Abstract
The purpose of medicines is to improve patients' lives. Stakeholders involved in the development and lifecycle management of medicines agree that more effective patient involvement is needed to ensure that patient needs and priorities are identified and met. Despite the increasing number and scope of patient involvement initiatives, there is no accepted master framework for systematic patient involvement in industry-led medicines research and development, regulatory review, or market access decisions. Patient engagement is very productive in some indications, but inconsistent and fragmentary on a broader level. This often results in inefficient drug development, increasing evidence requirements, lack of patient-centered outcomes that address unmet medical needs and facilitate adherence, and consequently, lack of required therapeutic options and high costs to society and involved parties. Improved patient involvement can drive the development of innovative medicines that deliver more relevant and impactful patient outcomes and make medicine development faster, more efficient, and more productive. It can lead to better prioritization of early research; improved resource allocation; improved trial protocol designs that better reflect patient needs; and, by addressing potential barriers to patient participation, enhanced recruitment and retention. It may also improve trial conduct and lead to more focused, economically viable clinical trials. At launch and beyond, systematic patient involvement can also improve the ongoing benefit-risk assessment, ensure that public funds prioritize medicines of value to patients, and further the development of the medicine. Progress toward a universal framework for patient involvement requires a joint, precompetitive, and international approach by all stakeholders, working in true partnership to consolidate outputs from existing initiatives, identify gaps, and develop a comprehensive framework. It is essential that all stakeholders participate to drive adoption and implementation of the framework and to ensure that patients and their needs are embedded at the heart of medicines development and lifecycle management.
Highlights
Problem StatementDrug development times are around 10 to 15 years[1,2] and costs to bring a single new therapy to market are substantial.[1,2,3] From the industry perspective, not putting the unmet medical needs of patients first, early in the development process, can lead to wrong priorities, wrong decisions on research design, and potentially costly late-stage failure
Clinical or contract research organizations tasked with operational aspects of clinical trials are generally isolated from patients and patients’ needs
Selection of the term patient involvement rather than patient empowerment or patient engagement is deliberate and intentionally captures the central role that patients should play in medicines development and lifecycle management
Summary
Drug development times are around 10 to 15 years[1,2] and costs to bring a single new therapy to market are substantial.[1,2,3] From the industry perspective, not putting the unmet medical needs of patients first, early in the development process, can lead to wrong priorities, wrong decisions on research design, and potentially costly late-stage failure. Patients have a role to play alongside all other stakeholders in determining intended outcomes and priorities, acceptable uncertainty, as well as benefit/risk and value of a medicine Their recommendations and conclusions may be different from those of regulators, payers, academic researchers, other health care professionals (HCPs), and industry,[10] making it even more important that these opinions are well understood by all those making decisions. Serving patients requires a deep understanding of their medical condition, especially in terms of the challenges they face in everyday living, their goals, disease symptoms and side effects of therapies, and unmet needs in terms of therapy and quality of life. Once the needs are clearly understood, all stakeholders—including industry, regulators, patients, patient associations and advocacy groups, purchasers of medicines (including pharmacies and hospitals), HCPs including academic and community-based researchers, physicians and nurses, politicians and legal advisors, health technology assessment (HTA) agencies, and topic-related think-tanks—can work together to develop practical and implementable solutions and achieve more meaningful outcomes
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