In 2015, two issues related to medicine could be relied on to generate headlines: drug pricing and the proliferation of new value frameworks that claimed to define the value and even the price of drugs in seemingly easy-to-understand ways. In none of the high-profile skirmishes on pricing or frameworks was the voice or perspective of patients and patient groups very much in evidence. But that is beginning to change, in an evolution of a broader shift in the role that patients are playing in the research and development (R&D) enterprise.

A New Culture of Engagement

Patients and patient organizations are becoming ever more important drivers of medical research and development. Players, including academic institutions, biopharma companies, and regulators, have “gotten the memo” about the value to be derived from seeking the input of patients—qualitative and quantitative—from the early stages of research through the evaluation and approval of new treatments. In a recent article published in Science Translational Medicine, we documented the progression of patients over the last century from passive recipients of medical care to active partners in defining and prioritizing research strategy for their diseases.

Much uncertainty remains about how best to collect and utilize patient data and perspective in ways that satisfy the needs of all the parties involved. (FasterCures is working to help reduce that uncertainty through its Patients Count program.) But many—including influential entities such as the Patient-Centered Outcomes Research Institute (PCORI) and the Food and Drug Administration (FDA)—have made patient engagement part of their culture in an effort to fundamentally improve the R&D process. Health Affairs recently devoted an entire issue to “Patients’ and Consumers’ Use of Evidence” to improve research, practice, and policy; Editor-in-Chief Alan Weil referred to patient engagement as an “imperative.”

Patient organizations have been creating models and building infrastructure that are bringing fresh and critically important information to the table and adding value to PCORI’s and FDA’s work. Organizations such as the Accelerated Cure Project for Multiple Sclerosis and the Phelan-McDermid Syndrome Foundation are among PCORI’s 20 Patient-Powered Research Networks, operated and governed by groups of patients and their partners.

These networks are contributing a unique view of the real-world experience of patients’ diseases and treatments to the large outcomes research infrastructure PCORI is building. Parent Project Muscular Dystrophy paved a new path with the FDA when it recently proposed a draft guidance to the agency to help industry better understand Duchenne muscular dystrophy and design clinical trials in the disease; the agency revised and adopted it. The FDA is developing new ways of working more closely with patients to inform the regulatory process as part of its patient-focused product development initiatives across the agency.

Making the Value Proposition to Payers

One important health care stakeholder that has not yet “gotten the memo” is the payer community, which is the critical gatekeeper of access to treatments for patients and is increasingly questioning the value of existing and new treatments. They may not yet have seen a compelling value proposition for how engaging with patients and patient groups could help them achieve their mission of providing access to valuable health care products and services while controlling costs for their clients (including, in the case of the Centers for Medicare and Medicaid Services [CMS], U.S. taxpayers). In a series of workshops, we have heard from even those payers who are actively interested in learning more about patient preferences, experiences, and needs that they lacked systematic processes for soliciting and using that input.

Proliferating efforts to define value, such as those put forth by the Institute for Clinical and Economic Review, the American Society of Clinical Oncology, the Memorial Sloan Kettering Cancer Center, and the National Comprehensive Cancer Network, have as a stated goal to inform decision-making by patients and their doctors, and payers are using them in negotiations with medical product developers. But none incorporates significant input—qualitative or quantitative—from patients into their definitions of and models for determining value. In response, FasterCures and Avalere initiated a collaboration in 2015 to develop a patient-perspective value framework through a rigorous process and methodology that includes patient input as well as that of all other stakeholders.

Patient Groups Are Seeking to Influence Policies

While the same momentum toward patient-centricity may not yet exist in the value and coverage debate, patient-driven research and advocacy organizations are already experimenting in a variety of ways with how they can inform and influence the dialogue. Historically, patient groups have played a significant role in helping patients—individually and collectively—get coverage for specific treatments, advocated for changes in state-level Medicaid policies, and more. Now, we are seeing a new level of engagement taking root.

We looked across the 80 venture philanthropies—foundations taking strategic and entrepreneurial approaches with their research funding—that are active participants in FasterCures’ TRAIN (The Research Acceleration and Innovation Network) and discovered a surprising amount of activity that seemed to fall outside their traditional or originally defined roles. More than 20 percent were engaged in some kind of activity related to defining value and setting coverage policies.

Here are a few examples of the variety of ways patient groups are seeking to influence this dialogue.

Eight TRAIN member organizations are engaged in projects or initiatives with the intention of reducing cost, generating evidence, or demonstrating the value of treatments. The COPD Foundation’s PRAXIS program, for example, works to identify and eliminate gaps in care for the patient population it knows better than anyone, by creating a learning environment to help identify strategies for reducing hospital readmissions and educate providers and patients. It was created in response to needs identified by CMS’ Hospital Readmissions Reduction Program and other programs designed to tie payment to performance for chronic obstructive pulmonary disease (COPD).

