Medical Research: By Law, It's All About You

Your tax dollars fund medical research, but your say in what research actually gets done is

Now, a new, Congressionally-authorized nonprofit born of the Affordable Care Act is proposing a different model: What if medical research were driven not just by profit-seeking drug makers or academic researchers with niche interests? What if, instead, research pursuits bubbled up from patients and their caregivers based on the concerns, confusion and questions that arise from real-life dealings with the health care system?


The Patient-Centered Outcomes Research Institute (PCORI) is the first, and clearly the most ambitious, publicly-funded effort to integrate patients and caregivers more directly in figuring out what works in health care. They'll be able to push for what they need most, whether it's more effective asthma treatments, clearer information on childhood vaccines or preventing falls among the elderly.

Eventually, a new body of evidence — by, about and for patients — will be easily accessible to anyone trying to navigate the health care system or seeking reliable data on preventing, diagnosing or treating an illness, says PCORI's executive director Dr. Joe Selby, formerly the director of research for Kaiser Permanente, Northern California..

What's Best For Me?

In a radical rethinking of what constitutes "health research," patients are central to this endeavor and participate in every stage of the process: from generating and selecting study topics to determining the most effective strategies for communicating the results (not everyone subscribes to The New England Journal of Medicine, the thinking goes). PCORI has already spent $31 million to fund 50 pilot projects (out of 856 submitted) and it estimates $427 million in research commitments will be made by the end of 2013. By the close of the decade, PCORI expects to invest about $3 billion in research.

Currently the group is soliciting specific questions from patients and caregivers nationwide that might ultimately be developed into research projects. The questions can be on anything with a clinical focus: basically, any question that begins: "What's best for someone like me?" qualifies.

Last week, at a meeting in Boston, the board identified three specific research areas to pursue based on input from patients, caregivers and health-related organizations: treatment options for uterine fibroids and for severe asthma and fall prevention in the elderly.

PCORI's overarching research agenda is broad and includes figuring out the effectiveness and safety of alternative prevention, diagnosis, and treatment options; reducing health care disparities in the U.S.; and comparing approaches to communicating and disseminating research findings.

But Sue Sheridan, PCORI's director of patient engagement, says the heart of the endeavor comes down to patients. I came to know Ms. Sheridan after writing about the terrible medical errors she endured after giving birth to her son Cal, who, as a result, was left with permanent brain damage. She acknowledges that PCORI is still in its infancy and faces challenges, but says eventually patients will be armed with robust new evidence that will allow them to better take control of their family's health care decisions.

"As a mom," Ms. Sheridan said. "I'm faced almost daily with decisions that often I really have no support in making and sometimes it's frightening. And so my son and I make those decisions...often without the support, without the evidence, without the information. So as a mom, I can see how PCORI can really be serving the caregiver, the mother population and the clinicians so we can make those better decisions."

Parents, Children and Vaccines

"PCORI is one of the most exciting developments in research," said Tracy Lieu, Director of the Division of Research at Kaiser Permanente Northern California and formerly of the Harvard Pilgrim Health Care Institute and Harvard Medical School. "As investigators, we get used to being the ones to generate ideas. PCORI insists that patients be included, it can't just be investigators sitting around and deciding what to do — there's a higher bar."

Dr. Lieu received $641,817 as part of PCORI's first round of funding for a project on: "Incorporating Parent Preferences in Decision Making About Childhood Vaccines." As a pediatrician and a mother, Lieu said the idea for this project came to her because so many parents have such highly polarized notions about vaccines. "It's a problem that just bothers me," she said.

For instance, she says, one of the common concerns among parents is that children are receiving too many vaccines in one doctor's visit. Yet national vaccines recommendations appear to ignore this concern and instead specify simultaneous administration of all vaccines due at a visit.

"I see these conversations play out — parents have been increasingly concerned about whether they have enough support to make well informed choices for their children," Lieu said. "There are a lot of parents out there with legitimate questions and concerns and pediatricians feel stuck in the middle because they'd like to meet parents' needs and there is often not enough time to cover all the issues during a typical 15-minute well-child visit."

