Patient Involvement in Scientific Advice

The Scientific Advice Program includes patient input as part of the process for developing advice. There are 2 main sources of patient perspectives that are used:

1.   Information Submitted by the Applicant in the Briefing Book (if available)

Applicants to the Scientific Advice Program are asked to describe the patient engagement activities they have conducted to date. As part of the Briefing Book submitted, applicants indicate any consultations or interactions they have had with patients or patient groups related to their drug development program regarding input on the design of clinical trials. If this information is available, CDA-AMC takes it into account when preparing Scientific Advice for the applicant.

2.   Patient Interview

For each application, we contact at least one relevant patient group to arrange for a patient representative to speak with CDA-AMC staff. When a patient group does not exist for a given disease or condition, we will attempt to engage an individual with the disease or condition. No written submissions are required of the patient representative(s), and the patient representatives do not attend the Scientific Advice meeting between CDA-AMC and the applicant for the Standard Scientific Advice process. For the Parallel Scientific Advice process with CDA-AMC and National Institute for Health and Care Excellence, patient representatives engaged by each organization may attend and participate in the Scientific Advice meeting. Patient representatives are provided an honorarium for their involvement.

The interview between CDA-AMC and the patient representative(s) occurs at a time between application approval and the Scientific Advice meeting between CDA-AMC and the applicant. A summary of the patient interview will be shared with the Scientific Advice team and the patient's perspective will be considered in the development of the Scientific Advice. If permission is granted by the patient representative(s), CDA-AMC will provide the summary of the patient interview to the applicant as an appendix of the Record of Scientific Advice. Relevant patient group input from past submissions to CDA-AMC may also be included in the appendix, when available.

As background information for the patient representative(s), applicants to the Scientific Advice Program are asked to complete and submit a Patient Drug Information Form. This form requests information about the drug and the planned trials that will allow for a relevant conversation about the application between CDA-AMC and the patient representative(s). Completion of the form is optional for applicants and the form may be completed in whole or in part (i.e., all fields are optional except drug name and applicant name, as these are needed for identification). Applicants are encouraged to complete the form to allow for input of highest relevance from patient representatives.

Confidentiality and Patient Input Process

Patient representatives are required to sign the Nondisclosure Agreement and to comply with the Confidentiality Guidelines for Scientific Advice and the Conflict of Interest Guideline for Contractors.

To protect the identity of the drug name and the applicant's name, there is no public call for patient input. Instead, CDA-AMC staff liaises directly with patient groups to solicit patient representatives. The drug name, applicant name, and the Patient Drug Information Form (if applicable) are shared with the selected patient representatives only after the Nondisclosure Agreement has been signed. This information is not shared with other members of the patient group.

Although patient input is part of the process for every application to the Scientific Advice Program, confidential information from the applicant and information produced as part of the Scientific Advice program is shared with patient representatives only with the express permission of the applicant. Applicants indicate permission to share information in the online application form for Scientific Advice.

Note that submission of the Patient Drug Information Form constitutes the applicant' agreement to share the completed form with patient representatives involved in the application.

Evolution of Patient Involvement in Early Scientific Advice

At this point in time, best practices for involving patients in Scientific Advice programs are not established. The process may evolve.