his year, the Canadian Annual Meeting featured its first plenary session on patient involvement. Panelists were given the challenge of providing context to this evolving field and responded with examples of current patient involvement initiatives, collaborative partnerships, and other resources that support patient involvement and encourage best practices.

Key Takeaways

• Patients have applied, lived experiences and are uniquely positioned to enrich traditional drug development and regulatory processes with this knowledge.
• Patient involvement needs to be meaningful and established in partnership.
• Patient involvement in the drug lifecycle is relatively new and evolving.
• Early patient engagement provides an opportunity to inform decisions throughout the drug lifecycle and to establish meaningful patient outcomes.
• There is a need for guidance, engagement platforms, and tools to foster patient involvement across the broader drug sector.

Evolving for the Future

Both Health Canada and the FDA presented examples of how regulatory agencies have evolved over time in response to patient involvement. For example, Health Canada continues to involve patients in different ways, with its two most recent Scientific Advisory Committees on Health Products for Women and Oncology Therapies integrating patient member representatives into their structure. FDA provided an overview of its Patient-Focused Drug Development Initiative, including its enabling legislation (i.e., the Prescription Drug User Fee Act or PDUFA I-VI, or the 21st Century Cures Act), and how it has evolved over time.

Collaborative Partnerships

Health Canada and the FDA are also employing collaborative partnerships to assist in ongoing efforts toward establishing best practices, guidances, and tools that help support and facilitate patient involvement in drug development and regulation.

FDA’s Patient-Focused Drug Development Initiative provides an example of collaboration with patients through meeting series, guidance development, and transparency initiatives. These have helped shape the FDA’s discourse and relationship with patients into one that is more engaged and collaborative. In addition, guidance documents have helped establish an expectation for future patient involvement in drug development, and the meeting series has created a model that can be adopted by patients as a mechanism to engage with their regulatory authority.

From a patient perspective, there was a noted shift from disengagement to being valued collaborating partners, but the need for further progress and the removal of barriers was emphasized. The role of patients as collaborators and researchers identifying and fulfilling unmet needs was highlighted as were patient contributions to policy work led by regulatory authorities and health technology assessment organizations. The development of a Canadian Patient Group Pathway to Accessing Cancer Clinical Trials, adapting the American Clinical Trials Transformation Initiative (CTTI) model for the Canadian context, was one example of this collaboration. A vision of the future, where patients are recognized as people and experts with a lived experience that can add value to drug development was put forward by the patient representative on the panel, noting current ongoing challenges around compensation, recognition, and early involvement.

International Initiatives

International initiatives such as the SISAQOL and PROTEUS Consortiums are helping improve the quality of endpoints, analysis, collection, and reporting of patient-reported outcomes (PROs) within clinical trials and have developed supportive tools to assess and measure PROs, trial design, and data quality. Broader, global guidance being developed by ICH and CIOMS have integrated patients into their guidance development processes, ensuring that the patient voice is not only considered but is integrated and shapes best practices for patient involvement in the development and safe use of medications.