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P52: Patient Engagement in Cell Therapy and Gene Therapy Clinical Trials

Poster Presenter

Tim Turnham

Vice President
VOZ Advisors
United States

Objectives

Identify how and why cell therapy and gene therapy trials across multiple therapeutic areas require a more sophisticated, holistic approach to patient engagement than more traditional trials do, and how this new approach may raise expectations of patients for all future trial experiences.

Method

A roundtable was held, comprised of advocacy leaders, patients, and parents of pediatric patients in therapeutic areas impacted by cell therapy and gene therapy to discuss unmet patient needs around these studies. Findings of this poster reflect consensus real-world feedback from those participants

Results

Cell therapy or gene therapy trials (CGT) are highly technical, involve new terminology & techniques, & place unusual restrictions on patients. Participants report little understanding of basic words & concepts, yet in many cases felt they could not take the time to gain necessary knowledge before agreeing to participate in the study. Some urgency is due to the aggressive nature of the disease, but also the lack of trial slots resulted in pressure to enroll quickly. This has implications for informed consent; many participants report signing consent documents without little understanding of their content or willingness to report toxicities. CGT trials often require patients to travel or remain on-site for weeks. Travel support characteristic of traditional studies is not sufficient in these situations. Participants encountered difficulties in managing pragmatic aspects of dislocation. Travel increases the financial burden of engaging in a trial. Pediatric patients reported closing their business, quitting their jobs, taking an extended leave of absence—all at a time when unreimbursed medical expenses were increasing radically. Patients are frustrated that confidentiality agreements often bar them from receiving or sharing insights & advice from other patients. The information gap extends to data regarding the study itself, as company investors may have more knowledge of a trial that is available to the trial participants. Most patients are treated for their disease locally, but could not participate in the study through their local physician. In many cases the local practitioner had little or no knowledge of CGT in general or this trial in particular, and study sites did not share information with the local treating physician. At times this meant that patients were given a particular test—e.g., MRI, CT Scan—at the study site & then were forced to have the same test repeated locally because the study team would not share the test results with the local physician.

Conclusion

Sponsors can demonstrate patient engagement by embracing a broader approach to patient education that includes clearer explanations of the science of the trial, better ongoing information about the study, and pragmatic guidance on what to expect. This approach to patient education cannot be a single document, but should involve multiple touchpoints throughout the study and the short-term follow-up. In particular, a “guidebook” that serves as a companion to the trial will create a better patient experience. This guidebook should contain pragmatic advice about travel: how to manage air travel, from security to luggage pick-up, while transporting a child with disabilities who is in a wheelchair; how to find a vegetarian restaurant in the site city; how to deal with laundry. The guidebook should be designed in collaboration with patients who have been through the study. Sponsors should also create better communication channels between the study team and the local treating physician. This ensures continuity of care but also reduces redundant tests and concomitant disruption and cost. The idea of trust is woven through the findings of this roundtable. Lack of transparency reduces trust, even if that transparency involves non-clinical aspects. In rare diseases patients know the community well and who the sponsor chooses as principal investigator can impact trust, positively or negatively. CGT offers great promise, and with more than 900 studies currently registered at clinicaltrials.gov, this is clearly a rapidly expanding aspect of drug development. Focusing on patient centricity now can result in faster accrual, better compliance to follow-up, and better outcomes. A new, more robust approach to listening to, communicating with, and caring for patients and their families may become not only the standard for CGT trials but also best practice for trials more broadly.