Involving patients in medicines regulation

Medicines are developed to benefit patients, yet historically, their views have been overshadowed by the views of healthcare professionals. Regulators have tended to follow this pattern too, but are now committed to fully involving patients in decision making wherever possible. The move of patients from being a passive voice in healthcare to an active one has helped bring about this change. Patients have not only become better informed, but also increasingly vocal in what they desire in terms of healthcare. A number of patient groups have formed worldwide alliances to represent their views.

Patients have been involved in the development of new regulations around the world, but not in a consistent manner. The AIDS crisis led to considerable patient pressure being placed on regulators in the US to ensure that new medicines were developed. Similarly, the legislation concerning orphan medicinal products for rare diseases has been greatly influenced by the work of patient groups in the EU, US and other regions.

Historically, the US has been seen as further ahead in recognising the views of patients in medicines regulation. The FDA has a section called the Patient Network, whose role is to ensure that the perspectives of patients, family members, caregivers, and patient advocates are not overlooked during the review process. The FDA also put in place a Patient-Focused Drug Development (PFDD) program to systematically involve patients and this features regular meetings for different disease areas. The European Medicines Agency (EMA) has interacted with European patients since it was founded in 1995, but is seeking to formalise this type of engagement. The Agency works via a network of over 30 European patients’ and consumers’ organisations that must fulfil certain eligibility criteria. There is a framework to direct how interactions between regulators and these groups take place. Currently, patients can be asked to become involved in a variety of activities, ranging from taking part in scientific advisory groups to being involved in the preparation of guidelines. The EMA Human Scientific Committees’ Working Party with Patients’ and Consumers’ Organisations (PCWP) was established to provide recommendations on all matters of direct or indirect interest to patients.

In September 2013, the EMA held a workshop in London that brought together representatives of patients, consumers, healthcare professionals and the pharmaceutical industry with members of the EMA’s scientific committees and staff. The aim was to get a better understanding of how patients could become further involved in the assessment process for medicines, particularly when it came to benefit and risk evaluation. The EMA appears committed to further involving patients in its activities and will be using the outcomes of the workshop to make further improvements to its processes. Presently, it produces an annual report on its involvement with patients that includes a satisfaction questionnaire.