Thursday, April 23, 2015

Live from #PCTUS: Siteless Studies, Barriers to Patient-Centered Trials, and Recruitment for Rare Diseases

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

Wrapping up today’s preconference activities were the Think Tank sessions. Attendees split up in open roundtable discussions to brainstorm solutions to industry challenges. In the Patient Engagement in Clinical Research track, the three topics up for discussion were “Overcoming Regulatory Barriers to Execute Patient-Centered Trials”, “Patient Recruitment Hurdles in Rare Disease Indications”, and “Exploring Siteless Studies”. Each group discussed one topic at a time, and then reconvened to share highlights and key findings.

It was fascinating to hear these topics being discussed from different perspectives, including sponsor, CRO, site, patient, among others. There were a lot of great conversations, and participants were very engaged in this interactive exercise. Here are some of the conversations that took place.

What does patient-centricity mean to you?

- Driving toward how we can communicate better with patients to help them be more compliant, improve the data we collect, and then share the results of that data with the patient
- Trials that patients are willing to enroll in
- When sponsors are willing to listen to patient feedback
- Bringing the trial to the patient
- Advocacy and engaging with patients as individuals
- Patient-centric trials aim to reduce the burden and increase the benefit to the individual patient, and doing so will improve recruitment and retention
- Creative trial design
- Helping patients with travel, stipends, and providing them with trial summaries (translated into language that’s actually understandable)

What are some regulatory barriers that stand in the way of patient-centered trials?

There are new approaches that are better, and technology is enabling us to do things differently, yet if it’s not validated, we avoid it (we don’t want to be told “no” so we don’t even ask). The FDA will have to move forward to become more patient-centered.  However, some barriers aren’t regulatory, but internal – from outdated SOPs to the excuse, “It’s always been done that way.” Old methods or resistance to change can be overcome, but it won’t always be easy. We have to push for it anyway, because it’s a fight worth fighting.

How would you define a siteless study?

- Patients don’t have to come to a central location; they can do everything remotely (from informed consent to specific procedures)
- A hybrid model where we can minimize or reduce what happens at the site
- Use of ePRO
- “Siteless” sounds like you’re giving something up. Instead, call it “site-free” or virtual site

This topic brought surfaced many other discussions, including the advantages and disadvantages of site visits, debate around if the quality and validity of data entered by a patient or parent is as credible as physician or nurse, and who would have oversight in siteless trials.

Can siteless studies work for every study?

After discussing a model that could work, it’s obvious that it doesn’t fit every study. It may be great for the right studies, but it also doesn’t mean that it’s the end of site studies (at least in the near future).

What is being done to aid in recruitment for rare disease studies?

Because extensive travel is typically involved, it’s not uncommon to use a travel concierge service, or whatever it takes to get the patient there. It’s stressful enough to travel long distances (sometimes overseas) with a sick family member, we don’t want them to worry about flights, rental cars, or where they’re going to stay. Some sites even have a “navigator” who facilitates as much or little as the patient needs.
Having someone there to guide and make arrangements is a great benefit, though it’s very time-consuming and rare outside of rare disease trials.

These topics and discussions were great reminders of how important patient-centricity remains in planning and conducting clinical trials and an excellent end to the first day of the conference. Tomorrow brings several exciting keynotes, as well as a variety of sessions and workshops. Keep following along for more #PCTUS highlights.


About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC​at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015. 




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