In rare disease, every patient matters and it is important for CRO and sponsor medical staff to effectively monitor and support investigative sites with patient screening to achieve timely and efficient patient enrollment. Allucent is a clinical research organization (CRO) focused on the support of rare disease clinical research, and have developed an effective eligibility review process that has enhanced our delivery or research in rare indications.
The work we do in rare diseases is challenging and exciting and sets the groundwork for new therapeutic treatments to be developed for patients who have limited to no options. The diversity of the indications, the ways those conditions manifest, and how we need to monitor them requires our team to be creative and patient focused in ways that other therapeutic areas do not require. In this article, we highlight aspects of our medical monitor eligibility patient review process designed specifically to support rare research studies.
A Recent Example Involving Acromegaly
Recently, Allucent provided CRO services for a rare disease study that examined acromegaly.
Acromegaly is a rare disease. It occurs in roughly 50 to 70 people per million, with ~three people per million developing the condition every year, typically developing in patients that are 40-59 years old. Furthermore, the symptoms in the earliest phases of acromegaly may go unrecognized. The condition is underdiagnosed. Obviously, this means that finding patients with acromegaly is a challenge in and of itself.
With this level of incidence and study sample sizes typically being small by necessity, each patient counts for the Per-Protocol analysis (PPA) set. To prevent exclusion of patients from the PPA set and to prevent or minimize deviations from the Inclusion and Exclusion criteria, we implemented an eligibility review process in our prior acromegaly studies that has resulted in very positive results.
Overview of the Eligibility Process
- Each patient is approved by the Medical Monitor (MM) before randomization or dosing;
- The approval of each subject is granted through the Eligibility Approval Form (EAF) as prepared by Pharm-Olam MM in conjunction with the Sponsor; and,
- The Medical Monitor reviews the relevant eligibility data in the eCRF and de-identified, documented evidence of active disease as detailed in the study protocol.
Managing Enrollment in Rare Disease
Depending on the severity of the indication, the targeted age group, or the frequency of comorbidities, locating patients who fit the inclusion guidelines established in the study protocol is often challenging. There may be relatively few people in the world who have the condition and even fewer will meet the inclusion criteria while avoiding exclusion attributes.
These issues are not unique – all studies experience them. The problem arises in that rare diseases already have comparatively few people who have the indication being studied. Even fewer of them will meet the criteria so reaching the target sample size can be a challenge.
It is not unusual for people with rare indications to have other diseases and conditions that would make it impossible for researchers to isolate the impact that specific therapies would have on those individuals, so they may have to be excluded from study participation. Furthermore, those patients under consideration may be under treatment for the indication being studied or another one that they possess. These drugs or therapies could interact with the drug being assessed by the study in question. In turn, those people may be excluded from study participation.
The eligibility process we used in the acromegaly study is one example of how a CRO can successfully navigate rare disease indications. This customized approach helps our team to work with the Sponsors, Investigators, and subjects to meet enrollment needs for rare diseases.
Meeting Target Enrollment in Rare Disease
Meeting enrollment in rare disease studies requires several strategies often unique to that disease which ultimately lead to successful study execution. Allucent is adept at working with our sponsors and our sites in rare disease and to formulate those creative solutions, so we can go the distance for the patients in need and the research required.
From finding the right sites distributed across the globe, to deploying the effective eligibility review process discussed here, to providing patient-centric strategies that reduce and ease the burden for patient and caregiver participation, these and more are essential to enrollment success in rare disease.