Gene Therapy in Rare Disease Research


Applied Clinical Trials

Applied Clinical TrialsApplied Clinical Trials-06-01-2018
Volume 27
Issue 6

A look at home and dosing site considerations.

With the rise in gene therapies entering clinical trials, it is important to address the operational challenges associated with these types of trials. Some gene therapy trials necessitate centralized dosing like those that require patient samples to derive the investigational medicinal product (IMP), which call for a limited number of dosing centers.

In this case, the patient may initially be seen at one center for evaluation, dosing, and initial follow-up, and subsequently seen at another center for their longer-term follow-up activities-in some cases, these may be in different countries. Because the prevalence of patients in the rare disease space are just that-rare-special considerations must be made to ensure patient access to the clinical trials.

This creates significant logistical challenges not only from a patient and family standpoint but from a regulatory and data collection standpoint as well. We must make sure we consider all facets of these activities to make participation in this type of trial seamless to the patient and their family.

Regulatory considerations

From a regulatory perspective, country-specific requirements must be assessed from the outset. For example, when subjects are dosed in a country other than their originating country we must understand and be conscious of the requirements of both the originating and dosing nations. Even though there is no IMP and no dosing in the subject’s originating country, the regulatory authority may request to review everything related to the IMP. Unless a patient relocates for the full duration of the study, regulatory and ethics approvals will be needed in both the patient’s originating country as well as the dosing country.

Also, informed consent forms (ICFs) specific to patient travel and reimbursement are necessary. In the dosing country, regulatory and ethics may require approval for foreign patients to travel to the dosing country. Study documents must be available in the patient’s native language, including the dosing country ICF. For both nations, there is shared hospital liability and country specific insurance requirements.

Patient and family logistics

From a patient and family travel or relocation perspective, there are many points to consider, including travel logistics and relocation assistance. Imagine what must go into uprooting your family to another country or region to participate in a drug development program. There are passport or visa requirements; language barriers; and the need for an interpreter, school support, support for the rest of the family, including financial support, social support, insurance, etc. Additionally, given the length of gene therapy follow-up requirements, patients and their families will likely go through many changes (i.e., changes with family dynamic, relocation, etc.). Planning for all of these logistics at the outset are imperative for success in these types of trials to attract patients and ensure they remain in the study for the entire trial.

Data considerations

Lastly, because patient data will need to be monitored at the originating site as well as the dosing site, different sites will need access to the same patient data; and that access could cross country lines. Collaboration between the dosing center and originating center physicians is imperative to ensure continuity of care and, ultimately, for patient safety. Prior to participating in this type of study, a site must ensure that their data collection capabilities allow for such scenarios.

In summary, gene therapy adds additional complexity to clinical trials given the logistical challenges for specialized therapies, but it also allows for great scientific gains. When embarking on this type of study, we must always prepare for the regulatory intricacies and data needs early on and consider the patients, their caregivers, and their families to ensure that every enrolled patient stays enrolled throughout the entirety of the study.


Mariah Baltezegar is Executive Director, General Medicine, Syneos Health, and co-leads the company’s Health Rare Disease Consortium. She can be reached at

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