Trial decentralisation
Case study
Enhancing clinical research accessibility and reducing burden on rare disease patient population.
Clinical trial participation exposes rare disease participants to significant pressure financially, logistically and psychologically, and these incremental pressures can negatively impact a patient’s ability or willingness to enrol or remain in a clinical trial.
Challenge
ICON partnered with a drug development sponsor to assess and mitigate the burdens that could impact enrolment for a planned Phase III study in a rare endocrinology indication. Available evidence suggested that the proposed protocol would require 6.3 more on-site visits per year than the Standard of Care (SoC) for patients with this indication. Using a combination of evidence and operational strategy, ICON developed a hybrid decentralised study solution that would mitigate the burden on participants, deliver higher retention rates and increase overall study efficiency for the Sponsor.
Study description
A randomised Phase III trial with an open-label extension examined the safety, tolerability and efficacy of an investigational product in combination with SoC over the counter vitamins to placebo in adult patients with Hypoparathyroidism.
ICON Services: Therapeutic Area: Study Phase: Services: | Study Duration Regions | No. of Clinical Sites Target No. of Patients |
Key points considered during this analysis:
- Will decentralisation of these assessments significantly reduce the burden on patients enough to improve recruitment and retention?
- Will allowing flexibility for site visits accelerate timelines by allowing us to gather data faster?
- Will these changes reduce the workload for clinical trial sites, resulting in time and cost efficiencies?
- Will each country have unique regulatory requirements for the use of decentralised methods (home-health, mobile)?
Challenge
Healthcare is a highly regulated industry that can pose some challenges to the adoption of elements of decentralisation. Yet as technology progresses so should the evolution on the traditional site-centric clinical trial model that can impact a rare disease participants ability or willingness to participate in that clinical trial. To help remove unnecessary burden, the ICON team began by reviewing every assessment at each study visit and identifying those that could be replaced by mobile or home-health solutions to more closely mimic SoC. We analysed the most efficient way to communicate with patients (i.e. tablets and smartphones). We also analysed ways to reduce the frequency and duration of in-office visits by performing assessments in the patient’s home (home-health) and by shipping study drug directly to the patient. After identifying which method (home-health, mobile, in-office) would be most appropriate for each assessment, we then determined how each study visit could be best operationalised.
Primary endpoint
At 26 weeks of treatment, the proportion of subjects with:
- Albumin-adjusted serum Calcium within the normal range
- Normal 24-hour urine calcium
- Not taking active vitamin D supplements
- Taking ≤500 mg of calcium supplements
Initial protocol design
Initial protocol design structured 100% of assessments to be completed in-office for each visit.
Reduced site & patient burden: % of assessments
ICON identified that certain visits could be managed remotely which would reduce study burden on the patients and could benefit recruitment and retention.
Notes: Abbreviated term ‘LV’ = Lab Visit while ‘V’ = Visit
Remote visits are completed off-site and in a patient's home or local lab of choice. Flex visits can be done on or off-site pending
Primary Investigators medical assessment of each individual patient. On-site visits are conducted at designated study site.
Solution
With our data intelligence providing insights on SoC and the patient pathway, combined with our therapeutic expertise and local country regulatory knowledge, ICON presented an innovative operational strategy with decentralised elements that reduced risk to recruitment and retention while offering additional study conduct efficiencies.
Outcome
ICON worked with the Sponsor to amend the protocol to allow for the purposed elements of decentralisation, which enabled:
- The option for 80% of the total visits to be performed as a home health visit instead of in-office
- Accelerated patient recruitment by + 0.17 p/s/m higher than the initial projected enrolment rate
- Increasing participant diversity
- Improved reliability and accuracy of data
- Easier reporting and analysis of results