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Pediatric Rare Disease Studies: Patient-Centric Approaches

14 Sept 2022

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The phrase “patient-centricity” can mean many things but at heart, it means designing a treatment, clinical trial, or other healthcare solution based on what the patient or their family needs or wants. For clinical researchers, it means making the clinical trial process the easiest and best it can be, from the patient or family’s perspective. A patient-centric approach should start at the time a study or trial is devised. Clinical trial design is one of the most important places to involve the patient voice. For pediatric rare disease natural history studies or therapeutic trials, it is especially important.

This is because trials and studies in rare and pediatric diseases typically have little or no previous track record in clinical research, so feedback from children, parents, and patients is especially important, as they know more about the disease than anyone else.

Possible options include hosting patient groups, working with patient advocacy organizations, or analyzing real-world evidence (assuming there is some), so that trial eligibility criteria reflect the patient population.

For pediatric rare disease studies/trials, the protocol requirements are very important to the family’s decision to participate in the study. In order to design a protocol attractive to pediatric patients and their family, it is important to have early engagement with appropriate patient-centricity groups such as youth advisory groups, who have access to input from affected families and often links with the key investigators. Of equal importance is the need to involve regulatory authorities at an early stage to obtain agreement on study design and endpoints, and to present feedback from patient centricity groups.

A common strategy to build clinical trial and disease awareness is to give the study an identity or brand. Carefully done, this can reflect some of the aspirations of the study. Developing a proactive recruitment strategy is essential to ensuring that the study patient profile is known to all colleagues and healthcare professionals who may potentially refer patients to participating study sites. A strong study image with a memorable name or logo is helpful for referring investigators. A study website with a section for referrals and a section for patients, social media outlets like Facebook and Twitter, may also assist patient recruitment and enrollment efforts.

It is important to recognize that participation in any clinical study can be stressful for families. Providing pediatric rare disease patients and their parents with detailed study information ahead of time will reduce anxiety and support a pleasant trial experience. A well thought out, customized patient/parent information which clearly specifies what procedures will be performed, how long they will take, and any discomfort that procedure may be caused is always a useful add-on to the consent process. This proactive approach to educating the families/patients provides a comfort level with their obligations and sets up realistic expectations for study participation.

It is also important to realize that a pediatric research patient really needs family. Thought must be given toward the accommodation of the patient, parent(s) and siblings, even for a study of short duration. It is recommended to incorporate patient/family reimbursement into the study contract as well as ensuring participating site(s) have processes/infrastructure to minimize family burden by supportin