The Focused Ultrasound Foundation consults with researchers it funds to ensure that studies are designed not only to demonstrate safety and efficacy but also to meet insurers’ requirements for superior long-term benefits, quality of life, and cost-effectiveness. The foundation is also designing and implementing patient registries to track long-term treatment results that insurance carriers can use to evaluate the efficacy of focused ultrasound treatments. And The Juvenile Diabetes Research Foundation (JDRF) funded an early and large clinical trial to establish the efficacy of Continuous Glucose Monitoring to satisfy payers and help ensure patients would have access to this life-changing technology.

Seven TRAIN organizations are engaged in advocacy efforts to encourage coverage for targeted and innovative treatments or diagnostics and to protest access restrictions. Myeloma UK, for example, was extensively engaged in the public dialogue in the United Kingdom around the National Institute for Health and Care Excellence’s proposals about shifting to a value-based pricing regime. It leveraged its role as a trusted intermediary representing patients’ interests to host a Parliamentary debate, comment on proposals, convene a stakeholder roundtable, and produce a position paper. As the United States moves toward value-based payment models, this example could be particularly informative.

Six foundations have conducted studies and surveys aimed at illuminating the burden of disease on patients, their finances, and society. The National Psoriasis Foundation funded a white paper based on its extensive survey data about patients’ experiences with treatments and under-treatment, intended to help payers understand the serious medical consequences of non- and under-treatment.

Five patient groups are involved in convenings by their own organizations to understand how they can play a role in influencing coverage decisions and to bring patients’ perspectives to other stakeholders’ discussions about definition of value. In 2014, the Michael J. Fox Foundation for Parkinson’s Research convened a roundtable of payers, physician researchers, and policy experts to discuss the state of Parkinson’s disease (PD) research and challenges to the patient population. Out of this meeting came a survey and study of patient perspectives on the burden of disease, starting with the unique phenomenon of “off-time” experienced by PD patients. A resulting white paper and journal article are being shared with payers.

One organization, the National Multiple Sclerosis Society, has produced a set of principles outlining components of high-quality care, including affordability, which will inform its activities going forward. And another, the American Heart Association, produced its own value framework in 2014 with the American College of Cardiology, which seems to have escaped attention in the current focus on oncology and other therapeutic areas. They bluntly stated that “the time has come to include cost-effectiveness/value assessments and recommendations in practice guidelines and performance measures.”

Organizations like these and others are increasingly aware that their work to stimulate more scientific research in their disease areas and to shepherd promising discoveries through the development process will be for naught if patients don’t have access to treatments. They are increasingly positioning themselves as key opinion leaders with access to capital, research infrastructure, and research participants interested in answering the same questions as other stakeholders: Do these treatments work, for whom, under what conditions, and what value are they generating for patients and for society?

Where Do We Go from Here?

There is currently no clear roadmap for engagement between patient groups and payers, but there is strong interest in and precedent for creating one.

Payers need to see a clear value proposition compelling them to invest the time and energy needed to build relationships with patient organizations. Elements of such a value proposition might include:

  • Patient-relevant outcomes drive value; payers need to understand how care is impacting patients’ functioning in the real world if they are going to pay for value.
  • Patient organizations have access to unique sources of real-world evidence in the form of patient registries, biorepositories, social networks, and mobile health initiatives and are often the most trusted intermediaries for and stewards of patients’ data.

Many patient groups still aren’t aware that there is a role for them in the value and coverage arena and how to go about plugging in; existing activity is fragmented. They need to understand what they can bring to the table, and how they can bring it.

Foundation funders of research can provide data to shape product design and utilization, resulting in better alignment between regulatory and post-market evidentiary needs. The Cystic Fibrosis Foundation, for example, has used the extensive patient data contained in its 60-year-old patient registry to support the development and approval of new treatments for cystic fibrosis, and is now engaging with payers to determine how those data can be used in coverage decisions. Foundations can promote and support a systems-based approach to assessing value. And they can help clear waste in health care by helping to identify where there might be inefficient or ineffective care in their disease area to save money and make room for more innovative treatments.

But many of these groups need to “up their game” to be more effective partners. Not all organizations are as sophisticated in their approaches as some of those discussed here; there needs to be more capacity-building in the sector. Even for the sophisticated groups, they can be stretched thin across the full continuum of research and development, from funding the earliest discovery to now having to figure out whether and how they should help make the case for the value of treatments to their patients. Patient organizations need to better understand the payer landscape and payers’ evidentiary needs. And they need to build relationships with developers and payers early, before there’s an “ask.”

Following the lead of the clinical development community, payers and patient groups need to build a common framework for soliciting and using patient input. Mark Skinner, president of the World Federation of Hemophilia, summed up the task at hand succinctly at a FasterCures workshop in 2015: “Patients, clinicians, developers, and payers need to get together to agree on outcomes that represent value and what evidence is required to demonstrate it.” It’s that simple.