As regulatory and health technology assessment (HTA) bodies put increasing emphasis on identifying patients’ perspectives to inform their decision-making, a similar move is happening in the pharmaceutical industry from developing medicines for patients towards doing this with patients. This means including representative patients’ perspectives in critical decisions throughout the medicine lifecycle, from early development through product conceptualisation, development strategy, clinical study design and conduct as well as informing regulatory and payer submission dossiers and post-launch product extension or improvement cycles. Tools and techniques to explore needs, experiences, and preferences of patients include participatory collaboration as well as research methods. To provide consistency and meaningfulness for product development and commercialisation, a well-formulated strategy needs to guide the planning of such activities starting from early research phases and applied concomitantly to the entire product life cycle. Transforming a company to being patient-focused requires significant change management. This includes transparent procedures, communication, guidance, training, and quantitative as well as qualitative measurements of performance indicators, reflecting the impact of patient involvement and facilitating learning by industry and patients. Doing so, helps strategically equip products to demonstrate value in HTA, and may lead to resource improvements through more efficient clinical trials, and greater success in commercialisation.

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Patient Involvement Throughout the Medicine Life Cycle: A Pharmaceutical Industries’ Perspective

  • Anke-Peggy Holtorf,
  • Michaela Dinböck,
  • Alexandra Moutet,
  • Elin Haf Davies

摘要

As regulatory and health technology assessment (HTA) bodies put increasing emphasis on identifying patients’ perspectives to inform their decision-making, a similar move is happening in the pharmaceutical industry from developing medicines for patients towards doing this with patients. This means including representative patients’ perspectives in critical decisions throughout the medicine lifecycle, from early development through product conceptualisation, development strategy, clinical study design and conduct as well as informing regulatory and payer submission dossiers and post-launch product extension or improvement cycles. Tools and techniques to explore needs, experiences, and preferences of patients include participatory collaboration as well as research methods. To provide consistency and meaningfulness for product development and commercialisation, a well-formulated strategy needs to guide the planning of such activities starting from early research phases and applied concomitantly to the entire product life cycle. Transforming a company to being patient-focused requires significant change management. This includes transparent procedures, communication, guidance, training, and quantitative as well as qualitative measurements of performance indicators, reflecting the impact of patient involvement and facilitating learning by industry and patients. Doing so, helps strategically equip products to demonstrate value in HTA, and may lead to resource improvements through more efficient clinical trials, and greater success in commercialisation.