Guest Column | February 8, 2022

4 Tips To Optimize Engagement With Patient Organizations For Rare Disease Clinical Trials

By Davelyn Eaves Hood, MD, MBA, director of scientific and patient affairs, Rezolute


In addition to the challenges that come with conducting clinical trials during a pandemic, recruiting for a rare pediatric condition introduces a host of new barriers. For example, there are simply fewer patients with the condition and even fewer who may fit the parameters of the trial. For rare disease studies in a primarily pediatric population, there is the added complexity of parents making the challenging decision to participate in a study that includes a yet unproven investigational product for their children. This makes the need for thoughtful, transparent, and informative communications imperative for companies embarking on clinical trials in this population. Often these communications are enabled by working closely with advocacy organizations but doing so effectively requires special attention by staff knowledgeable of the rare disease space.

While advocacy organizations are becoming more sophisticated, some may still have limited experience in the drug development process. Also, some rare disease organizations may only have a handful of staff attempting to accomplish big agendas, which can compete with the desired timelines of drug development.

As we at Rezolute near the completion of enrollment in a Phase 2b study for patients with congenital hyperinsulinism (HI), an ultra-rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas, I’d like to share my four reflections on what has helped us to overcome some of these barriers.

1. Assign a dedicated point of contact

To ensure timelines are met and advocacy partnerships are meaningful, identify appropriate staff to serve as a dedicated point of contact and make intentional efforts to educate and collaborate with advocacy organizations whenever possible. This individual becomes the “listener” to fully understand what is most important to the patient community. This in turn leads to better designed trials that include endpoints that not only meet scientific and regulatory expectations but also help meet the expectations of the patient as end users of a therapy.

2. Collaborate to provide needed resources to patients

It is also important to financially support the broader mission of advocacy organizations as this expands the advocacy groups’ capacities and capabilities and creates a landscape that ultimately facilitates development of new therapies. Advocacy organizations have projects and programs they have developed, or designed but not yet launched, based on the needs they have identified in their patient communities. Companies should look to uncover existing advocacy work and project plans and identify opportunities where existing advocacy work can meaningfully bolster drug development. This could include, for example, website development for greater patient awareness and recruitment, printed condition-based materials that may also improve the patient experience in the clinical trial, or patient-focused scientific research and publication efforts. Advocacy collaborations are a cost-effective approach that also directly supports the broader patient community.

3. Treat each advocacy organization like the unique community it is

When you’ve met one rare disease organization, you’ve met ONE rare disease organization. This saying is absolutely true. Each organization is unique based on staff, structure, and the characteristics of the given patient condition each represents. It is important, during outreach and collaboration efforts, to appreciate the distinctiveness of various patient organizations you may encounter and tailor efforts to the goals and culture of each group. Perhaps the most important thing to realize is that patient advocacy groups are disease experts and should be respected as such. There is no better way to get the full understanding of the burden and impact of the disease on the patient and their family than through interactions with patient advocacy organizations.

4. Avoid the checkbox approach

When beginning partnerships with patient groups or enhancing existing relationships, simply making a call, having a meeting with patient advocates, and ticking a box that the patients’ voices were heard will not suffice. A true partnership has the potential to bear many fruits. For example, when we launched our Phase 2b study, we attended family conferences and worked with advocacy organizations to review our study protocol, refine our understanding of the patient experience, and go over our communications to patients to make sure we were on point. We also worked together to optimize the best locations for trial sites and, even now, are working with our advocacy partners on product formulation, to prepare for regulatory discussions, and plan for future payer interactions. Not only is our work with advocacy organizations helping to successfully keep our RIZE study ongoing and substantially on track through the toughest of times during a pandemic, what we learned from patients and families via the organizations that represent them provided a deeper understanding of the congenital HI experience for our employees, board members, investors, and analysts.

At a time when industry experts are noting marked study delays due to factors such as patient recruitment, supply chain challenges, and lack of clinical facilities and staff to conduct the trials, we are experiencing a more modest delay in completing our planned enrollment goals. Perhaps we are simply lucky, but maybe it’s something more. The measures we’ve taken during COVID-19, including our approach to working with advocacy groups, can be used by others to alleviate challenges in recruiting for and enrolling patient-focused rare pediatric disease studies.

About The Author:

Davelyn Eaves Hood, MD, MBA, was a long-standing member of the patient advocacy group Congenital Hyperinsulinism International (CHI) and now serves as the director of scientific and patient affairs at Rezolute. She has served in various scientific and leadership capacities, such as the president of the board of directors of CHI and principal investigator for CHI’s HI Global Registry. Hood’s breadth and depth of experience within the HI community helps to inform Rezolute’s development program of RZ358 for the treatment of congenital HI by capturing the needs of the patients and fostering collaborative relationships within the scientific community.