New partnership aims to improve access to rare disease trials, includes NC clinical research site

For patients with rare diseases, finding a relevant clinical trial can be a difficult and fragmented process, but a new partnership is creating a more connected pathway for patients and providers.

The collaboration brings together myTomorrows, a health technology company that helps connect patients and physicians to pre-approval treatment options, and Rare Disease Research (RDR), an independent U.S. clinical research site network focused exclusively on rare disease studies.  

RDR’s Hillsborough, North Carolina, location is one of the clinical research sites that the collaboration will initially support. Edward Smith, M.D., serves as the medical director and principal investigator for the site.

The collaboration aims to make it easier for patients, families, community physicians and specialist centers to connect with relevant RDR studies through a more structured and coordinated referral pathway.

"This partnership strengthens our ability to connect with patients earlier and more effectively through the physicians and specialist centers already involved in their care," said Han C. Phan, M.D., CEO of Rare Disease Research. "More structured referrals and clearer eligibility alignment can help our teams focus on the patients most likely to be a fit for a given study, while creating a more efficient and supportive experience for families and referring providers."

Bridging trial access gaps

Clinical trials often have complex eligibility criteria and rapidly changing recruitment needs, making it challenging for patients with rare diseases to find appropriate studies. Another hurdle is that referring physicians often lack a practical and consistent way to connect potentially eligible patients with specialized research sites.

Through the new partnership, RDR plans to use the myTomorrows platform to help referring physicians and specialist centers identify potentially relevant RDR trials and then submit structured referrals directly to RDR site teams.  

The platform is designed to support preliminary matching against study criteria while improving the completeness and consistency of referral information, before site-level review takes place.

Once a potential match is identified, providers can submit referrals to an RDR clinical research team and communicate through a centralized workflow designed to improve coordination. RDR investigators and site teams will still perform the required protocol-specific review and make all decisions about patient suitability and enrollment.

The collaboration will also help patients and families seeking information about RDR trials. For example, myTomorrows patient navigators can help gather relevant medical information, review potential trial options and support referrals of potentially eligible patients to RDR for site follow-up.

Boosting efficiency

“RDR is driven by a mission to accelerate the development of safe, effective treatments and provide access to innovative investigational therapies,” said Marcial Almaraz, site network operations manager at RDR. “By leveraging our specialized site network across Georgia, North Carolina, Florida and New Jersey, we aim to create a more connected pathway that reduces friction for families and advances translational science.”

RDR’s network supports more than 40 active clinical trials in pediatric neurology, pediatric emergency medicine and pediatric cardiology, with a focus on neuromuscular, neurological, lysosomal storage, metabolic and mitochondrial diseases.  

By enabling more structured, better-qualified referrals, the partnership is intended to help RDR teams spend less time managing incomplete or mismatched requests and more time supporting patients who are likely to be a fit for active studies. This will create a smoother experience for families and referring physicians while improving site-level operational efficiency.

"For trial sites, complex recruitment workflows can create unnecessary administrative burden and slow patient access," said Michel van Harten, M.D., CEO of myTomorrows. "By supporting RDR with structured referrals, preliminary trial matching and coordinated patient engagement, we aim to help simplify the path from referral to site follow-up."