Patient-Centric Solutions for Cell and Gene Therapy Trials
Cell and gene therapy clinical trials can experience a high screen failure rate as targeted treatments are often based on specific genetic markers. Additionally, these complex trials often require patients to make significant commitments, including undergoing invasive screening procedures, intensive treatment regimens, participating in long-term follow-up assessments and managing potential side effects.
To help drug development sponsors reduce delays associated with recruitment and advance the studies of potentially life-changing therapies, our Patient Recruitment and Engagement team at Fortrea is sharing a few of its patient-centric practices for fostering a sense of empowerment and partnership among trial participants and prioritizing their needs.
Providing patient education on screening and eligibility
Potential participants in a cell and gene trial should understand the rationale for inclusion and exclusion along with the process of screening, eligibility, trial, and treatment procedures such as apheresis (autologous cell therapy trials) and should also be informed of other available treatment options. Participants should not only understand why they may be suitable for the trial but also why they may not be suitable for the trial considering the life-changing potential of a cell or gene therapy treatment option. In the pre-testing phase, genetic counselors could play a supporting role in a clinical trial by providing patient education on genetic testing.
Understanding the complexities of long-term follow-up
In addition to the complex screening and treatment required in a cell and gene therapy clinical trial, long-term follow-up (LTFU) represents a significant commitment to the patients who participate in a cell or gene therapy trial. The patient journey in a trial, including the LTFU, can be complex. Sponsors, sites and CROs need to help patients understand what is involved in LTFU and how the study will consider their evolving needs over the long term, such as changes in their health, lifestyle, or social/family life.
It’s important to note that some cell therapies may require a lifelong LTFU, not limited to the 15-year LTFU typically seen in oncology studies of cell therapies. To be successful, these studies must examine how to drastically remove the patient burden associated with participation. For example, to monitor efficacy, surrogate measures can be identified for remote measurement or collected as part of the standard of care. The use of highly specialized tests for efficacy should be avoided or performed at a reduced frequency, if possible.
A flexible protocol could also allow for physical examinations and face-to-face assessments to be carried out by a healthcare provider who is not an investigator. The protocol could also include a lower-burden follow-up for patients who are too sick to travel to the site or who are receiving palliative care.
Sharing participation requirements upfront
Patient retention can be strengthened at consent by giving each patient a clear understanding of the study rationale and the requirements. With this information upfront, patients will better understand the motivations to commit to the study and learn how their compliance contributes to the study’s overall success.
Creating inclusive, patient-friendly study communications
Information should be created in a patient-friendly format with plain language and explanation of terms to account for health literacy and learning preferences of the targeted patient population. These materials can include:
- A combination of study awareness materials and educational tools
- Tailored informed consent media
- A study welcome pack
- A study overview flip chart
- A visit calendar
To promote diversity in recruited patients, any implicit bias should be examined in the materials. All study communications should be inclusive in their language and accompanying visuals should be representative of the patient population. Related to these efforts, Fortrea can develop a diversity action plan that helps sponsors enroll participants from underrepresented populations.
At Fortrea, our primary goal is to improve patients’ lives by compassionately bringing them life-changing therapies. Our dedicated global Patient Recruitment and Engagement team provides valuable support—from the start—to ensure that we engage patients and their families while supporting sites to appropriately consent, educate and maintain compliance with protocol requirements.
