Last update: 11 July 2023
Introduction
An example of patients involved through patients and patient organisations providing inputinto a Cushing’s disease clinical trial design. The consultation took place between Novartisand nurses from different teams (USA, Canada, and Brazil), Cushing’s disease patients
(USA) and caregivers and patient organisation representatives.
- When does it happen? – Phase III
Description of the case
The objectives for this case were to:
- Obtain specific feedback on sections of a draft protocol from patients, caregivers,advocates, and research study coordinators on Cushing’s disease clinical trial development.
- Identify potential areas of concern from the patient and research community that may impact the Cushing's disease trial accrual process in the future.
- Identify ways that Novartis can help support new product development with related tools and educational materials for patients with Cushing’s disease.
Type(s) of patient (advocates) involved
- Patients with personal disease experience.
- Expert patients / patient advocates with good expertise on disease, but little R&D experience.
- Expert patient / patient advocate with good expertise on disease and good R&D experience.
Benefits of patient involvement
The following topics were discussed and then given to the development team:
- Multiple issues to consider when designing a rare disease trial including patient-investigator communication, enrolment, education, psycho-social support, access,tracking, reporting and follow-up.
- Study length, transportation and visit schedule were named among the main barriers from the patient perspective.
- Working with patient advocate groups in clinical trials is crucial. Patient advocate groups should be used to announce and disseminate information about trials to boost enrolment.
- Consideration on the proof of concept and possible importance of the medicine being studied for the patients.
Results
- Trial design was better tailored to patient’s needs.
- Different perspectives and insights allowed to better inform a study design.
Challenges and barriers
No specific barriers were experienced.
Learnings
The need provide better preparation for the patient advocates to participate in the discussion, including topics such as:
- What a clinical trial is.
- Why a clinical trial is important.
- What to expect in a clinical trial, along with the time commitment it requires.
- Why certain tests in a clinical trial are essential, potentially providing tips to facilitate
management of these tests. - What a trial crossover means, and why it is needed.
Attachments
- Patients Involved Case Report – Cushing’s clinical trial
Size: 450,012 bytes, Format: .pdf
An infographic describing a patients involved case report of patient input into a clinical trial design for a Cushing’s clinical trial.