Patient Take-over, Make-over, or Roll-over?

The growing influence of the patient perspective is evident everywhere these days. It achieved initial official recognition in the European Medicines Agency's active interactions with patient groups dating back to the creation of the Agency, when it held dialogues with HIV patients. By 2000 patients had become full members of its Committee on Orphan Drugs, and a Patients and Consumers Working Party was created in 2006. The trend has been repeatedly confirmed by successive moves to give a voice to patients, and even, most recently, to give patients input into deliberations on specific medicines. EMA has always said that stakeholder groups bring a ‘real-life’ experience as well as specific knowledge and expertise to scientific discussions on medicines and on the impact of regulatory decisions. Nowadays, patients are routinely involved at the EMA as members of its management board and its scientific committees, and in consultations on disease-specific requests by scientific committees and working parties, as well as in discussions on the development and authorization of medicines and the preparation of guidelines.   In parallel, the European Patients Forum has pursued becoming "a driving force to advance patient empowerment and equitable patient access to care in Europe", through promoting patients’ organizations at national and regional level. Its mission is "to advance meaningful patient involvement in the development and implementation of health-related policies, programs and projects in the EU", and its personnel and policies are regular features of European discussions of medicines.   The European organization of patients with rare diseases, Eurordis, has similarly elevated itself to a role of major influence in European policy on medicines research and provision. It is currently recruiting candidates for the second edition of its Winter School on Scientific Innovation & Translational Research next March, which will give patient advocates deeper understanding of how pre-clinical research translates into real benefits for rare disease patients.   European Union-funded projects have also aimed at wider and more constructive patient engagement. COMPAR-EU is a project identifying, comparing, and ranking the most effective and cost-effective self-management interventions for adults in Europe. It focuses on SMIs within four high-priority chronic conditions: type 2 diabetes (T2DM), obesity, chronic obstructive pulmonary disease (COPD), and heart failure. EUPATI was a four-year project designed to educate and train patient representatives so they could become more effective advocates of patient interests in discussions with drug developers and regulators. And a successor project, the Patients Academy, is running a webinar in early September about patient engagement in the process of research and development of medicines, with an emphasis on how to make patient engagement possible for patients who may have more specific needs, such as people with dementia and children and young people living with a medical condition. Most recently, another EU-funded project, PARADIGM, is working towards "structured, effective, meaningful, ethical, innovative, and sustainable patient engagement" that can contribute to research priority setting, design of clinical trials and early dialogue.   The growing interest in personalized medicine has given the process an obvious boost, with numerous calls from all quarters for recognition of the need for patient engagement in new forms of diagnosis and treatment, and for a shift in the relationship between healthcare professionals and patients. As the action plan of the International Consortium on Personalized Medicines says, right up front, "Personalized medicine uses information specific to an individual to target therapeutic and prevention strategies more accurately, putting citizens and patients at the centre of healthcare and innovation".   The World Health Organization is backing the move strongly. A just-released video (https://www.youtube.com/watch?v=pj-AvTOdk2Q) emphasizes the importance of people-centred care: "Too many people are not informed or consulted in the care they receive", it says. It goes on to point out that in some countries, only one in three people have the risks of their treatment explained to them. One in five people have at least one unnecessary test. And up to 40% of health spending is wasted due to inefficiency, it says. The point it makes is that people live with their conditions 24/7, not only when they seek care. So healthcare services should be tailored to people's needs, and care should be provided in partnership with them, and not just handed to them.   It hasn't been plain sailing. The needs of patient groups for support has made them occasionally careless in the past over their choice of sponsors and backers, giving rise to criticisms that they risked becoming little more than the poodles of the drug industry. Recent correspondence in the BMJ highlighted concerns that "most patient organizations now see their interests through the same lens as their corporate funders. Those prepared to critique the for-profit basis on which the industry rests are underfunded and outnumbered at consultations with drug regulators, if they are heard at all." The tone reflects long-standing scepticism over the independence of patients' views-and not all sceptics have been entirely reassured by agreements on codes of practice and increased transparency from industry and patient groups.   Against that background, a new study on 'Benchmarking the Patient Movement 2018' (http://www.patient-view.com/bull-benchmarking.html) finds markedly greater impact on society than over the last half-decade: "The patient movement as whole has increased both its stability, and its forms of financing, since 2012. Today's patient groups are generally accepted by other healthcare stakeholders as suppliers of unique and valuable information into what life is really like for patients." But, it concludes, the patient movement remains fragmented, overly competitive, and its component patient groups continue to lack confidence about their role in national healthcare systems. "Patient groups believe their overall reputation to remain largely unchanged for the last six years-and sitting at a relatively low point," it says. "Furthermore, of the 22% of patient groups that have a relationship with regulators, only 40% believe regulators consider them 'very good' at fulfilling the needs of the patients and people they represent."   So forty years on from the start of the patient movement, its influence on drug policy is still limited: there has been no take-over of the direction of drug development. Its reputation has improved as it has become more mature, and its input is at least widely sought, even if not necessarily followed: so to some extent, compared with its earlier days, its moves towards transparency and clearer governance have helped a positive make-over. But until it can find ways of better leveraging its still widely-dispersed potential, its voice may be heard but not listened to-meaning that the drug development establishment will continue to call most of the shots, amounting, for patient groups, to a comprehensive roll-over.   Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium