Note: We occasionally ask leaders in the life sciences community to contribute to our Industry News section. Chris Learn from Parexel International spoke at the inaugural CRO Summit on “Cell and Gene Therapy Trials: A Site Collaboration Success Story.” We asked Chris to provide some additional insight about clinical trials for cell and gene therapies for this guest blog.
For many of us in the drug development industry, cell and gene therapies (CGT) can create intoxicating levels of hope for the patients who need them. This field is rapidly expanding, offering exciting possibilities for treating a wide range of diseases while providing veritable cures for some forms of hemophilia, sickle cell disease, blindness and deafness.
However, the complexities of CGT trials present unique challenges for the trial sites that first bring these unapproved investigational drugs to test subjects. For trial sites, CGT trials often result in intense resourcing workflows and requirements, leading to under-resourced healthcare teams. This hinders the advancement of these potentially life-changing treatments.
These challenges can include complex protocols and logistics, as well as the need for specialized resources to deliver the protocol to patients on a day-to-day basis. These factors contribute to site overwork, fatigue and burnout, particularly among academic research centers heavily invested in CGT trials.
To address these issues, improved collaboration among key stakeholders – sponsors, vendors, and sites – must occur. Collaboration can help:
- Streamline protocol design, especially around scheduled assessments and primary endpoints,
- Enhance communication about what sites can and cannot deliver in terms of timing and processes in support of specific protocol tasks, and
- Provide additional material support to sites per their requested needs and not just superfluous tools that burden the site staff with having to learn and use.
Simply, drug developers and their partners need to be better listeners and proactive stewards when using a clinical site’s resources. To this end, drug sponsors and their partners can play a crucial role in facilitating site collaboration by aligning on primary endpoints and ‘nice to have’ exploratory endpoints, investing in site development through site staff enablement options, and providing supplemental Q&A guidance for protocols.
To do this, locking down key protocol workflows through site consensus, conducting feasibility assessments of protocol Schedule of Events, and providing supplemental information and support to sites before it is requested are a must.
Regional hospitals – an alternative to academic institutions’ sites
CROs can contribute to site collaboration by also identifying and supporting non-academic institutions capable of conducting CGT trials. This includes identifying community-based researchers and regional hospitals and providing them with the necessary information, support and expertise to successfully initiate and run these trials.
Primary academic research centers now have so many clinical trials competing for specific patients that enrollment across all trials can become limiting. Notably, if only 3-8% of all eligible patients participate in a clinical trial for a particular disease, casting a net beyond academic centers is good for the sponsor’s study enrollment milestones. And it is more inclusive of a larger patient population that may benefit from and pay for these therapies.
In summary, the complexities of CGT trials demand a collaborative approach to ensure the successful development and delivery of these innovative therapies. Providing adequate support to clinical sites is crucial for success. This includes offering recurring protocol lifecycle training, sharing best practices and lessons learned, and creating protocol-specific guidance documents.
By working together, sponsors, CROs, and sites can reduce some of these challenges while potentially improving the patient journey. The future of CGT relies on effective collaboration among its key stakeholders, and a commitment to supporting participating clinical sites is at the forefront of this exciting field’s most pressing needs.
Chris A. Learn, PhD, PMP, is senior vice president for Parexel International’s Cell and Gene Therapy Center of Excellence.
