Helping Patients in Patient-Centered Approaches to Health

The growing insistence on making healthcare more patient-centered—Europe is currently awash with declarations of intent along those lines—is generating a new level of interest in helping patients (or even citizens) to meet the expectations that the new orthodoxy is creating. After all, there is little logic in putting the patient at the center if the patient has no idea what to do once he or she is there. The world of clinical evaluation is among the most committed to such an approach, for a raft of reasons ranging from efficacy to ethics. In late October, the European Medicines Agency recommended granting a marketing authorization under exceptional circumstances for a treatment for erythropoietic protoporphyria—in a procedure that, for the first time ever, involved patients directly in the assessment of benefits and risks of a medicine. The EMA's scientific committee made its recommendation after hearing from patients treated with the product on a compassionate-use basis, who reported improvements to their quality of life. It is just one manifestation of a trend that is being driven both by new technologies—permitting more individualized treatment—and by the increasing challenges that many trial sponsors encounter in recruitment of subjects. At the same time, patients and patient groups are also taking a more assertive stance in some therapy areas—inspired by the successes of the AIDS/HIV campaigners of earlier decades—and demanding access to innovations such as the new generation of hepatitis C treatments.

But closer or wider or deeper or more constructive involvement of patients—in clinical trials or in other areas of medicines development and delivery—depends itself on how far patient engagement and alertness and goodwill (and a lot of other group characteristics) are developed across the broad patient or citizen population.

Patient organizations are taking these new responsibilities seriously. The European Patients Forum is upgrading its programs in capacity building for its members, "acknowledging a large variation in levels of skills of patient organization representatives." It is adapting proven materials and resources to a ‘tailor-made’ approach for national or thematic patient organizations, aimed at improving their organizational capacities and advocacy skills, so they are better equipped to convince policy-makers with strong and well documented evidence, and to negotiate with decision-makers. In Romania and Hungary, over the last twelve months, local patient organizations were coached in strategic planning and needs assessment, and similar programs are nearing completion in Slovakia and Bulgaria.

Regulatory authorities too are active in their own domains. The EMA's breakthrough inclusion of patients in its recent product assessment was no one-off event. It is boosting its own system of involving patients and consumers in its work by ensuring that it can take into account patients’ views on a more regular basis. This is in line with work program recommendations for integrating patients’ values in the benefit/risk assessment, and is a further reflection of the agency’s emphasis on stakeholder involvement.

Similarly, the rapidly-developing health technology assessment operations in Europe—ever more closely involved in the discussions of medicines access and medicines development—are accepting and even soliciting wider stakeholder involvement, including from patients. The EUHTAnet, one of the principal European initiatives in seeking clearer and more consensus-based EU-level approaches, now operates a stakeholder forum that encompasses not only patients, but also industry.

Another parallel track is being pursued by a consortium bringing together patients, regulators, academia and industry, with the aim of training patients as experts in drug development. The European Patients’ Academy, or EUPATI as it is known (declaration of interest: this writer sits on the advisory board for the project), has just launched a training course for 53 participants. It is creating a series of educational programs designed to meet the needs of patients and patient representatives and to impart expert-level knowledge about the whole spectrum of the medicine research and development process. "The course is specifically designed to meet the needs of participants who are motivated with the skill set to acquire and apply this expert-level knowledge,” said Jan Geissler, the EUPATI Director.

One of the aims is to help patients to engage in representing a patient perspective in the medicine development process, in interactions with scientific committees, health technology assessment agencies, industry, regulatory bodies, academia, and other relevant stakeholders. The ambition is for patient experts also to help raise awareness about patient involvement in medicines research and development within the patient community and among the general public.

As with personalized medicine—where multiple initiatives are concurrently aiming at defining the best way ahead—the bid to make a reality of patient-centered care from the patient's point of view is at present a wide-open field with many players pushing competing (and sometimes conflicting) agendas. The hope must be that the intense focus now being directed at the issues raised by putting patients at the center does deliver results – rather than leaving them scattered around the perimeter of healthcare planning.