While there is still plenty of work left to do in 2017—trials to enroll, data to collect—it’s always a good idea to pause briefly to survey the landscape ahead. Here are 4 trends that the PEPtrials team will be watching, and trying to positively impact, in the new year:
- Patient-centric enrollment programs will gain traction
The Food and Drug Administration (FDA) supplemented its Patient Focused Drug Development program with the first-ever meeting of its Patient Engagement Advisory Committee. It took 2 years from when the committee was announced to its first meeting, but the fact that this committee even exists is a sign of positive change.
On the industry side, we have seen a wave of patient engagement programs and pilots over the past year. Many of these have been misfires: one eye-opening survey showed that 68% of pharmaceutical companies that have attempted patient-centric initiatives have failed.
This has not been the experience at PEPtrials. We see our clients learning from their initial challenges and coming to embrace one of our core principles: effective trial recruitment requires effort, empathy, and engagement. There are no silver bullets, no shortcuts, no “disruptive” technology that will magically bring patients to trials–but if we put in the time and energy to connect with patients on their terms, we can use real patient centricity to deliver real results.
- Minority recruitment will need focused attention
Then-FDA Commissioner Robert Califf declared 2016 “The Year of Diversity in Clinical Trials” in an enthusiastic blog post at the beginning of that year. That pronouncement may have come more from wishful thinking, as it turned out not be a realistic prediction of what was going to happen that year. In fact, white patients continued to be heavily overrepresented in trials in 2016 (representing 76% of enrollees), only 3% lower than in 2015.
With the release of the FDA’s Clinical Trial Snapshots Summary report due this January, we will have a third year’s worth of data to better understand how we are doing. We do not expect the numbers to show much improvement, and the current senior leadership is not expected to make minority participation in clinical trials a priority.
At PEPtrials, we continue to connect with underrepresented communities to bring them information about clinical trial options. We know firsthand that minority patients will enroll in trials when engaged in an open and transparent manner, and we will strive to bring that approach to many more trials.
- Trials will continue to get slower and more burdensome, requiring better enrollment strategies
Five years after “Eroom’s Law” documented the fact that growth in R&D costs has consistently outpaced new drug approvals, we still find that our trials are growing less and less efficient. This is occurring despite better technology and more precise therapies. Or perhaps it’s because of these very things.
While we are all glad that the era of paper case report forms (CRFs) is (mostly!) at an end, the advent of eCRFs has rather perversely made it easier to make everything more complicated.
The same thing has happened on the patient side: the growth of ePRO on mobile devices has led to an explosion of diary assessments. And the more cost-effective tests and imaging becomes, the more numerous and invasive the testing at each visit.
And of course, the more targeted our therapies get, the narrower the slice of the population that will actually be eligible for the trial. That may translate into smaller sample sizes, but it also makes patients extremely difficult to find. Given how generally poorly sites are compensated for screen failures, it should not be surprising that this still nets out to longer enrollment periods.
PEPtrials will continue to bring the voice of the patient into the earliest stages of clinical trial planning, with a goal of producing trials that are patient friendly, while still maintaining scientific rigor.
- Patients will continue to need new therapies and treatment options
Many of us in the clinical trial world have a tendency to blame an improved standard of care for slow enrollment. No doubt available treatments are on the whole better now than in the past. But the fact remains that few diseases have cures, far more have treatments with limited effectiveness, and a large number have no cure at all. Patients with Alzheimer’s, ALS, and metastatic cancer, among many others, are in desperate need of new therapies.
In 2018, PEPtrials will be pushing ahead with the words of Dr. Paul Janssen ringing in our ears: “There is so much more to be done; the patients are waiting.”