Through the Patient Perspective: Collaborating to Improve Research and Development

Applied Clinical Trials

At the October Metrics in Patient Centered Drug Development conference hosted by DIA, many of these key constituents gathered to talk about how to implement and measure the impact of patient input.

In the October issue of Applied Clinical Trials, editor-in-chief Lisa Henderson reported on the increasing role of collaboration in clinical trials as well as in the entire clinical development process. An ever-expanding area of collaboration is occurring between patient organizations, industry, regulatory, and governmental organizations to ensure that new drugs developed both represent value to the patients they aim to treat, and that the timeframe for providing new innovations to patients is accelerated. Leading the way are patient organizations who have formed sophisticated programs with the express purpose of integrating the patient’s voice, preferences, and interests into the drug development process. At the October Metrics in Patient Centered Drug Development conference hosted by DIA, many of these key constituents gathered to talk about how to implement and measure the impact of patient input. A highlight of the DIA meeting was learning about specific initiatives that are ongoing in the cancer, Parkinson’s, and rare disease patient communities to bolster this collaborative effort, as well as understanding what, from the patient perspective, constitutes meaningful engagement.

Engagement with Patient Communities

“Return on engagement” (ROE) metrics have been discussed primarily from the industry side to answer the question: what has a company gained from patient input into the drug development process? Most companies understand the why and that their success depends on putting the patient at the center of all they do. However, the how, i.e.-the way this is done, what works best, and how you capture value for purposes of refining and replicating best practice strategies is a main driver for the increasing emphasis on measuring impact. Sample metrics include reduction in the number of protocol amendments, faster accrual to trial, or increased retention rate due to patient satisfaction with the trial process.

Patients and patient organizations are invested in making sure new treatment innovations are both available and offer value aligned with patient needs, and know their voice is integral to the process. For that reason, they have fought for years to have a ‘seat at the table’ to ensure their perspective on unmet medical needs and the impact of treatments on quality of life are central to the drug development process. Patient organizations have taken it upon themselves to train patients on how to have input into the science of drug development. AIDS Treatment Action Group (A TAG) pioneered this in the 1980s and breast cancer took those learnings to create the National Breast Cancer Coalition’s (NBCC) “Project Lead,” a science training program for activists that significantly impacted breast cancer research and public policy. The result of A TAG and NBCC's work changed the way scientists, government agencies and industry design and implement research and programs.

Patient Voice Input Gathers Momentum

As demand has increased, so has the intricacy of these efforts, with the European Patient’s Academy (EUPATI) having trained 96 patient experts on medicines development, clinical trials, medicines regulations, and health technology assessment. Additionally, the National Health Council in the US has brought together key stakeholders to outline key elements of what an FDA guidance on patient engagement might consider. Finally, organizations across multiple disease areas have built registries to systematically track patient values and unmet needs.

The range of activities across patient organizations are diverse and progressive. For example, Ronald Bartek, Founder and President of the Friedreich’s Ataxia Research Association (FARA) identifies basic science resources that organizations are creating alone or in collaboration with industry, including gathering natural history studies and developing and providing translational tools (cell & animal models, biorepositories). Organizations are building patient registries to report on real world evidence and outcomes. The Cancer Support Community (CSC), under the leadership of Senior Vice President Joanne Buzaglo, PhD, has developed the Cancer Experience Registry to document insights from patients across the cancer experience spectrum and illuminate key areas that impact patients’ lives. Key areas captured in the registry include cancer-related distress, aspects of quality of life, information on treatment decision-making and planning, side effects and symptom management, and experiences in clinical trials. CSC uses the data to shape responsive programs and policy positions, as well as to share with researchers and industry partners to improve treatments and care.

New Potential for Collaboration: Examples

Patient input into clinical trial design and protocols offers opportunity for sponsors to more closely align R&D activities with patient preferences. Two program examples illustrate new possibilities for collaboration.

The Parkinson’s Foundation has trained a cadre of Parkinson's Advocates in Research (PAIR). Led by Karlin Schroeder, Director of Community Engagement, Research Advocates are people with Parkinson’s and their care partners who have been trained in both the clinical research process and how to collaborate with researchers in industry, academic centers, and government. Research Advocates prioritize research to draw attention to unmet needs, inform studies to improve protocols, influence stakeholders to facilitate patient engagement with regulatory agencies and ultimately, accelerate trial accrual. Examples of input to trial development include selection and prioritization of secondary outcomes for people living with Parkinson’s as well as altering patient reported outcome (PRO) tools to more accurately capture the Parkinson’s disease experience. The work of the Parkinson’s Foundation has ensured that research into unmet needs, including gastrointestinal dysfunction and cognitive impairment, is moving forward.

LUNGevity’s Patient Focused Research Center (Patient FoRCE) initiative intends to change the paradigm in lung cancer from assumptions being made about patient wishes to evidence-based conclusions about what patients value. President Andrea Ferris and her team create uniquely patient-focused projects that involve multiple stakeholders across five broad categories: deriving patient preferences about treatments for lung cancer, implementing biomarker testing to ensure patients benefit from precision medicine, understanding gaps in information patients receive throughout their journey, executing effective lung cancer screening, and identifying misperceptions of patient attitudes by health care providers. Through collaboration with Johns Hopkins School of Public Health, they created Project Transform to apply novel methods (e.g. discrete choice experiments) to enhance understanding of how patients perceive short and long term side effects and quality of life issues, associated with their cancer treatment. One study conclusion of a pilot study was that lung cancer survivors value progression free survival as the most important component in their treatment choice.

It is easy to see from these examples how collaboration with patients and patient advocates can enhance research and development efforts. What constitutes meaningful engagement and ROE for patients can be summarized as measuring how patients’ voices are heard and incorporated into research, policy, and health care practice behavior. As Ron Bartek puts it, these “more shots at the goal” translate into increased options for patients and faster recruitment to clinical trials with potential for bringing innovations to market more quickly. These are metrics we can all get behind.


Ellen Coleman, MSSA, MPH, is Senior Vice President with MK&A. She can be reached at