LONDON – Moves to increase patient involvement in drug development will ramp up in the next few months as the European Patients' Academy on Therapeutic Innovation (Eupati) enrolls 60 new students to its expert training course and launches an online toolbox of educational resources for patient advocates.

Patient power has been on the rise for some time but to date the involvement of patients has revolved around their experience of disease. Now, the aim of Eupati is that patients become experts in the process of drug development – with the knowledge required to be involved along the chain from setting the research agenda to the commissioning of new drugs – and with the confidence to change and improve the system from within.

Among other aspects, students starting the 14-month Eupati training course in September will learn how to set up and run clinical studies and give feedback on existing protocols, in a way that is informed both through their experience as patients and by an understanding of trial design.

The trainees also will study statistics, evidence-based medicines, ethics, regulatory affairs, pharmacovigilance and the principles and practice of health technology assessment.

The overall effect will be to "raise the bar of what a patient advocate is, and what a patient advocate can be," explained Jan Geissler, director of Eupati. The experts training program, the educational material and other initiatives planned by Eupati "will hopefully translate into a new paradigm of increased patient involvement across the entire medicines research and development process," Geissler said.

While patient engagement has been gathering pace, there remains a tendency to see patients as a resource to be tapped as and when required, rather than as equal partners with a say in the process.

Eupati's ambition is to change the nature of patient involvement, ending the lip service that is paid to the importance of their contribution and giving patients the means to bring about change.

In the highly regulated environment of drug development, patients can only become meaningfully involved if they understand the system, Eupati said.

The need for greater involvement of patients was acknowledged by the FDA and EMA in June when they agreed to establish a new collaboration to share experience and best practices in involving patients in development, evaluation and post-authorization activities.

In a joint statement, the two agencies noted that patients in the U.S. and Europe are well informed and expect that their voices are heard by regulators in the way studies are designed and in the assessment of the benefits and risks of specific medicines.

"Involving patients in the evaluation discussions adds meaningful perspectives to the process," the statement said. Under their collaboration, the EMA and FDA aim to expand patient input during the regulatory process, for example, to better understand how patients view the quality, safety and efficacy of medicines.

The EMA has patients' representatives sitting on its committees and currently is running a one-year trial in which it is seeking patients' opinions in the assessment of marketing applications. Toward the end of last year Clinuvel Pharmaceuticals Ltd.'s Scenesse (afamelanotide) treatment for extreme sensitivity to sunlight became the first example of the EMA, which heard testimony from sufferers of a disease when making the final assessment of a drug. (See BioWorld Today, Oct. 28, 2014.)

In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) has been making moves to increase patient involvement over the past three years and in September the first patient representative, British wheelchair fencing champion Matthew Campbell-Hill, will become a non-executive director of the agency.

Last month, the MHRA and the UK Bioindustry Association issued a report on a joint conference held to consider further moves to increase patient involvement. One of the major conclusions was that the key to increasing efficiency in drug development is to put the patient at the center. "Consulting and engaging patients and enabling them to be active partners creates the logic for process improvements to reshape clinical development," the report said.

In a notable example of how patient power is starting to influence regulatory processes, the first set of drug development guidelines to have been drawn up by a patients' group are in the last stages of being finalized by the FDA. "Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment," which currently are open for comment, originally were written and submitted to the FDA by Patient Project Muscular Dystrophy (PPMD).

Among other considerations, PPMD has forced the FDA to acknowledge it may not be ethical or appropriate to run placebo-controlled trials in Duchenne and that historical controls are sufficient. The patient group also has called on the FDA to recognize that those living with Duchenne have a different tolerance of risk and that the focus should be on endpoints that are meaningful to patients, rather than on clinically meaningful outcomes that are defined by clinicians.

A WIDER EDUCATION PROGRAM

As patient engagement grows, so does the need for patient experts. Eupati was set up to deliver education and training across Europe to ensure a supply of advocates who are equipped to participate on equal terms with clinicians, academics, company researchers and regulators. Although funded by the European Commission and supported by the pharma industry, Eupati is run by patients' organizations.

Three years' on from its launch, Eupati is starting to make a real impact, enabling patient involvement in R&D in a "systematic and meaningful way," according to a review of progress to date by Geissler and co-authors in Medicines Development Sciences.

While a second cohort of 60 students embarks on the experts training program next month, in December the first cohort of 47 will graduate as qualified patient experts. Around the same time, Eupati will launch its online toolkit, available in seven European languages, which it hopes will be used by 12,000 patient advocates.

Eupati also has fostered the formation of six national networks to engage with regulators, industry and clinicians at a local level, with a further six networks planned.

The academy's next goal is to launch a wider education program to increase awareness of the process of drug discovery and development among 100,000 members of the general public.