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When it comes to medical research, it’s time to stop, listen, and learn from patients

Successful public health campaigns often include a pithy slogan to help us remember the action we’re supposed to take. We teach kids to stop, drop, and roll if ever in a fire. Australia successfully battled skin cancer by asking its citizens to slip, slop, slap (slip on a shirt, slop on sunscreen, and slap on a hat). American teenagers were targeted with “click it or ticket” to remind them to wear seatbelts. When it comes to medical research, maybe a catchy slogan would remind key players to stop, listen, and learn from patients.

Industry thought leader Leonard Kish once wrote “if patient engagement were a drug, it would be the blockbuster of the century and malpractice not to use it.” So how is the system (e.g., federal agencies, nonprofits, and the commercial life sciences sector) doing vis-à-vis patient engagement, and what could be gained from a “stop, listen, and learn” approach?

There is intense focus on patient engagement and the evolving science of patient input among federal agencies. From bench to bedside, there is widespread discussion about how patient input will enhance the process of conducting and evaluating research, and how to determine value to the patient. Perhaps the most visible new project related to how we engage patients is the “All of Us” Research Program. The National Institutes of Health (NIH) launched the campaign to gather data from one million people to accelerate research and ultimately improve health. But collecting data is just one component of the initiative. All of Us has potential to offer up a new playbook on how to reframe the equation so that patients are partners in their care, rather than merely participants. The NIH National Center for Advancing Translational Sciences recently launched a toolkit to aid patient organizations in their quest to provide high-quality data about their patient populations.

FDA meetings gather patient input

Over at the Food and Drug Administration (FDA), there is much activity related to collecting and evaluating patient input and ultimately incorporating it into regulatory decisions. Regulatory transformation may create trepidation in the market because pharmaceutical and biotech companies want to understand which direction to chart their course.

The FDA’s Voice of the Patient meeting series – which began under the recently reauthorized Prescription Drug User Fee Act (PDUFA) – gathers information from patients about their conditions and available therapies. PDUFA was enacted in 1992 and must be reauthorized every five years. The meeting series began with PDUFA V in 2012.

Each of the 20 or so public meetings focused on a specific disease. Many nonprofits are conducting their own meetings while actively working with the FDA to learn from them. Resources and collaboration with external experts and partners can be key for the agency to move forward with the various deliverables that flowed out of the PDUFA reauthorization and the 21st Century Cures Act.

The FDA-NIH Joint Leadership Council could be one avenue to create a horizontal look at the scientific and grant-making infrastructure at NIH in tandem with the FDA’s regulatory science mandate around patient input (see the FasterCures Rx for Innovation report).

“Impatient patients” push for better engagement

Many nonprofit disease foundations have been banging the drum on patient engagement/centricity for a good long while. Many of these foundations have to fundraise from zero every year. Many were started by “impatient patients,” as John Walsh, a founder of the Alpha-One Foundation and COPD Foundation, used to say, and they all have unique structures and even missions. For example, a group might be created to map the scientific needs of a disease and provide money to the field as a research and development (R&D) accelerator. While becoming a data hub on the science of patient input is often a natural progression, it is important for the group and the ecosystem to understand that the hub might not have resources.

Nonprofits are increasingly taking on a wide range of roles, from policy and reimbursement experts to care delivery and research roles. Many commercial life science companies have created their own infrastructures and mechanisms to help ensure they are engaging with patients to ensure their voices are heard – in both the discovery and dissemination process of therapy development. This has created further demands on nonprofits as they navigate numerous gatherings and consultations being hosted by government and industry.

Platforms, business planning, and specific tools can go a long way in saving time and resources, allowing nonprofits to focus more attention on the specific needs of their patient populations. Government and industry groups should consider the stop, learn, and listen rule, and be open to learning in a different way. Each organization needs to consider how best to convey information about patient populations within various constraints e.g., time, budget, and ability. Capacity building in the nonprofit space is likely a must do if we expect to realize the full potential of patient input. Given that this methodology is still being built, each organization will need to reflect on where it is in the continuum of understanding the science of patient input from each patient population. Some organizations may need help in data gathering, some in interpretation of data, others in patient outreach etc.

Industry might see the light, but it doesn’t yet feel the heat

The pharmaceutical, biotechnology, and commercial life sciences sector has seen the light when it comes to the science of patient input. But in the words of Dr. Freda Lewis-Hall, chief medical officer at Pfizer, you don’t change when you see the light, you change when you feel the heat. That is where these three sectors are often intricately linked. The value proposition of developing products with consumer input is clear, and is a longstanding practice in most other industries.i But it is one thing to develop the next big app or phone based on consumer preferences, and quite another to develop a medical product that runs through the FDA’s regulator y review process.

Many companies are doing deep and thoughtful work to stop, listen, and learn from patient communities. But they also likely need regulatory certainty from FDA as it provides guidance on what is needed. This could provide some light as well as a bit of heat.

FDA Commissioner Scott Gottlieb recently announced that an Office of Patient Affairs will be launched soon. FDA solicited input from the community on this concept early this summer. Some groups say they see value in centralization and harmonization, but have concerns that this work could be sidelined or isolated, and disconnected from the overall workflow.

From my perspective, as long as companies are listening and learning from their patients, nonprofits are continuing to pioneer new approaches, and regulatory agencies are mapping out what is needed to move forward, we can expect positive changes around patient-centered care.

i On the path to a science of patient input, M.Anderson & K.McCleary, Science Translational Medicine 27 Apr 2016: Vol. 8, Issue 336, pp. 336ps11 DOI: 10.1126/scitranslmed.aaf6730 L. Kish, The blockbuster drug of the century: An engaged patient. HL7 Health Standards 28 August 2012. www.hl7standards.com/blog/2012/08/28/drug-of-the-century.

Author bio

Margaret is a managing director engaging across the federal health, nonprofit, and life sciences sectors where she is focused on advancing treatments and interventions for patients, as well as helping to improve the outcomes and efficiency of research and delivery systems. Prior to Deloitte, Margaret advocated for cross-sector collaboration, cultivated a culture of innovation, and engaged patients as partners while serving as executive director of FasterCures, a Washington DC-based center of the Milken Institute. She has worked on biomedical and public health policy serving previously at the Academy for Educational Development, as program director at the Society for Women’s Health Research, and as a health science analyst at the American Public Health Association.


Margaret has served on national boards and committees including the NIH National Center for Advancing Translational Sciences Advisory Council and Cures Acceleration Network Review Board, and National Health Council. She currently serves on the boards of ACT for the NIH, Asthma & Allergy Foundation, and Melanoma Research Alliance. Anderson holds a bachelor’s degree from the University of Maryland and a master’s degree in science, technology & public policy from George Washington University.