“Meaningful patient involvement in research and policy is key to developing personalised medicine in a way that will truly benefit patients” – European Patients’ Forum
Personalised medicine is an important emerging area for patients with chronic diseases and has potential to bring about fundamental changes to improve the delivery of healthcare. Patients welcome the development of personalised medicine, as it could result in improvements in the management of chronic disease through earlier and more accurate diagnosis by enabling treatments to be tailored for better effectiveness: the right drug at the right dose for the right patient at the right time. It could also lead to the development of personalised cell and gene therapies.
The role of personalised medicine is part of personalised health solutions, including all aspects of patient-centred chronic disease management. This implies a holistic approach to chronic disease management: treating the “whole person” and their family and carers, and considering physical and mental health as well as social aspects. In this sense, medicine has always been “personalised”. The term “stratified medicine” is in fact preferred by some patients, as it explains more accurately what personalised medicine is all about.
Fighting Blindness believes that personalised medicine should be developed to provide equitable access to safe, high-quality, patient-centred treatment and address unmet medical needs. In order to achieve this goal, the development of personalised medicine must be based on the principles of patient-centred healthcare.
1. Patients must be equal partners in healthcare decisions. The patient’s role is undergoing a transformation as health systems increasingly move towards a patient-centred model of healthcare. With personalised medicine, a collaborative approach between patient and health professional is crucial in order to make the treatment decision that is best for the patient.
2. Health literacy and high-quality, accessible information is key to empowering patients to participate in managing their health in equal partnership with the health professional. Patients groups across the EU are calling for a strategy on information to patients, encompassing a programmatic commitment to health literacy, to equip European patients and citizens as they meet these future challenges.
3. On the health professionals’ side, professional training will be needed to equip them with the skills to communicate with patients and enable collaborative decision-making.
4. When it comes to translating scientific developments into clinical practice and patient care, a number of legal, ethical, political and economic issues must be resolved. A clear, patient-centred regulatory framework is needed to encompass personalised medicines and accompanying diagnostic devices, in order to ensure timely and equitable access for patients; avoid discrimination of patients on the basis of genetic information; and monitor and regulate any commercial use of genetic/genomic information.
5. One concern for Fighting Blindness and an example of the above is the growing unregulated market in direct-to-consumer genetic testing and risk profiling services, the results of which are variable and often inaccurate, while their clinical relevance is questionable. It is crucial that patient organisations are involved in the development of policy, standards and guidelines for products and services, both at EU and national level. This is why Fighting Blindness launched the Target 3000 programme: to ensure that Irish patients with inherited retinopathies can have equitable access to genetic testing that will allow them to have better information on their conditions. Through Target 3000, this will be carried out in an effective way, ensuring that results are transmitted accurately and stored in a safe environment, and that these results can be utilised – with the consent of the patient – to further the research effort.
6. Another concern is access to available treatment; there are presently huge disparities across the EU in access to healthcare, and in the standards of care in many chronic diseases. While it could help improve the long-term sustainability of healthcare systems, innovation tends to be costly. EPF believes healthcare must be based on the fundamental principles of equity and solidarity, and personalised medicine must not result in the exacerbation of health inequalities. Innovative treatments should be made available and affordable to all patients, not only those who can pay for them. It will also be crucial to ensure that genetic information will not be used as a tool for discrimination in access to treatment.
7. Meaningful patient involvement at all levels is key to success. To ensure that personalised medicine is developed with patients’ needs at the centre, patients and their organisations need to be involved in research and development, and at all levels of health policy-making. Patient involvement throughout the drug development process, from the “idea” stage to the proven intervention, is key to ensure that the results of research are relevant to patients. Patients’ experiential knowledge has been shown to improve the results of clinical research and should be used more effectively; further, patient involvement can help improve flagging participation rates, as well as build public trust and confidence in clinical trials and in research generally.
8. Patient involvement in clinical trials must be enshrined within the EU legislative framework and supported by appropriate capacity-building programmes.
9. To ensure equitable access to personalised therapies, patients should be involved in Health Technology Assessment, prioritisation and reimbursement processes at national level. Patients’ evidence needs to be
10. There needs to be more European cooperation in the field of HTA and for principles and guidelines to be developed at EU level for good governance, transparency and stakeholders’ involvement in HTA.better valued and incorporated at different stages in the HTA process.
Fighting Blindness would like to thank the European Patients’ Forum for providing the background information to this document.