Learning to Listen: Voice of the Patient Grows Louder in Product Development

Days before Christmas 2017, a nationwide call went out requesting nominations of patients and caregivers to join a new public/private-sponsored Patient Engagement Collaborative.

Among the promised duties for this yet-to-be-filled body of 16 patient community representatives: Propose new collaboration models in which patients and patient advocates are partners in healthcare product development and U.S. Food and Drug Administration product reviews.

The collaborative’s creation, a little more than a year after the 21st Century Cures Act became law in the United States, is the latest step in an accelerating patient-centered drug and device development movement. This worldwide effort is being fueled by ever-improving consumer-controlled social and health data technology, advancements in personal genetic understanding, and increased healthcare cost-sharing in some quarters. Patient-driven changes are systematically beginning to inject more than token patient participation and viewpoints into all stages of drug and device development and slowly, but methodically, chipping away at the clinically isolated way drugs and medical devices are developed.

Patients first?
Many healthcare leaders love to claim they put patients first in developing new medicines, therapies, and medical devices. But the reality of product development in its current state is far from ideal, particularly in the United States. Too many scientific research teams remain in ivory towers, inwardly focused on drug development processes and procedures rather than on letting patients guide them to what’s important.

Patients are less likely to be consulted on specific drug research and development in the United States than in Europe, where patient input is regularly solicited throughout the medicine’s lifecycle through the European Medicines Agency’s more established patient input system.

The new Patient Engagement Collaborative, created through a partnership between the FDA and the Clinical Trials Transformation Initiative, is being modeled after the EMA Patients’ and Consumers’ Working Party, a decade-old exchange platform for EMA, patients, and consumers. The FDA and EMA last year created a workgroup on patient engagement called the FDA/EMA Patient Engagement Cluster so each agency could glean important learnings on the topic from one another.

The FDA and drug and device developers who need the agency’s OK to reach market are moving rapidly to increase their interaction with patients. Under the 2016 Cures Act (Sec. 3001), the FDA is required to report any patient experience data that was used to support a drug’s approval and to publicly provide aggregate reports on agency use of this data at five-year intervals. This means those who want to have new drugs approved by the agency need to provide more of this patient-driven data. The agency defines patient experience data as data collected by any persons — including patients, family members, patient caregivers, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers — that is intended to provide information about patients’ experiences with a disease or condition.

The term specifically includes data regarding the impact of the disease or condition, or a related therapy, on patients’ lives, and patient preferences with respect to treatment of the disease or condition. The agency has said it will start releasing more detailed guidance on patient-focused drug development by mid-2018.

Some of the guidance will be surely driven by insights gleaned from a just-completed four-year Patient-Focused Drug Development Initiative that involved the FDA holding 24 patient-focused drug development meetings. From those meetings, each involving a different disease area, the agency developed a series of Voice of the Patient reports that systematically recorded patient input on their condition and how it affects their lives.

Other guidance is likely to derive insights from private, patient-focused disease advocacy groups like the Parkinson’s Foundation, which has used technology and its social and financial reach to extend patient influence and partner with others to more directly conduct clinical research and product development.

New patient-centered research
In November, the foundation awarded $60,000 to help fund six innovative, patient-centered research projects that help patient-scientist teams develop innovative models of patient engagement. The funding is a next step in the foundation’s innovative and widely praised Parkinson’s Advocates in Research program. PAIR has created a nationwide network of 280 so-called research advocates who are trained by leading experts from the field in the science of Parkinson’s and the development of new treatments. Each one lives with Parkinson’s or is a patient caregiver. They individually work as primary partners in research alongside scientists, industry, and government to drive development of better patient-focused treatments.

Early successes by patient-driven programs like PAIR have led other foundations and associations, along with pharmaceutical and biotechnology companies, to form a growing number of Patient Advisory Boards that are becoming more involved in the drug and device development lifecycle. PABs and other efforts are increasingly broadening the traditional study-participant role to that of an active partner throughout the process.

But PABs and similar structures by themselves are not enough for true patient-centered and patient-focused drug and device development to happen on a systematic basis. Numerous researchers have found that this requires cultural and structural changes at organizational and systemwide levels.

Models are emerging for this to occur. One oft-cited model for patient-focused drug development, by Eleanor M. Perfetto, Ph.D., M.S., and her colleagues at the University of Maryland lays out where and how patients have a role from pre-discovery to post-approval surveillance of a drug or therapy. For the model to yield tangible benefits, they say, all healthcare stakeholders — the patient community, industry, academic researchers, government, health systems, providers, and payers — must collaborate.

The power of data analytics
Leading health organizations, including the Institute of Medicine, maintain that such collaboration should be powered by a self-regenerating Learning Health System “… in which progress in science, informatics, and care culture align to generate new knowledge as an ongoing, natural byproduct of the care experience and seamlessly refine and deliver best practices for continuous improvement in health and healthcare.”

Put more simply: Take the power of data and analytics learned from every patient and feed the knowledge of what’s working back to clinicians, drug, device and therapy product developers, public health professionals, patients, and others to create cycles of continuous improvement in healthcare delivery.

Nearly 120 leading healthcare organizations that have joined the Learning Health Community endorse the approach of placing patients as leading partners in finding and delivering better healthcare solutions. There were fewer than 70 such organizations three years ago.

In the past, industry has inconsistently considered the views of patients in developing new drugs, devices, and therapy products. The trend toward more inclusion of these viewpoints is a gratifying development that will enhance the development of these products and improve patient outcomes.

Author Details

Katharine Chorlton
Katharine Chorlton is a Director in the Global Regulatory Affairs Department providing strategic regulatory advice and support for regulatory submissions. She has over twenty years experience in the pharmaceutical/biotech industry, the majority of which have been in regulatory affairs. Her key experiences are within Immunology, Inflammation, Oncology, Respiratory, Dermatology and Women’s Health therapeutic areas. Katharine has an MSc in Regulatory Affairs and a BSc(Hons) in Pharmacology from Sheffield University.
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