Screening and exit interviews are particularly beneficial to inform the development of drugs for rare diseases
Patient interviews can be helpful to obtain direct patient feedback regarding a variety of topics and can provide you with stronger support to underline how your new treatment is relevant and meaningful to the patients.
Especially in the case of rare diseases, the direct patient feedback that can be obtained from patient interviews can provide highly valuable input for your orphan drug development.
For example, interviewing patients entering a trial can help document the burden of disease, which in the case of rare diseases can be difficult and thus is very often underdescribed. In addition, it can help you better understand the benefits of a new drug despite the often small and heterogeneous sample of rare disease patients participating in a clinical trial.
Direct feedback from trial patients with rare diseases is also welcomed by the regulatory bodies.
The FDA acknowledges the value of getting direct patient feedback in clinical trials
In the FDA’s draft version of their PFDD Guidance 2 they provide the following examples of potential strengths associated with conducting screening/exit interviews (and survey instruments):
- In rare diseases, they can contribute cumulative evidence on demographics, medical history, and aspects of the patient experience.
- They can inform initial development or refinement of a clinical outcome assessment in early medical product development through cognitive interviews as part of a mixed method approach.
- They can add greater depth to data in rare diseases (or possibly other diseases with not much patient input) where stand-alone qualitative studies are less feasible.
- They can be used to obtain participant input on meaningful outcomes or meaningful change by eliciting patient definitions of symptom improvement, stability, or worsening.
Source: https://www.fda.gov/media/131230/download
At Clinigma®, we use our years of expertise in interviewing clinical trial patients to support pharmaceutical companies in obtaining direct patient feedback – including feedback from patients with rare diseases – to strengthen their (orphan) drug development.
Do you want to know more about how to interview patients in (rare disease) clinical trials? Please click here to visit our website.
Join us in examining the application of PROMs in clinical trials
Following the increasing attention from regulatory authorities on actively including trial patients’ voices in clinical trials, we have developed a survey to help gather insights on how pharmaceutical companies apply PROMs in their drug development to ensure patient feedback in clinical trials.
If you are working with PROMs in clinical trials and haven’t participated yet, please follow the link below to start the survey and sign up to receive the results.
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