When National Health Council (NHC) official Marc Boutin, J.D., speaks to an audience of pharmaceutical company executives about patient-focused drug development, he underscores the topic’s importance to the life sciences industry by stating, “Look at the person sitting on your right. Now, look at the person sitting on your left. One of them likely will be unemployed in five years.”
The reason, Boutin said, is that the individual’s company will have failed to effectively incorporate the patients’ perspectives at multiple points along the drug development continuum, which he defined as encompassing R&D portfolio selection and prioritization; identification of research questions, outcomes, and comparators; clinical trial design and recruitment strategy; regulatory review; and commercialization and postmarket surveillance. “To understand patients, you must engage them,” said Boutin, executive VP and CEO of NHC, whose member organizations include nonprofit patient advocacy groups as well as life sciences and insurance companies. “When people with chronic conditions are involved in a meaningful way through the drug development continuum, we greatly increase the probability of producing the kinds of drugs patients want, need, and use,” he added.
David Verbraska, VP, worldwide public affairs and policy at Pfizer, agreed. “The patient perspective brings a universal truth to what we’re doing. Patients help define the unmet medical needs, value of the science, and the patient-reported outcomes and surrogate or direct clinical trial endpoints that should be used in a clinical trial,” he said. “By being patient-centric and adding transparency and interaction all along the R&D and market life cycle, patients help us achieve the best public health outcomes and avoid the worst-case scenario,” added Verbraska. The scenario: a drug that is approved by the FDA but does not meet patients’ needs. Through patient engagement throughout the R&D process, those needs would have been identified and addressed, he said.
Engaging patients in clinical trial design could help improve the study’s efficiency by boosting patient recruitment, protocol compliance, and retention. It also could help insure that the study generates the type of information that will be most important to patients. For example, although Parkinson’s disease impairs mobility, many patients have reported that they are more bothered by the impaired sleep patterns and depression that are associated with the neurological disorder. For these patients, a clinical trial of a new drug for Parkinson’s disease should evaluate the agent’s impact on these symptoms as well as mobility.
The Wall Street Journal recently reported on the role of patients in clinical trial design. The Sept. 29, 2014 article, “Design Power: Patients Play Researchers in Drug Trials,” described a new clinical trial for a prostate cancer drug. Based on patients’ suggestions, the researchers designed the trial to track such measures as the number of days when the patients felt well enough to go to work.
Until recently, the term “patient engagement” was usually used to describe programs to empower patients with the knowledge and tools to take responsibility for their health. Today, “patient engagement” is often used interchangeably with Patient-Focused Drug Development (PFDD), the name of the FDA’s five-year study to systematically obtain patients’ perspectives. “We want to learn about the clinical context of each disease from the patients’ point of view and experiences,” said Theresa Mullin, Ph.D., director of the Office of Strategic Programs in the FDA’s Center for Drug Evaluation and Research (CDER).
At these public meetings, each of which targets a chronic, symptomatic disease, Dr. Mullin and other FDA officials also ask patients to assess their available treatment options and the therapeutic benefits that matter most to them. By the end of 2017, the FDA will have organized 20 patient meetings, each on a specific disease. Each meeting results in a “Voice of the Patient” report posted on the FDA’s website. The report captures the perspectives of patients who participated in the meeting either in-person or by webcast. Also summarized in the report are comments submitted during the 60-day period following each meeting.
The FDA’s PFDD initiative has spurred several pharmaceutical companies to broaden the scope of their patient engagement activities. “In the past, the industry’s approach to patient engagement was primarily anecdotal and ad hoc, with a project here and there,” said Verbraska. “What is very different now is that Pfizer and other leading companies are more formal and systematic in soliciting the patient’s voice, and we’ve gone from just listening to being more action-oriented in what we do, by taking what we’ve learned from patients and embedding that information into our decision-making processes.”
SEEKING THE PATIENT’S VOICE
Pfizer is capturing the patient’s perspective in a toolkit that will be broadly distributed to internal global project teams in the company’s core therapeutics areas. The toolkit for each disease area targeted by Pfizer describes the patient’s journey, including the impact of the disorder on such quality-of-life measures as relationships. The toolkit, which is now under evaluation in a pilot study, also suggests tangible ways that project teams can engage with patients to learn more about their needs. “We realize that systematically incorporating the patients’ perspectives in decision making requires process and cultural changes, particularly in our R&D community,” Verbraska said, “and the scientists are the most energized about making that happen. Our goal is to build an ecosystem in which the patient is at the center.” To help build the ecosystem, Pfizer leaders have established a “community of practice,” a group of colleagues whom Verbraska described as evangelists for patient engagement within the company.
For pharmaceutical companies such as Pfizer, there are several potential barriers to fully engaging patients in the drug development continuum, especially in portfolio selection and clinical trial design. The most imposing barrier is the industry’s uncertainty about the impact of the FDA’s restrictions on companies’ communications about drugs that have not yet received marketing authorization from the agency. “Companies are understandably reluctant to discuss an unapproved drug for fear of facing enforcement action and fines by the FDA or the Federal Trade Commission,” said Boutin. “How can we insure meaningful patient engagement at the front end of drug development while mitigating the risks of engagement for companies?”
That question will be on the agenda of an early 2015 invitation-only meeting of senior leaders of pharmaceutical companies, the FDA, patient advocacy organizations, insurers, and providers. NHC is organizing the meeting, which Boutin described as a dialogue of stakeholders that will begin to lay the foundation for a practical framework on how patients and their perspectives should be continuously integrated throughout the drug development continuum. Boutin said he hopes the stakeholders will develop a shared definition of patient engagement that embraces the concept of incorporating the patients’ perspectives in the drug development continuum. Kenneth Kaitin, Ph.D., director of the Tufts University Center for the Study of Drug Development, has been quoted as saying, “There is agreement about how important patient-centered drug development is for pharma and biotech, but clearly no agreement on the definition of the term.” Also on the agenda is a discussion of promising patient-engagement methods. In addition, participants will discuss barriers that hinder meaningful patient engagement and potential ways to eliminate them. The results of the dialogue will be subsequently communicated at an open-to-the-public event for a larger audience of stakeholders.
Pharmaceutical companies should not wait for the results of these meetings to broaden their patient-engagement activities. They should consider:
Dr. Mullin added that companies “could play an important role in collaborating with patient groups and researchers in follow-up work to develop clinical outcome assessment tools or patient-reported outcome measures for clinical trials that will better capture the patients’ perspectives.”
NHC has developed a tool that could help companies collect and organize data about patients’ perspectives on their diseases, the quality of their lives, and treatment options. The “Patient Perspective and Disease Impact Stratification Tool” was originally designed to assist patient advocacy in preparation for the FDA’s PFDD meetings. However, the tool will be useful to anyone who wants to understand the full scope of the patients’ experiences with diseases, said Boutin.
In the past two years, Boutin said he has witnessed a dramatic shift in how individual companies and the FDA view patient engagement. “My sense is that we’re in a very exciting place.”