First meeting of the CIOMS Working Group XI: Patient involvement in the development and safe use of medicines 19-20 April 2018, Geneva Mary O’Hare, Roche This presentation is being made on behalf of the Policy team within the GPQO team (Simoni Lopes Brand, Mary O’Hare, Sarah Cloke, Lorna Bailey, Serena Pulcini)

Scope Concept paper - very broad scope - attention will need to be targeted in a few key areas to have an impact. Patients have been historically and consistently the most under-represented group across the whole drug development, approval and post- approval process, and yet they are, of course, key. An increased patient-centric approach fits very well with development of the personalised healthcare concept.

Issues to be considered by the Working Group: Patient involvement in drug development Patient involvement in drug pharmacovigilance and benefit-risk management Use of digital technologies and social media as a source of PV information

Patient involvement in drug development Identification of patients/patient groups who wish to be involved in drug development. Patients are individuals. Educate patients in the importance of reporting AEs/side effects, ensure they understand the benefits. Encourage patients to report side effects during a clinical trial – often patients are terrified they will lose their place on the trial. Facilitate patient reporting without the influence of a healthcare professional, as doctors can under- & over- estimate side effects in terms of importance or relevance to a patient.

Patient involvement in drug pharmacovigilance and benefit-risk management Patient groups can be an effective way to obtain a patient perspective / input: Access to common patient experiences in terms of disease, treatments and side effects Ability to mitigate some of the concerns around data privacy for individual patients. Provide vital input into the discussion regarding acceptable benefit - risk balance for patients. Provide clear & effective advice to patients and their physicians on how to manage adverse events. Learn from previous experience, such as the HIV story - as people with HIV lived longer and the disease became more treatable, management of side effects became more important.

Social media - major challenge with: Use of digital technologies and social media as a source of PV information Social media - use computer algorithms for trending and identification of potential signals. Avoid processing individual potential AE report as an ICSR in the traditional way. Social media - major challenge with: Quality of data, Ability to identify duplicates, Lack of opportunities for follow-up, Data privacy issues (consider recent Facebook scandal) The power of social media to identify new safety issues or new aspects of known safety issues remains very unclear.

Potential Priorities for this Working Group: Improving the effectiveness of risk mitigation activities Increasing understanding of and adherence to labelling requirements Encouraging participation in data collection via organised schemes (eg registries) Increasing understanding of the utility of unstructured social media type data in PV

Concerns: Patients are heterogeneous and this needs to be considered in interpreting their input. Factors may include background/ history, local healthcare system, acute vs chronic disease, cultural considerations, geographic location, age etc.. How can any guidance take all of these issues into account? Ability to successfully balance high quality, controlled data with high volume, unstructured data (eg registries versus social media). Continuing exponential rise in volume of AE reports - has this improved the patient experience & AE management outcomes?

Summary: Industry very much welcomes more patient involvement and more focus on patients in all aspects of drug development and marketing, but is concerned to ensure: Changes really add value Expectations are managed (ie cannot address all needs / priorities for each individual patient)

Doing now what patients need next