Patients Involved – Clinical trial design

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.

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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

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