Several private-public partnerships, as well as government agencies have established models that reflect the benefit, and opportunities, for advocacy organizations and drug development sponsors to collaborate. The following are provided as references to support organizations to assess where collaboration may be the most pertinent for a drug development program.
The Center for Information and Study on Clinical Research Participation, Inc.’s (CISCRP) core mission is to provide accessible, relevant, useful, high-quality educational resources, programs and services that increase awareness and understanding of the clinical research process; recognize and appreciate the unprecedented gift of participation in clinical trials; enhance and enrich the participation experience for patients and their families; and promote engagement and partnership between clinical research professionals, patients and the public. CISCRP helps patients and their families locate ongoing clinical trials through its free searchclinicaltrials.org service and it helps professionals better understand study volunteer experiences through patient advisory boards, clinical trial journey workshops, and global surveys, like the Public & Patient Perceptions & Insights Study.
The Clinical Trials Transformation Initiative (CTTI) is a public-private partnership created in 2007 between the United States Food and Drug Administration (FDA) and Duke University for the purpose of identifying practices that will increase the quality and efficiency of clinical trials.
The Council for International Organizations of Medical Sciences (CIOMS) is an international, non-governmental, non-profit organization established jointly by WHO and UNESCO in 1949. CIOMS represents a substantial proportion of the biomedical scientific community through its member organizations, which include many of the biomedical disciplines, national academies of sciences, and medical research councils. CIOMS’ mission is to advance public health through guidance on health research including ethics, medical product development, and safety. CIOMS Working Group XI – Patient Involvement report, Patient involvement in the development, regulation and safe use of medicines 2022.
To help guide biopharmaceutical companies to high-quality patient-informed development, the US Food and Drug Administration (FDA) has issued a number of documents:
• Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data
• Assessment of the Use of Patient Experience Data in Regulatory Decision-Making
•Enhancing Benefit-Risk Assessment in Regulatory Decision-Making
• FDA Patient Listening Sessions
• Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience
At IHI, our core goals are to translate health research and innovation into tangible benefits for patients and society, and ensure that Europe remains at the cutting edge of interdisciplinary, sustainable, patient-centric health research. Health research and care increasingly involves diverse sectors. By supporting projects that bring these sectors together, IHI will pave the way for a more integrated approach to healthcare, covering prevention, diagnosis, treatment, and disease management
Project factsheets | IHI Innovative Health Initiative (europa.eu)
Notable guidances, recommendations, and frameworks:
• IHI Paradigm: Active engagement of patients in research & development (formerly IMI Paradigm)
Patients active in research and dialogues for an improved generation of medicines: advancing meaningful patient engagement in the life cycle of medicines for better health outcomes
• IHI PREFER: Patient Preferences in benefit-risk assessments during the drug life cycle
The ICH Reflection Paper on Patient-Focused Drug Development (PFDD) identifies key areas where incorporation of the patient’s perspective could improve the quality, relevance, safety, and efficiency of drug development and inform regulatory decision-making. It also presents opportunities for the development of new ICH guidelines to provide a globally harmonized approach to the inclusion of the patient’s perspective in a way that is methodologically sound and sustainable for both regulated industry and regulatory authorities.
The MRCT Center has four primary areas of focus that address pressing issues in multi-regional clinical trials.
1. Clinical Trials & Research
Tackling critical issues in the ethics, design, conduct and oversight of clinical trials to maximize safety and rigor, we develop practical and pragmatic guidance, resources and tools.
2. Capacity Building
Promoting convergence of local requirements and internationally accepted best practices, we work with country-specific and local stakeholders to develop culturally-relevant solutions including training, policy development and support to regulators, research ethics boards and clinical research personnel.
3. Quality & Transparency
Engaging the participant in understanding clinical trial and data solutions, we seek to improve the quality of clinical trials and trial reporting, leading to increased transparency in and value of clinical research.
4. Patients & Participants
Supporting and preparing individuals considering participation in clinical research, the MRCT Center provides educational materials to various audiences, including participants, friends/family members, health proxies, parents/guardians, and children.
PCORI helps people make informed healthcare decisions and improves healthcare delivery and outcomes, by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community.
PFMD’s goal is to improve global health by co-designing the future of healthcare for patients WITH patients. Its mission is to bring together initiatives and best practices that integrate the voice of the patient thereby speeding up the creation and implementation of an effective, globally standardized framework – that involves patients as partners – as well as the necessary tools, services and support to allow the adoption of the framework by various stakeholders. PFMD, as the patient engagement platform, has all ingredients to be nurtured & matured: Agile, Holistic, Inclusive, non-Competitive, to take patient engagement to the next level.