Therapeutic advances become successful when research outcomes are meaningful for patients. Patients live with the day-to-day impacts of their disease, treatment and the trade-offs in potential adverse effects. Including patients and the public as stakeholders in medical and regulatory innovation provides a unique perspective, vital to generate data that promote positive changes in clinical practice. 

Why is structural patient and public involvement in drug development so important?

Dr Lisa Campbell, Medical Advisor at NDA, co-authored an article titled "The opportunity for greater patient and public involvement and engagement in drug development and regulation" published on 28th February 2023 by Nature Reviews Drug Discovery. The article highlights a number of patient and public involvement and engagement (PPIE) initiatives by regulatory agencies and regulatory science centres. In addition, the authors present some areas where further developments could be valuable in order to improve the uptake of new therapies and medical technologies. 

A successful PPIE process is an opportunity for all stakeholders. It starts with a clear strategy that should be timely implemented, meaning that early engagement is preferred. The most important aspect is that the contributions should be meaningful and serve a sincere purpose for the patients.

PPIE initiatives

The current initiatives made by different regulatory agencies:

FDA

  • The Patient Representative Program is one of the avenues for recruiting patients and caregivers with lived experiences of a condition, or medical device.
  • Patient Engagement Advisory Committee (PEAC) ensures that patients’ needs, and experiences are incorporated in FDA’s considerations on the regulation of medical devices.

EMA

  • Patient representatives are also members and alternates in different scientific committees They are appointed by the European Commission, where they are systematically involved in the core business of regulating the development, approval, and supervision of medicines.
  • Patient representatives of their own organisation - Representatives of patients and consumers organisations are also members of EMA’s Patients and Consumers Working Party (PCWP), which provides a forum for the agency to obtain the input of these stakeholders.

MHRA

  • The MHRA’s Patient Group Consultative Forum is a large pool of patients, their caregivers, patient groups and the public and the participants can attend workshops, provide feedback on draft documents and participate in surveys that inform the agency’s work.
  • Patient and public representatives sit on expert panels established by the Commission on Human Medicines to advise on issues associated with access to medicine.

Suggested developments

Patients have inherent knowledge of their experience with a disease and their voice is increasingly recognized in drug development.  The processes to engage patients and the public could however be more efficient and harmonised.  The article proposes:

  • For best output, PPIE should be embedded in the broader infrastructure of the organisation.
  • Continuously sharing of best practices, standardised methods of measuring and structured monitoring of key performance indicators.
  • Greater inclusion leads to more accurate trial results and better treatments for all the patients who need them and the population that will use the medical product once it is approved.

Dr Lisa Campbell observes:

“Science and innovation are paving the way into an incredible new world of drug development, changing the course of diseases, and curing the once incurable.

Patients should be embodied in the drug development process, right from the very point of conception of the idea for a new drug. In todays crowded competitive drug market it’s the patient input into clinical trial designs that could make the difference between a drug that makes it to the end of the drug development journey and one that does not.

Drug developers, like regulators, need to listen to patients and only in doing so will drug development ultimately be ‘successful’ with  patients receiving  medicines that make a positive contribution to their lives. The patient voice is a critical piece of the drug development puzzle, let’s not underestimate its importance!”

Author

Dr Lisa Campbell

Senior Director

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