Are We Grossly Underestimating the Returns on Engagement in Clinical Research?
According to a recent study, every dollar invested in patient engagement activities in clinical trials can return 500! As we see patient engagement use more widely during trials, this could easily be an underestimate.
Tessa Richards’ post in BMJ brought this shocking number to my attention recently. She was kind enough to mention me in the first line of the post, quoting a line I wrote back in 2012 on the “Blockbuster Drug of the Century” being an engaged patient.
Tessa’s post is a welcome update to what’s happening with patient engagement in research settings, and she provides some ample evidence that we are underestimating the value of having “patients as partners” in research.
(Dr. Larry Chu has built a conference, MedicineX, and courses at Stanford on this very subject and coined the term “patients as partners”. I imagine an emerging generation of physicians is starting to see the light about including patients in their care because of this.)
For medicine to truly change, however, we’re going to have to start driving the cold, hard economic benefits of having patients as partners in research, and in their own care. As much as we might like to believe otherwise, it’s economics that drives most decisions in health care. Sometimes economics and outcomes are not are misaligned in US healthcare, but the outcomes results are staggering as well.
Results from Kaiser in Colorado that led to my original post showed a 76% reduction in mortality by doing simple things like emailing cardiac patients during their post-event care.
When patients communicate about what’s happening with them, they care for themselves better, and they get better care.
With these kinds of results in outcomes, how can better patient communication and self-reporting NOT be part of the standard of care?!
In clinical trials, where the economics of patient engagement are well aligned, we’re just starting to scratch the surface. If you’re not thinking about so-called “Real World Data” (RWD), and the evidence (RWE) it will generate, as part of your trial design, you’re already behind. Regulation and economics are quickly stacking up behind engaging patients during trials. When results from those trials emerge in a few years, it may be too late to recover. Patient engagement during trials may determine which drugs get to market. The 21st Century Cures Act, which passed in 2016, places additional focus on the use of data that comes from communications and at-home care to support regulatory decision making, including approval of new indications for approved drugs.
But peer-reviewed research is also making the economic case for including patients as partners. In her BMJ post, Tessa highlighted a landmark 2017 study in Therapeutic Innovation and Regulatory Science showing a 500x return on patient engagement during clinical trials due to reduced amendments, better recruitment and fewer drop-offs! The paper does a great job of modelling potential outcomes of research and showing how patient engagement efforts could reduce both the length of trials and reduce costs as well. While cost reductions do make an impact, the greater benefit comes in the financial model from being earlier to launch.
I’ll quote some examples from the paper directly as examples of where patient engagement can help in reducing amendments and improve enrolment:
“The top reason for amending a protocol is to modify study eligibility criteria as a result of changes in study design strategy and difficulties recruiting patients.”
“66% of phase 2 trials and 77% of phase 3 trials have at least 1 amendment. Twenty-two percent of these amendments are classified as “somewhat avoidable” and are due to recruitment difficulty or feedback from sites or investigators.”
“On average, one amendment adds 90 days to the development timeline and entails direct costs of $141,000 for phase 2 trials.55 Indirect costs (primarily costs associated with sponsor and contract research organization personnel time to implement an amendment) are estimated to be 3 to 4 times larger.”
“Making the informed consent easier to understand, making the trial less demanding for patients, and providing in-trial feedback to patients may increase protocol adherence and reduce patient dropout rates.”
“We assume patient engagement in protocol design will reduce enrolment time by 25%.”
These results, while astounding, don’t even include the value of the data that could indicate how well patients will adhere to their medication once the drug is on the market! The paper looks models both Phase II and Phase III benefits.
In Europe, there’s a project to understand how to include patients and engage them at a deeper level in clinical trials, and effort to understand the “Return on Engagement” https://www.imi.europa.eu/projects-results/project-factsheets/paradigm. I’m really excited to see further evidence of how much value patients can add as partners in research. We’ll be all the better for it. If a 500x return on engagement is just the starting point, we’re going to see a big change in how research is conducted in the very near future.