Clinical Research Considerations For Rare Disease Patients: Part 1 — Reinvigorate Patient Centricity

By Caitlin Horbacewicz, inSeption Group

Patients need to feel valued and supported. While most individual employees work to meet deliverables with this ethos in mind, it must be acknowledged that biopharmas face immense pressure to choose paths that both reward investors with the highest possible return on investment (ROI) and secure the organization’s longevity and profitability. These drivers often impact protocol, study design, and country selection, as well as patient-centric processes and accommodations, due to the study team’s requirement to adhere to cascading timeline, plus budgetary and commercialization priorities. Unfortunately, this dynamic has contributed to a negative stigma around clinical research in general.
We can overcome the negative stigma in the rare disease space through collaboration with patients, engagement with patient advocacy and support groups, and utilization of patient-oriented vendors and platforms. Even during clinical trial design (protocol development), some biopharma companies have begun incorporating patient focus groups to obtain feedback and perspective prior to finalizing the protocol to gauge whether any procedures, schedules, or regimens may contribute to enrollment challenges; this approach should be embraced as a best practice across the industry, especially the rare disease space, to better understand the patients’ previous treatment experiences, as well as scheduling and transportation challenges for the particular disease indication.
Patient-centric change must be driven within organizations: set an example and be vocal about it. The power to change the world’s mindset around pharmaceuticals and clinical trials is our responsibility. Carefully consider which products may provide the most impactful benefits to patient safety and treatment efficacy. When patients know a trial’s sponsor and investigators truly care about them, will not overburden them, and will take them into consideration as people, rather than generic sources of data, it forces the rest of the industry to follow suit.