Patient, heal thyself: The next generation

April 28, 2015

Forty years ago, patients had no role in the medical research process aside from donating their blood or money, noted Kim McCleary of FasterCures, moderator of “Smart on Science: How Today’s Patients Are Driving Cures” at the 2015 Milken Institute Global Conference. But all that’s changing. As panelists explained, today’s patients are more active, engaged and integrated into the decision-making process.

“Patient input is important for all stakeholders,” said Bray Patrick-Lake of the Clinical Trials Transformation Initiative. She shared her own experience as a patient in a clinical trial that was halted due to “low and slow enrollment.” Patrick-Lake expressed frustration that “patients aren’t getting what they need from the system” and highlighted the benefits of involving patients throughout the process, including in clinical trial design. Without their input, the necessity of a placebo arm, for example, may not be understood by participants and the trial may not succeed.

Getting a drug approved by regulators is not the finish line, however. “You have to demonstrate its value to patients,” said Mace Rothenberg of Pfizer Oncology, Inc., who noted that industry is recognizing the importance of integrating patient perspective in drug development. Indeed, no matter how innovative a product, it will fail if it doesn’t have value to patients. Pfizer experienced this with inhalable insulin, an innovative product that faced two key challenges: It didn’t replace insulin injections, and the device required to administer it was too cumbersome and unwieldy for patients. Rothenberg noted it was a “commercial failure because the company didn’t take the patient perspective into account.” 

Taking the idea another step further, Lou DeGennaro of the Leukemia & Lymphoma Society highlighted the President’s Precision Medicine Initiative as an effective way to shift the focus toward patient-centeredness. Specifically, when it comes to cancer and cancer treatments, having the ability to focus on the individual driver mutation for patients rather than the tissue of origin can have huge benefits for all cancer patients. DeGennaro noted that there is real “value in innovation.” A discovery in one area may lead to treatments in others, and that value can’t be overlooked.

Precision Medicine Initiative’s effort to launch a national cohort study presents “a great opportunity to have 1 million current and future patients speak to us,” said Tony Coles of Yumanity Therapeutics. This cohort, he said, will enable a level of engagement that goes beyond traditional conversation between patient and physician, and will allow for the sharing of medical histories, genomic data, symptomology and other information that is critically important to making progress in biomedical research.

Likewise, patient involvement is a cornerstone at the Patient-Centered Outcomes Research Institute (PCORI). Its executive director, Joe Selby, described the institute’s motto of “research done differently.” Through patient engagement and a focus on patient-centered research, PCORI has an emphasis on finding treatments for rare diseases or those with no existing treatments. By engaging patients on their research teams, researchers are challenged to evaluate different outcomes. This can shift the goal in comparative studies in clinical settings, from simply evaluating which treatment is better, to “finding out which ones work better for which patients.”

McCleary closed the panel by asking each participant to identify what the “future perfect” looks like. The answers ranged from using technology more effectively to engaging in new and effective collaborations. As Rothenberg said, “Patients will be asked to give more than blood or money, they will be asked to give their ideas.”