Lieu's study, in collaboration with Eve Wittenberg, Ph.D, a decision scientist at the Harvard School of Public Health, has two phases:

Initially, researchers will conduct one-on-one interviews with parents who have either declined or delayed a vaccine for their child. (As Lieu says, it's the "worried group in the middle" she's focusing on here, not the militant anti-vaccine crowd, nor the public health boosters who believe vaccines-can-do-no-wrong). These interviews will include questions about parents' attitudes about vaccines and where they get their vaccine information (is it from vehemently pro-or anti-sites they Googled?).

From there, Lieu says her team will design a national Internet survey to solicit parents' views on what might help them even more when it comes to vaccine decisions. Finally, the group will develop recommendations on what health care systems or physicians can do to help parents make better-informed choices.

This may not sound like earth-shattering research but in many ways it is. "One of the things that's very important to PCORI is that a stakeholder group is involved in the design. We have a national stakeholder group that includes parents whose children were injured by vaccines and some who have served in government [vaccine] advisory capacities. We are trying to bring together people with different perspectives to aid us on how to design the study. The stakeholder group advises the researchers. If this was an NIH grant, they typically don't insist that involved. This is really different."

It's Your Money, So Get Involved

Soon, PCORI will touch most Americans lives directly. Everyone insured through Medicare, private health insurance or a self-insured plan will be assessed a small fee to subsidize PCORI ($1 a year starting in 2013; $2 the following year and through 2019); the rest of the group's funding comes from the U.S. treasury. So anyone with a good idea that might lead to improved care should submit a question to the group's website where it will be evaluated for funding. Here's your chance to drive research into what is most important in you and your family's life!

(The group's next public forum, “What Should PCORI Study? A Call for Topics from Patients and Stakeholders” will take place December 4 in Washington, D.C. and there will be a free, live webcast provided.)

With it's charge to further so-called comparative-effectiveness research (essentially comparing which medical treatments work compared to others) PCORI may face some major obstacles.

Sarah Kliff wrote in The Washington Post earlier this year:

...comparative effectiveness research can be tricky. Study methods can be faulty and results contradictory; a particular medical intervention may work for one subset of patients but not for a larger population. These studies can also be controversial, and they sometimes meet with accusations of rationing. Government recommendations on PSA tests for prostate cancer and to change the frequency of breast cancer screenings met with public uproar.

Should I Eat This, Should I Take This Pill?

But folks like Danny van Leeuwen hopes this organization is different, and he's jumped on the PCORI bandwagon in an effort to spark change in the system. An Arlington, Mass. resident with multiple sclerosis who has worked as a nurse for 40 years, van Leeuwen addressed the PCORI board in Boston to offer several recommendations on enhancing the process of engaging patients with their own medical research. On his blog, Health Hats, he made the point that patients and clinicians make health decisions in radically different ways and those differences should be acknowledged.

"So many decisions to make in the journey toward best health. Should I take this medicine, should I have that surgery, should I stay home from work, should I worry, should I use a cane, should I tell my employer or my spouse, should I eat this, should I buy that insurance, should I see both doctors on the same day, and on and on and on. Clinicians and e-patients [which he defines as empowered, engaged, expert, enabled, equipped, emancipated] approach decisions so differently. For clinicians it's studies published in academic journals that show an increased likelihood that x works better than y for a specfic population. It’s habit and common community practice. For e-patients it’s often spur of the moment decisions, trusting someone. It’s about me.

Still, van Leeuwen is cautiously optimism about the organization's prospects. "PCORI has a chance of really making a difference — it crosses some pretty strong boundaries," he said. "This is fertile ground for significant change but it's going to take continued attention. I think the people who are running this are very cool, very well-meaning — to me this is a good use of government. But they are still fighting against the inertia of how we've done it all along."

This program aired on November 30, 2012. The audio for this program is not available.

Headshot of Rachel Zimmerman

Rachel Zimmerman Reporter
Rachel Zimmerman previously reported on health and the intersection of health and business for WBUR. She is working on a memoir about rebuilding her family after her husband’s suicide. 



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