Frameworks

Patient testimony in the regulatory process expands in the second half of this decade and the Food and Drug Administration (FDA)Food and Drug Administration’s (FDA) evaluation of new medicines (United States). During clinical trials, researchers study whether new medicines are safe and effective for patients and whether the medicine’s benefits outweigh the risks.
www.fda.gov and the European Medicines Agency (EMA) EMA is a decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU www.ema.europa.eu join forces!

In addition to the European Medicines Agency EMA recently revising their original framework for interaction between the agency and patients and consumers and their organizations (2014) :

• 2015: The FDA launches the Patient Engagement Advisory Committee (PEAC)Patient Engagement Advisory Committee (PEAC) – Food and Drug Administration (FDA) committee providing advice to the Commissioner or designee on complex issues relating to medical devices, the regulation of devices, and their use by patients.
  www.fda.gov/patients to provide advice relating to medical devices . CoPI: Patient Preference Information (PPI)PPI: Qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. is established to describe this patient engagement. PPI framework and guidance released for the Center for Devices and Radiological Health (CDRH)(CDRH) recognizes that scientists, clinicians, device developers, and regulators play critical roles in evaluating and communicating the benefits and risks of medical devices. However, only patients live with their medical conditions and make daily choices regarding their health care. Their voice and perspective are critical to understanding the impact of medical devices.
  www.fda.gov/about-fda/fda-organization medical device decision-making.
• 2016: FDA and the EMA create a new workgroup on patient engagement called the FDA/EMA Patient Engagement ClusterWork group that allows FDA and EMA to share best practices involving patients along drug and biologic regulatory lifecycles. Information that is discussed is covered by confidentiality agreements signed by the FDA and EMA.
  www.fda.gov/patients to share best practices involving patients along with drug and biologic regulatory lifecycles.
• 2017: FDA launches Patient Engagement Collaborative The Patient Engagement Collaborative (PEC) is a group of patient organizations and individual representatives who discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions at the FDA. The PEC was established by the FDA and the Clinical Trials Transformation Initiative (CTTI), a public-private partnership that brings together organizations and individuals representing academia, clinical investigators, government and regulatory agencies, industry, institutional review boards, patient advocacy groups and others to develop evidence-based solutions to clinical research challenges. www.fda.gov/patients with the Clinical Trials Transformation Initiative (CTTI)The FDA, together with the Clinical Trials Transformation Initiative, established a group of patient organization and individual representatives to discuss topics focusing on enhancing patient engagement in medical product development and regulatory discussions at FDA.
  www.ctti-clinicaltrials.org/ to develop guidance materials for including patients in the R & D continuum. This collaborative was modeled after the European Medicines Agency (EMA)’s Patients’ and Consumers’ Working PartyEMA is a decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU. The Patients’ and Consumers’ Working Party (PCWP) provides a platform for exchange of information and discussion of issues of common interest between EMA and patients and consumers. www.ema.europa.eu/en/committees.
• 2017: EMA involves young people in their activities and establishes the principles for the involvement of young patients, consumers, and their carers, in the Agency’s scientific committees and working parties in a consistent and efficient manner .
• 2017: EMA begins holding public hearings to give European citizens a voice in the evaluation of the safety of medicines and empower them to express their views on issues related to the safety of certain medicines and the management of risks.
• 2017: EMA expands opportunities to acquire patients’ perspectives on benefit/risk considerations within the Committee for Medicinal Products for Human Use (CHMP)(CHMP) is the European Medicines Agency’s (EMA) committee responsible for human medicines. The CHMP replaced the former Committee for Proprietary Medicinal Products (CPMP) in May 2004.www.ema.europa.eu/en/committees meetings.

CoPI: Regulatory reviewers, working groups, advisory panels, Patient Preference Information (PPI)

Tools: Frameworks – regulatory; Guidance Documents – patient engagement

Context: Regulatory process, clinical trials, Research & Development, health technology assessment (HTA), decision aids, young adults