout the process were primarily academics (though only about half of academics who responded discussed this important aspect of IRA implementation) and non-profit or advocacy groups (National Health Council, Alliance for Aging ResearchIVI, Personalized Medicine Coalition), and manufacturers.

Even among those who commented on patient input, discussion primarily centered around patient perspectives on clinical benefits, ignoring the importance of patient voices in identifying unmet need, appropriate treatment alternatives, and distinct patient preferences for treatment attributes and effects. 

NPC’s research highlights the importance of the patient voice in drug evaluation given diverse patient preferences and experiences with drugs. Our research showed that patients may respond differently to available treatment options due to pharmacologic differences, genetic risk, or social determinants of health, creating unmet need despite existing treatments. 

Our research also showed that patient preferences for treatment characteristics and outcomes vary, demonstrating how new drugs and formulations provide patients and providers with preference-aligned treatment options, and that comprehensive approaches to measuring patient-centered value, including incorporating factors beyond effectiveness and side effects, will result in more meaningful evaluations.

In NPC’s comments to CMS regarding the initial guidance, we pointed out that the patient voice is needed to inform the evaluation of unmet need; the identification of treatment alternatives that are both clinically appropriate and satisfactory to patients; and the evaluation of the clinical and humanistic benefits of a selected drug. Patient input on clinical benefits should identify what benefits are most important to patients and capture benefits – including those to societynot captured in conventional measures of health gain. These are sometimes referred to as “novel elements of value.”

Important questions remain on how patient input will be incorporated in the IRA process as we prepare for the next step in IRA implementation: the expected release of the list of ten drugs chosen for negotiation. While CMS’s revised guidance announced the Agency would hold patient-focused listening sessions this Fall, they provide no details on the structure of those sessions – or how patient input will inform and influence each stage of the drug evaluation process.

The importance of getting this engagement right cannot be overstated, and CMS has a longstanding commitment to beneficiary engagement. As CMS begins this process with this list, it will be critical that they use evidence-based best practices for meaningful patient engagement that ensures they are receiving comprehensive and representative information directly from patients. Let’s hope we get more details soon.