Patients as partners in medicines development – the EUPATI project

As the head of EUPATI, a consortium of organisations working to put patients at the heart of drug R&D, Jan Geissler has seen first-hand how bringing patients and industry together can help keep research on track and benefit all.

In 2001, at the age of 28, I was diagnosed with Chronic Myeloid Leukaemia (CML), a rare cancer that usually affects people of an average age of 65. What I learned about the disease looked pretty grim: a bone marrow transplant was the standard treatment for young patients like me, with a treatment-related mortality of about 25 per cent and a probability of severe long-term complications of another 20 per cent.

As I have the good fortune of speaking English well, I found internet communities in Asia and the US talking about trials with a new drug demonstrating promising early results. I identified a clinician about 400km away from my home who was running a small phase I/II clinical trial assessing the combination of two investigational drugs.

I was lucky and this treatment worked well. But I realised that no information was available in German for patients in a similar situation, so I started to translate scientific research abstracts into German lay language and set up an online blog to publish them, Leukaemie-Online.de. Today, this is one of the most frequented online communities for German-speaking leukaemia patients.

Research then became a key theme of my patient advocacy work. I became one of the founders of the European Cancer Patient Coalition in Brussels, working to introduce more patient-centricity to the revisions of the EU Clinical Trials Directive or other EU health policy, and set up the worldwide CML Advocates Network, which connects leukaemia patient organisations. These groups are collaborating closely with health professionals, researchers and industry, to provide understandable information to patients, to make trial design and informed consent more patient centric and to empower patients to take responsibility for their own lives.

Over the years, I have evolved from being a patient and ‘subject’ of research to become a respected partner of those designing and running medicines research. With only a business degree and working in media and telecommunications, I learned the hard way how clinical trials operate, and how to match patients’ needs with technical and regulatory realities.

European Patients’ Academy on Therapeutic Innovation

In 2011 I was asked by a group of organisations to co-ordinate a proposal to the EU- and European Federation of Industries and Associations (EFPIA)-funded Innovative Medicines Initiative. This was to manage a project developing training courses and educational materials on medicines R&D for patient advocates and the health-interested public – to which I agreed enthusiastically.

The newly-formed consortium won the bid and, since February 2012, I have been leading the European Patients’ Academy on Therapeutic Innovation (EUPATI), a five-year public-private partnership of 30 organisations including patient advocacy groups, NGOs, academia and industry.

“Patient and public involvement in all aspects of medicines research and development has an ever-increasing role, and is having an effect at all stages of the research cycle”

Patient and public involvement in all aspects of medicines research and development has an ever-increasing role, and is having an effect at all stages of the research cycle for medicines and clinical research – from commissioning and funding research, clinical trials, health technology assessment (HTA), associated regulatory processes through to the dissemination of information to clinicians, patients and patient organisations.

Patient involvement has grown rapidly from an advisory activity to active involvement as equal partners in research design, implementation and oversight. Patients bring invaluable knowledge of a lived experience of illnesses and treatments and, importantly, shape the process to re-focus on real patients’ needs. The goal of patient involvement is not just liberating; it aims to improve public health and to make everyone involved in medicines R&D focus on what patients really need.

While patient involvement is gathering momentum, patient organisations are struggling to provide enough adequately trained patient advocates to meet the needs of the research community. EUPATI, led by the Brussels-based European Patients’ Forum, aims to bridge this gap and fill the information, education and training needs and enable effective patient involvement across Europe. Well-informed patients and patient organisations can be effective advocates and advisors.

Patient Expert Training Course launched

The EUPATI project is now approaching mid-term and much has been achieved. Its Patient Expert Training Course, a 250-hour certificate training course for patient advocates, covering pre-clinical development, clinical trials, pharmacovigilance, risk-benefit assessment, health technology assessment and regulatory affairs, was launched on 6 October 2014, with 53 patient advocates from 21 countries taking part.

Most of the course will take place via e-Learning, complemented by two face-to-face course weeks. Subsequently, EUPATI will develop an internet library and toolbox in seven languages, and national platforms are being initiated in 12 European countries to facilitate education at national level. Three public conferences have consulted the community on its needs, as did EUPATI-run surveys and focus groups in different European countries.

“Objectivity, transparency and independence are vital prerequisites to ensure EUPATI achieves its goals”

Being a public-private partnership of patient organisations, academia, NGOs and industry does not come without challenges. Objectivity, transparency and independence are vital prerequisites to ensure EUPATI achieves its goals and is accepted and valued by both the patient community and the wider public.

The role of industry is under special public scrutiny, given its key role in developing new medicines, but also with regard to commercialising products when development finishes. To address this, EUPATI has established a robust governance structure, led by major umbrella patient organisations, as well as a multidisciplinary project advisory board, a regulatory advisory panel and an ethics panel, comprising experts in ethics, media, medicines agencies, evidence-based medicine institutions and patient advocacy groups. This is complemented by a transparent policy on disclosure of interests.

EUPATI is not educating patients and the public about disease-specific issues or therapies. Its focus will be on the general process, its complexity and all issues related to new medicines development, as well as regulatory issues in general. Indication-specific information will only be used to illustrate examples on specific issues of medicines development.

Barriers to overcome

But will academic research and industry be ready to involve patients in their R&D processes when the first EUPATI trainees complete their courses in Autumn 2015? While there is a lot of buzz around patient-centricity, much needs to be done to really involve patients in R&D in a systematic and meaningful way.

A workshop of EUPATI member organisations in July 2014 revealed important insights into benefits and barriers, challenges and solutions, codes of conduct, and potential guidelines on patient involvement in industry-sponsored medicines R&D. A total of 22 best practice cases from areas like rare diseases, paediatrics, oncology, neurological diseases and HIV illustrated real-life examples of where patient involvement in R&D led to tangible benefits, and how perceived and real barriers were overcome. It was clear how important it is to share, collaborate, guide and advocate (externally, as well as within companies and institutions) in order to move patient involvement in R&D from theory to widely adopted practice.

I was lucky to access an early phase trial that later turned a deadly cancer into a chronic disease, but such an outcome is the exception rather than the rule in most serious conditions. Even in CML, cure is not yet an option for most patients.

However, patient advocates in different disease areas are working hard with the professional community to identify better trial designs, research schemes and collaborative models that will provide real impact and improvements in patient outcomes.

There is still too much waste in the system, too much duplication of work, too much energy spent on research that does not meet what patients need. Patients have a lot to contribute, and EUPATI is working hard to empower them to do so.

About the author:

Jan Geissler holds a business degree. He worked in media and telecommunications before taking charge of the European Patients’ Academy on Therapeutic Innovation (EUPATI) in 2012.

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