Patient Access and Enrollment Strategies for Multi-site NASH Clinical Trials

Heidi Guthrie - photo portrait

Expert Spotlight

Heidi Guthrie

Senior Director of Clinical Strategies and Logistics, ProSciento, Inc.

ProSciento is renowned in the industry for its deep scientific and operational expertise in metabolic diseases, including NAFLD and NASH. The company has designed and conducted clinical trials in fatty liver disease for more than a decade and has a deep understanding of the unique challenges of NASH clinical research. ProSciento has a wide team of experts in fatty liver disease, including Heidi, who has honed her expertise in strategies and logistics for NASH clinical trials.

“ProSciento is unique in their NASH experience of more than a decade and unparalleled in their expertise in metabolic clinical research. I’m honored to be part of a team that is contributing in meaningful ways to the advancement of NASH clinical drug development, the discovery of medically relevant biomarkers, and the knowledge base of disease progression for NAFLD and NASH.”

Heidi Guthrie is one of ProSciento’s many experts in logistics for multi-site clinical research programs for metabolic drug development, as well as a leading authority in patient access and enrollment strategies for NASH clinical trials. Heidi joined ProSciento more than 10 years ago and, in that time, has contributed to the logistics and project management of over 100 clinical trials. Today Heidi is Director of Clinical Strategies and Logistics at ProSciento’s corporate headquarters and Lead for the company’s proprietary NASH PASS® program. When asked about her career experience at ProSciento, Heidi replied, “ProSciento is unique in their NASH experience of more than a decade and unparalleled in their expertise in metabolic clinical research. I’m honored to be part of a team that is contributing in meaningful ways to the advancement of NASH clinical drug development, the discovery of medically relevant biomarkers, and the knowledge base of disease progression for NAFLD and NASH.”

For our Expert Spotlight series, we had an opportunity to ask Heidi questions about the distinct challenges and corresponding strategies a sponsor or principal investigator should consider specific to NASH early phase drug development.

Q: What are the challenges that arise in conducting NASH clinical trials that ProSciento’s NASH PASS platform addresses?

A: NASH is considered the fast-growing cause of liver transplantation in the United States, but there are no approved therapies. This unmet medical need has generated substantial interest from pharmaceutical companies, patients, and physicians in the development of an effective therapy to treat NASH. The complexity of the disease adds to the complexity of clinical studies, including access to NAFLD- or NASH-diagnosed patients for enrollment and access to sites with advanced diagnostic techniques and the know-how to conduct NASH studies.

ProSciento’s NASH PASS directly addresses these challenges utilizing a proprietary methodology, an IRB-approved clinical protocol, a network of prequalified sites, and a team of NASH clinical research experts in operations, logistics and science. Together, these components are the foundation of NASH PASS, a science-driven, highly differentiating platform that provides our clients access to research-ready patients and prequalified sites ready for expedited start for their NASH clinical study.

The NASH PASS protocol and site network are utilized to identify and access at-risk patients and provide those individuals an opportunity to be screened for a diagnosis. For NASH clinical study enrollment, this means access to a large population of diagnosed, research-ready patients at prequalified sites ready for an expedited start. Moreover, patients screened and diagnosed via NASH PASS screening are reserved for NASH PASS client studies only, minimizing or eliminating competing protocols within a site.

Sponsors also have open access to the NASH PASS registry of searchable patient data and our team of experts to tailor trial enrollment according to their individual clinical development plan. This includes the conduct of feasibility analyses and modeling and optimization of I/E criteria, thereby reducing screen fails, avoiding delays in enrollment, and having an overall optimized study design.

Because of the nature of the data, NASH PASS is also an important, evolving platform for partnerships to enhance the scientific knowledge of NAFLD and NASH disease progression and the development of novel biomarkers for clinical research.

Q: What are some relevant metrics that would help an outsider understand the impact of ProSciento’s NASH PASS platform on the conduct of clinical trials?

A: For NAFLD and NASH clinical trials, NASH PASS is substantially reducing the timelines and costs by impacting start-up and improving enrollment. Study start-up for NASH clinical trials is generally a minimum of 4 months. This can be shortened to less than 3 months with the NASH PASS platform, as sites in the NASH PASS network are selected, prequalified and available for an expedited start.

Moreover, the NASH PASS database of prescreened, research-ready patients overcomes challenges and delays in enrollment. Utilizing NASH PASS, enrollment for clinical trials can be frontloaded, with up to 40% of patients enrolled in the first 3 months, and 100% of patients enrolled on time.

Simply stated, NASH PASS has proven to double enrollment rates, as well as shorten study startup timelines to less than 3 months and site activation to less than 30 days in most cases.

While quantifying the cost savings of using NASH PASS can be difficult, in phase 2 studies in NASH patients when NASH PASS is implemented with an adequate runway, we have seen cost savings in excess of $2 million on clinical trial execution alone.

Q: How early do you recommend implementing NASH PASS to impact study enrollment?

A: NASH PASS is a highly effective clinical research and patient access platform. Importantly, it is also a team of leading scientific and operational experts in NASH clinical research to support protocol design and development. As such, the ideal timing to begin implementing NASH PASS with full scientific support to inform protocol design and development and model I/E criteria through data analytics is a minimum of 6 months before study award. However, there are accelerated pathways when early scientific support is not requested, in which case a sponsor may choose to implement NASH PASS prescreening at 3-4 months prior to study enrollment.

Q: What are the primary qualifications a study sponsor should look for in a NASH clinical R&D provider?

A: A NASH clinical R&D provider should understand the science and operations involved in NASH clinical research and should be experienced in conducting NASH clinical trials. In particular, a NASH clinical R&D provider must have a comprehensive operational plan that addresses the unique challenges associated with patient access and the complex site infrastructure required  to support NASH clinical trials. The operational plan should be implemented by a project manager that has experience with NASH clinical trials, understands the issues associated with recruitment and enrollment, and has efficient processes to contract multiple (often 12-16) vendors and manage centralized reading and standardization of data. ProSciento, specifically, has more than a decade of scientific and operational experience supporting NASH clinical trials and specializes in patient access strategies and the conduct of high-quality clinical research for metabolic diseases.

For further resources about ProSciento’s NASH PASS: