Over the past two decades, health care researchers and policy-makers have worked to address the challenge of listening to patients – that is, of truly considering the lived experiences and perspectives of individuals with an illness or injury in health research and decision-making. In this context, CMS’ approach to implementation of the Medicare Drug Price Negotiation Program represents a missed opportunity.
Patients generally have a different – but critical – view of their disease, one that goes beyond the narrowly focused outcomes selected for clinical trials or those commonly assessed in real-world research. It’s crucial that those voices help guide our conversations about health policy, especially when those policies are designed to reshape the health care system.
So it’s disappointing the recent CMS guidance for the implementation of the Medicare Drug Price Negotiation Program, which uses a number of factors including relative clinical benefit of a drug to determine a government-set price for certain drugs, does not outline a clear plan to engage the patient community or prioritize research studies that were guided by meaningful patient engagement.
To be fair, CMS revised its original guidance to include patient-focused “listening sessions,” one-way, one-time opportunities for patients and the public to give input. But that is little more than going through the motions; feigned listening is worse than not listening at all. Patients need to be a part of a real back-and-forth, a true dialogue, and need to be able to see in a transparent way how their input will be considered in policy decision-making. CMS has yet to release any substantial information on these “listening sessions” and most importantly has not given any indication how they meaningfully incorporate feedback into their decision-making.
In pushing ahead with this limited approach to patient engagement, CMS is ignoring models already available to them on the inclusion of patients in research. The Patient-Centered Outcomes Research Institute (PCORI) has already created a playbook for engaging patients. An analysis of 126 studies highlights the many contributions patient engagement brings to PCORI-funded comparative effectiveness research, including determining appropriate outcomes and identifying appropriate comparators.
PCORI need not be the only source of inspiration. Other models for continuous patient engagement have been validated and widely applied, too, including the Mullins’ 10-Step Framework for continuous patient engagement. The 10-Step Framework provides a roadmap for how to meaningfully engage patients and other stakeholders from topic inception to disseminating final products to improve health services, health literacy, and health outcomes.
From our experience conducting patient-centered outcomes research, we highlight for CMS officials and staff, and health policymakers, three tangible benefits of applying patient experiences to strengthen their approach to the implementation of the Drug Price Negotiation Program:
1. Patients have first-hand knowledge about living with health conditions that clinicians or other experts do not have. Patients are an essential resource to share their experiences with alternative pharmaceutical treatment, including impacts on the physical effects of medications and therapies, and the emotional, social, and financial implications of therapy. This input can help CMS weigh the benefits and drawbacks of various therapeutic alternatives and choose those that align with patients' preferences and needs. Failing to include patients in research, especially when “differences exist between physicians’ judgement and patient preferences,” could lead to an inaccurate decision about the value of potential new treatments.
2. Patient input can help identify key parameters such as comparators, outcomes, and subgroups relevant to real-world decision-making. Patients’ unique insights into factors include what the treatment does, accessibility of treatments, side effects, and the complexity of taking the medication. For example, individuals with chronic obstructive pulmonary disease (COPD) report placing high value on improvements in fatigue and sleep quality and the frequency a treatment is delivered. Among individuals with Alzheimer's disease, family and caregiver costs and well-being and the ability to communicate and convey thoughts was considered high value. These are just a couple of examples of how patients value factors that are not usually represented in clinical trials or value assessment and may not be readily front of mind for government officials. As such, without these considerations, treatments may not always be equal substitutes in the eyes of individual patients even if the clinical effects are the same on average. This critical information for CMS will not be gleaned through real-world data analyses alone.
3. Patients can highlight unmet needs in current treatment options and identify areas where new or improved therapeutic alternatives are valued or needed. This information can help CMS prioritize certain treatments and evidence needs. CMS should consider the needs of relevant populations for the selected drug to capture patient experience diversity. For example, while stroke patients identified avoidance of major cardiovascular events as an outcome of interest, as one would expect, they also identified and prioritized "home time", time spent alive and outside a hospital.
As the Drug Price Negotiation Program is implemented, we urge CMS to hit “reset” on patient engagement and establish clear policy and procedures to meaningfully engage patients, families, and other stakeholders as part of the Medicare Drug Price Negotiation program.
To include patient perspectives in the new Medicare Drug Price Negotiation Program, PCORI offers a proven approach. CMS’ guidance lays out timelines that will be difficult to successfully implement. With that in mind, the PATIENTS Program at the University of Maryland and Applied Patient Experience are also currently engaging graduates from the PATIENTS Professors Academy to identify opportunities to improve their proposed process to ensure patient perspectives are incorporated.
We believe CMS should rely on PCORI and other existing models as objective, science-based resources to advance patient-centeredness, in its methods and in collecting and evaluating available evidence. Adopting PCORI's methodology allows CMS to benefit from more than 10 years and $3 billion in research by utilizing a framework that stakeholders have already vetted. To avoid early criticism and to build trust, CMS should explicitly acknowledge this challenge and commit to transparency in its decision-making by setting a high bar for patient-centered standards and processes.
Joe Vandigo, MBA, PhD is the Principal Scientist and Director of Value at Applied Patient Experience, LLC. Hillary Edwards, MPH is the director of methodological research and evaluation for The PATIENTS Program at the University of Maryland School of Pharmacy.
