Only about 5% of rare diseases have at least one approved treatment available for affected patients. To increase the availability of care options in this therapeutic area, sponsors need to ensure their clinical trials are as time-efficient and budget-effective as possible. But meeting timeline goals can be one of the challenges sponsors face with rare disease clinical trials.
The Ethics Committee approval process can cause delays in sponsors initiating the clinical trial. The rarity of the disease itself can cause delays in recruitment, if not enough patient candidates are available to participate in a trial. So how can sponsors speed up enrollment in rare disease clinical trials? Here are five clinical trial enrollment strategies to consider.
In the face of a life-changing, rare disease diagnosis, patients or their caregivers may take to the internet to search for solutions. Make sure your clinical trial and associated sites are easy to find online, and that your website includes the information patients or caregivers need to take the next step toward enrollment. Give them the opportunity to self-elect for your study by including useful information like:
Share clear information via clinical trial recruitment websites (like the Study Websites we build for sponsors) to increase the likelihood that the most eligible candidates are connecting with the site–those that are able to reach the site location, and those that match the criteria.
Among the challenges in rare disease clinical trials is site staff’s limited ability to connect with enough patients who have a rare disease, let alone enough patients who have that disease and meet all of the inclusion/exclusion criteria established in a protocol. If you are supporting sites with a referral campaign or by launching a website with information about your clinical trial, incorporate a digital pre-screener form as well. Without a pre-screener form, you risk driving more patient candidates that don’t meet inclusion/exclusion criteria to your trial sites, adding more work for site teams.
Implement pre-screener forms based on inclusion/exclusion criteria that do not require screening to determine adherence. A multiple sclerosis pre-screener form, for example, could help determine whether a patient has the right documentation of a specific EDSS score from a neurologist, or if they have received a previous treatment that conflicts with the investigational product.
At OneStudyTeam, we can either incorporate these forms into new Study Websites for sponsors, or we can build them into a sponsor’s existing trial finder. When patients are filtered through pre-screening before their information reaches the clinical trial site, site teams will not have to spend time pre-screening ineligible candidates. Rather, they are able to focus on patients that are eligible to move forward with the remaining screening tasks prior to enrollment.
When sponsors regularly gather insights from de-identified data shared by site teams, they are able to quickly identify opportunities to intervene earlier during enrollment. In the case of rare disease clinical trials, both source reporting and I/E criteria reporting can prove essential.
As mentioned above, sites enrolling rare disease trials often struggle to get enough patients that meet the protocol’s inclusion/exclusion criteria. This naturally slows down the enrollment process. But protocol amendments are not uncommon in rare disease trials, either–OneStudyTeam clinical trial associates have seen some reach as many as eight amendments.
To better understand patterns in criteria failure, sponsors can work with sites to establish pre-screening and screening practices. This should include the digital recording of reasons for each inclusion/exclusion failure per patient, within patient enrollment performance management platforms like StudyTeam. Sponsors can then easily review de-identified, digital patient logs to analyze which criteria are creating roadblocks, and whether those can be relaxed to speed up enrollment. For example, in a cystic fibrosis trial, comorbidity could be an exclusion criterion that could be relaxed if deemed safe for the patient population.
During the pre-screening phase, sponsors can also establish a practice of source reporting with their site teams. When sites are updating patient information in their digital logs, they can include the source of recruitment, whether that is a clinical trial website or a referral campaign. When they run reports on that de-identified patient log, sponsors can identify which sources to direct more effort and budget toward, in order to drive the most eligible patients to relevant trials and sites.
Sponsors can treat rescreening as a key opportunity to reduce screen failures during rare disease trials. By including allowances for rescreening within the protocol, interested patients from an already limited pool have another chance to enroll even if they failed initial screening per certain I/E criteria.
Sponsors who incorporate decentralized elements into their clinical trials, or trial components that take place beyond a physical site, can reach patients where they are. If enough eligible patients are already regularly receiving care at major hospitals the sponsor is connected to, a centralized site model could work well for recruitment.
If that’s not the case, consider decentralizing certain components of clinical trials in rare diseases to reach patients who may already be limited in number. Consider a model that allows pre-screening by phone, and sends site staff to a patient’s home to conduct certain tasks, such as collecting blood samples during the screening phase. Decentralized components that reduce the amount of travel needed can lower the burden on both patients and caregivers, increasing the likelihood that the patient will be willing and able to enroll.
In order to make progress in developing therapies for rare diseases in clinical trials, it’s critical for sponsors to not just reach the right patients, but to reach enough of them to gather adequate metrics about the efficacy of their investigational product.
Sponsors who work with OneStudyTeam gain access to an enrollment management platform (StudyTeam) to support easy pre-screening data collection processes and enrollment reporting, as well as additional tools like pre-screener forms and platforms like Study Websites. To start, see how StudyTeam can support your rare disease trials by booking a brief demo.