The Reducing Inflation Act (IRA) required the creation of the Drug Price Negotiation Program (DPNP), which focused on creating a process for Medicare that would involve manufacturers in reducing the prices paid for certain products under Medicare Parts B and D. While there are some restrictions on drug selection and what criteria the Centers for Medicare and Medicaid Services (CMS) can use in the new DPNP, the interpretation and implementation of this legislation creates new opportunities to engage patients and caregivers in meaningful ways .
The IRA specifically identifies nine factors that Medicare may consider “applicable to drugs as a basis for determining offers and counteroffers” when negotiating the highest fair price. At first blush, these nine factors may seem straightforward, but as CMS staff implement the program according to the law, they will undoubtedly face new problems that each factor presents. Five factors were categorized as manufacturer-specific and four described the evidence on alternative treatments. Manufacturer-specific data includes items related to research and development costs, production and distribution costs, federal funding received during the discovery process, pending and granted patents, and revenue data. Information specific to alternative treatments includes the extent to which the drug facilitates treatment of the disease, prescribing information from the Food and Drug Administration, the extent to which the drug addresses an unmet need, and comparative effectiveness studies of different treatment options ( CER) medical conditions.
The new mandate of CMS, particularly the mandate to consider factors relevant to comparative clinical effectiveness, has important implications for the ongoing effort to advance patient-centeredness in healthcare decision-making. CERs are designed to help us make more informed decisions that improve healthcare at the individual and population levels; patient engagement can be a key part of the CER process. While nothing in the legislation calls for a patient-centered approach, the evolution of patient engagement approaches and valuable lessons learned through patient-informed CERs could provide a path for CMS to implement established processes at the outset of creating the DPNP. By establishing a commitment to real patient involvement early in the process, the DPNP can work to build trust with diverse stakeholders and become a trusted program.
A patient-centred approach to addressing these CER negotiating factors can help ensure that the development of the DPNP formally includes a range of outcomes and data sources—including those relevant to patient and caregiver experience—that are fundamentally patient-centred so that Medicare drug benefits are implemented more in line with the goals of the patients they are designed to serve. Importantly, the patient-centredness safeguards contained in the IRA regulations are particularly relevant to CER considerations, underscoring the primary importance of this criterion when considering patient-centredness. In particular, CMSs need to consider “the relative clinical effectiveness of such drugs and therapeutic alternatives to such drugs in specific populations such as the disabled, the elderly, the terminally ill, children, and other patient populations.” When we assess how CMSs Certain patient-centred issues may arise when using CER evidence for drug price negotiations (see Exhibit 1).
Exhibit 1: Examples of patient-centered issues raised by several factors identified in the Reducing Inflation Act as the basis for determining offers and counteroffers during negotiations
Source: Author’s analysis. notes: FDA is the Food and Drug Administration. PFDD is patient-centered drug development. CERs are Comparative Effectiveness Studies.
As the IRA incorporates specific language into recognizing CERs as appropriate evidence for alternative treatments that can be used in drug price negotiations, advancing a patient-centered approach to CER stakeholder engagement is critical to informing decisions about drug pricing negotiations in Medicare.
In 2010, the Affordable Care Act mandated the creation of the Patient-Centered Outcomes Research Institute (PCORI) to prioritize CERs based on a multi-stakeholder approach involving patients as advisors. As a consultant, patients can provide critical feedback throughout a research project or program. Specifically, the law requires that appointments to expert advisory groups “shall include practicing and research clinicians, patient representatives, and experts with relevant subject matter experience in science and health services research, health service delivery, and evidence-based medicine.” However, due to Lack of consensus on how to apply the principles of community-based participatory research to CER and patient-centred outcomes research makes implementing this regulation challenging for researchers; in short, there is no one size fits all Patient Engagement Methods. However, certain methods may be applicable to various functions of DPNP. For example, a patient-centred advisory committee with representatives from several different patient communities can provide broad feedback to the DPNP during policy and program development, whereas a more disease-centred approach (e.g., patient/care Personnel interviews, focus groups, surveys) can be used when the DPNP makes decisions about specific medicines.
As noted previously, the IRA does not explicitly mandate that CERs used in drug pricing negotiations must be patient-centred; however, work in the PCORI research portfolio over the past 12 years has provided researchers and Participation-focused CER. PCORI’s 2019 reauthorization legislation expanded PCORI’s mandate to support research that captures data on economic impacts and costs, such as care-related out-of-pocket medical costs, as well as non-medical costs, such as informal care costs or reduced work site productivity. This reauthorization language complements new IRA language to help support CMS leaders responsible for drug price negotiations. PCORI’s extensive work in defining standards related to patient-centered research and ensuring patient participation in the research process points to key issues for CMSs to keep in mind as they begin to establish processes and standards for the DPNP.
Explicit consideration of stakeholder input throughout the work process increases transparency and builds trust in the process. CMSs can use a variety of engagement methods to build patient-centered decision-making processes into their DPNP. First, the DPNP can establish a patient-centered stakeholder advisory committee that includes patients and caregivers from diverse backgrounds with varying healthcare experiences for different types of diseases and conditions. This general advisory committee could be tasked with reviewing different aspects of the DPNP’s work (eg, identifying treatment alternatives, identifying specific outcomes of interest when interpreting CER results) and helping to disseminate DPNP reports to the general population.
Second, since DPNP requires more disease-specific feedback, targeted patient engagement strategies can be employed to ensure a more representative sample of patient experience is captured. This may include semistructured interviews, focus groups, Delphi groups (a consensus-building process developed by the RAND Corporation in the 1950s), and surveys for diseases of interest.
Finally, PCORI provides the agency with a potential resource to ensure it is capturing results and data that support patient-centered needs. It may be advantageous for the DPNP to collaborate directly with PCORI to ensure that CER results identified in PCORI-funded studies can be quickly integrated into the DPNP workflow. For example, a CMS-PCORI dashboard could be developed specifically for DPNP officers with up-to-date research information, including active research that has not yet been published.
As CMS staff work to develop and implement this new DPNP, incorporating patient and caregiver feedback early and often can help identify or incorporate factors relevant to the evidence for CERs being considered during negotiations. A systematic approach to collecting and interpreting public comments can further the DPNP’s mission while creating a framework that instills trust.
This work was supported by the Pharmaceutical Research and Manufacturers of America (PhRMA). PhRMA was not involved in the preparation of this article or the decision to submit the manuscript for publication. Dr. Mattingly reported receiving consulting fees from the Arnold Foundation, Johns Hopkins University, Vanderbilt University, and PhRMA, as well as collaborations from the Food and Drug Administration (FDA) and the National Institute on Minority Health and Disparities. This manuscript has nothing to do with research support. Dr. Mullins reported receiving personal consulting fees from Bayer, Merck, Pfizer, and Takeda, as well as grants and contracts to UMB, and he served on assignments for AstraZeneca, CareFirst Blue Cross Blue Shield, FDA, Lilly, National Institutes of Health Principal Investigator of the Institute Health, Novartis and Truist.