Stalled recruitment, patient drop out, and limiting protocols are common challenges we solve at Worldwide for our sponsors. We recently were approached by a sponsor looking for a rare disease CRO to assist in developing a rare cardiovascular study with an exceptionally challenging patient population. We used our patient-forward approach to clinical development and provided full services for the Phase III, placebo-controlled, double blind, randomized clinical study for pulmonary arterial hypertension (PAH). Our approach was unique and well received, and ultimately was key to the program’s success and has led to further partnership on the sponsor’s cardiopulmonary pipeline.
Though we solve problems for our sponsors on a daily basis, we know no two studies—especially rare disease studies—are alike. For this sponsor, we leveraged our experience solving similar challenges in the past but utilized a problem-solving mindset to build a custom strategy from the ground up. Informed by past successes and failures, we dove in.
Five of the moves we made were:
- Gained a deep understanding of the patient population, disease, and their needs
We knew from the start of this study that developing a comprehensive understanding of PAH, the patient, their symptoms, and quality of life, was going to be integral to a successful study. Patients with PAH experience symptoms that greatly impact their life, and characteristics of their disease can affect how they are recruited and retained.
2. Partnered with a PAH patient advocacy group
A key part of deepening our understanding of PAH patients was our partnership with phaware , a patient advocacy group dedicated to pulmonary hypertension. Phaware helped us develop a thorough patient profile and assess the patients’ needs, wants, and desires, and provided valuable insights which informed parts of our recruitment and retention plan. For example, they homed in on the need for patient transportation support, as some patients with PAH are unable or unwilling to drive. This insight directly resulted in our recommendation to the sponsor.
3. Implemented a site support model with extra support
It was evident from our research that a typical rare disease site management model was not going to support the sites to the extent they needed to support this patient population. This disease has region-specific traits in the patient population and varying standards of care by country. These variances in management would require a site management model that considered and accounted for these variances. The need for regional variance in support and our need for standard operations for efficiency and performance were directly at odds. Read the case study to see our solution.
4. Proactively managed enrollment risks
Working closely with sites, we generated recruitment forecasts which reliably tracked potential enrollees from the point of identification through to randomization or screen failure. Because we knew early in the enrollment process where patients were identified and screened out, we were able to accurately forecast enrollment and proactively solve for problems as they arose.
5. Amended the protocol to be more patient-informed
Because of our patient-forward approach, it became clear a large barrier to enrollment was a comorbid condition that was listed as exclusionary. This condition was especially present in some regions more than others and was affecting recruitment greatly in those regions. We explored possible workarounds and ultimately discovered the exclusion was unnecessary.
To learn more about what we did to produce a 128% increase in average monthly enrollment for our sponsors, read the full case study: Innovative Recruitment and Retention Strategies for Phase III Rare Cardiovascular Disease Study. Our rare disease team brings together scientific and therapeutic depth with operational expertise and patient engagement to help you navigate obstacles and turn hurdles into hope.