Drive for patient preferences to improve healthcare decision making
Historically, R&D has been driven by the bottom line: profits and hard patient outcomes. However, a new movement in the device and pharmaceutical research industry is focusing on the patient experience.
Patients do not make decisions based on whether a particular medication is profitable for the company that develops and markets it. Patients may prefer a drug that doesn’t have the spectacular results of another medication used for the same purpose, but that has lower associated risks or fewer side effects. Or, conversely, patients may be willing to accept more risk from a drug that promises better results.
Jean Slutsky, chief engagement and dissemination officer, Patient-Centered Outcomes Research Institute (PCORI), described the shift in focus: “Traditionally, medical research has largely been investigator-initiated and has not taken into account the decisional needs of patients or other stakeholders. Specific aims, key questions and outcomes usually evolve from investigator or funder priorities. This approach has led to innovation in treatments and helps steadily advance scientific knowledge, but it may not address the health needs most important to patients and communities.”
Bennett Levitan, M.D., Ph.D., senior director, Department of Epidemiology and Benefit-Risk Team Lead, Janssen R&D, explained how patient preference is incorporated into research. He said, “Benefit-risk assessment is the process of determining whether a treatment’s benefits outweigh its harms when compared to standard-of-care or other alternative treatment. In general, this involves a combination of clinical data and value judgments. Patient engagement is a growing field in which patients want to have a role in clinical development and approval—suggesting areas of research, providing input into clinical protocols, indicating which endpoints are most important to patients and providing the patient perspective on benefit-risk balance for treatments. Combined, these fields result in asking how the patients’ perspective on benefits and risk can be incorporated into a trial or into regulatory review in a transparent and defensible manner. That’s where the field of patient preference comes into play.”
Creating a patient-centered environment involves redefining the culture of research organizations and trial centers to move the focus to the patient experience. Employees, from researchers to marketers to sales staff, must be educated and trained in these new expectations. Many companies are implementing patient-oriented changes. Novartis, for example, has published a declaration for patients that lets individuals know what to expect from participating in the clinical trial process. LEO Pharma has a program entitled Engaging for Better Patient Care, which trains employees in patient-focused programs, emphasizing empathy and engagement, and provides a rewards system for employees who meet patient-focused goals. Janssen is focusing on collaborations to promote a patient-centric approach.
Levitan said, “The Innovative Medicines Initiative (IMI) is sponsoring the PREFER five-year project to develop requirements and standards for the assessment and use of patient preference data throughout the drug development lifecycle. ... The National Health Council is conducting numerous projects to support patient engagement methodology and policy.”
The FDA is also emphasizing the patient connection. “The FDA’s Center for Devices and Radiological Health (CDRH) strategic priorities for 2016 and 2017 include partnering with patients as one of their three key areas of focus. This includes both promoting a culture of meaningful patient engagement by facilitating interaction with patients and increasing the use of patient input as evidence in decision making, including the use of patient preference studies and patient-reported outcomes. On the FDA’s drug side, there is increasing discussion of how the patient perspective can be incorporated beyond the patient-focused drug development meetings begun under the Prescription Drug User Fee Act (PDUFA) V,” said Levitan.
Patients are making their voices heard. For example, when a drug for multiple sclerosis was discovered to be associated with progressive multifocal leukoencephalopathy (PML), it was pulled from the market by the manufacturer. The medication was eventually re-released when an FDA advisory panel took patient requests into account. Many patients wanted to use the drug, even with the risk involved. In another example, the Maestro obesity device was approved in 2015, even though it failed one of its primary endpoints. It was approved, in part, because of a preference study showing that a certain subset of patients would choose to use it. For those patients, the benefit exceeded the risk.
In the clinical trial realm, patients are demanding access to study results and greater transparency within the study itself. Patients can have input into study design and provide feedback on their experience in the study once it is completed. Patient perspectives may be used in all phases of R&D—from discovery through postmarket monitoring. “Patient partners can actually provide information about how useable informed consent documents are, how readable they are. ... They can help the investigator think about ways to reach patients in more comprehensive ways,” said Slutsky.
There are some barriers to creating this change, including a disconnect between the patient’s goals and those of the industry, and industry resistance to change. Communication between the industry and patients is also a barrier (e.g., disclosure of proprietary information).
“The quantitative work in preference studies has its origin in marketing, economics and decision analysis,” Levitan explained. “Not all clinical scientists are currently comfortable using patient survey data as a clinical instrument. Preference studies are different from patient-reported outcomes. They generally use hypothetical [questions] to find out how people would react to situations that don’t yet exist. There are still many barriers for the acceptance of patient engagement, though they are lessening. These barriers are related to corporate culture, financial concerns, process change, legal/regulatory concerns [about premarket discussions] and concerns about patient technical or clinical knowledge. While the business case for patient engagement is unclear for some companies, recent research ... is demonstrating the financial return on investment of patient engagement. Eventually, patient engagement will be folded into the normal drug development process, but it currently is new and often in the pilot stage.”
The paternalistic “doctor-knows-best” approach has fallen by the wayside, and patients are empowered to guide their care as never before. The push to create a more patient-centric environment can only help the clinical trial industry focus on those drugs and devices that are truly going to provide a patient benefit—however that is measured. If patients feel they are being listened to and respected, the industry benefits from increased recruitment and retention, not to mention the attainment of results that are clinically or personally meaningful to patients.
Noted Slutsky, “Patients are faced with making medical decisions about their care all the time. ... Creating research that is useful to patients will drive patient education, resulting in better healthcare decision making.”
This article was reprinted from Volume 21, Issue 15, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »