Patient groups have become a powerhouse in R&D. Here’s a look at their impact.

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Patient organizations are infusing billions into medical research, giving them substantial power over its direction. In the last five years alone, they awarded some $22.5 billion in research grants to promote their priorities, a trend that is likely to continue, according to a recent report by The IQVIA Institute.

Murray Aitken, executive director, IQVIA Institute for Human Data Science

Murray Aitken, executive director, IQVIA Institute for Human Data Science

Permission granted by IQVIA


“I think we’ll see more targeted funding by patient organizations for specific areas that they want the research to go in, whether that be biomarker identification or new modalities,” said Murray Aitken, executive director of the IQVIA Institute for Human Data Science. “They have the money.”

Patient groups are also exerting influence in other ways. They’ve invested $1.6 billion into therapeutics in the past 15 years and many are sponsoring clinical trials, creating registries, and driving trial enrollment. 

It’s not just the heavy hitters — such as the Michael J. Fox Foundation for Parkinson’s Research, the Cystic Fibrosis Foundation, and the Multiple Myeloma Research Foundation —  among the 3,300-plus patient groups in the U.S. that are steering industry R&D. Smaller organizations are also making a difference by striking deals with life sciences companies. 

“One of the lessons is that even smaller organizations that are not so well-funded can still have a big impact in helping drive and shape the focus of research,” Aitken said. 

Here’s more on how these organizations are moving the needle.

Partnering with drug companies 

Over the past 15 years, patient groups, including smaller organizations, have forged some 700 deals, totaling $2.4 billion with drugmakers. Some of these investments have gone into the search for disease biomarkers, as was the case with the Michael J. Fox Foundation, which recently celebrated a critical breakthrough in Parkinson’s. Established biomarkers can help identify drug targets, diagnose patients, find effective drugs, and measure how well they work. In other cases, the goal is to help speed research past a financial valley of death. 

“The intent of these arrangements are to lower the barriers to conducting research and de-risk investment by life sciences companies to translate academic discoveries into commercial products,” the report states. 

Groups also back promising therapeutics. Since 2017 the Leukemia and Lymphoma Society’s Therapy Acceleration Program funded four therapies that went on to receive FDA approval or were included in clinical guidelines.

Sponsoring clinical trial 

Patient organizations were involved in more than 2,000 trials in the past two decades, according to IQVIA, the bulk of them related to oncology or heart and blood vessel diseases. 

“As trial costs have soared and proof of efficacy has shifted to earlier phases, patient organizations are increasingly sponsoring [phase 1 and 2] trials, which account for 75% of their involvement in the past two years and are involved less in later trials,” the report states.

Infusing the patient perspective 

Federal regulations, such as the 21st Century Cures Act of 2016, have encouraged drug developers to listen more intently to patient voices and these organizations have stepped up to offer insights.

“They can help translate patient perspectives into what matters from a scientific and research perspective,” Aitken said.

An important change they’ve helped make is the addition of patient-reported outcome measures to clinical trials. As many as 27% of phase 3 oncology clinical trials included them in 2019, according to the IQVIA report. This outcome data helps prioritize treatments that address what matters most to patients.

“Patient-valued endpoints are becoming more important and can bring value to the life sciences companies,” Aitken said. “If they go to market, they have an endpoint that resonates with patients in addition to endpoints that resonate with clinicians.”

“One of the lessons is that even smaller organizations that are not so well-funded can still have a big impact in helping drive and shape the focus of research.”

Murray Aitken

Executive director, IQVIA Institute for Human Data Science

Establishing patient registries 

A growing number of groups have created patient registries, which is especially valuable in the rare disease space where it can be tough to recruit enough patients.

“There are now 159 patient organizations with registries in the United States, 62% of which focus on rare diseases where current understanding may be limited,” the report states. 

And because real-world data is increasingly being used to support functions ranging from patient recruitment to drug approvals, these registries can also shape trial design.

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