Andrzej Rys, Health Systems and Medical products and Innovation Director at the European Commission, opened the EFPIA Health Collaboration Summit 2016, from 9-10 November, by outlining the challenges faced currently by health systems, including demographic pressures. He stressed that it was necessary to examine whether we spend our resources wisely in the healthcare sector and underscored the need to identify waste. Mr Rys said that the EU understands the value of health sector not only for the wellbeing of citizens but also to the overall economy and society. It was, he noted, the only sector that did not lose jobs as a result of the economic crisis. He said that this was borne out by the inclusion of health in the European Semester – which sets out the macro-economic plan for the EU in a given year.

Mr Rys also pointed to actions that have been undertaken in order to improve the health sector, in line with suggestions from the ECFIN/EPC report on health systems and fiscal sustainability policy, which showed that there was indeed room for improvement. Highlights include ongoing public consultation on HTA in the EU, cooperation between Eurostat and Member States in the field of big data, and the work of the Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) – which seeks, among other things, to optimise regulatory tools and adopt a holistic approach to access issues.

23 November 2016, will see launch of next OECD health report, which would update European statistics and provide the health sector with much food for though, Mr Rys said. “Europe has the capacity to work together, can produce and is able to find the formula to work together,” he concluded.

The next speaker was Christina Akerman, President of the International Consortium for Health Outcomes Measurement (ICHOM), whose role is to unlock the potential of value-based healthcare. The aim is to be able to compare outcomes across the global, reduce variations and increase transparency, which in turn improves compliance. ICHOM Working Group members originate from 39 countries, including low and middle-income countries.

She noted that health systems are extremely capable at measuring processes, but far less successful in measuring outcomes, which themselves “vary incredibly” across the EU Member States.  Dr Åkerman sought to define outcomes as the results that people really care about: essentially, the ability to live normal, productive lives. She added that patients themselves needed to be involved actively in the definition of outcomes. “We need to see whether we can commission our healthcare based on outcomes,” Dr Åkerman said.

There is a need to drive international standardisation in this area, she stressed, and to this end ICHOM is currently developing a global benchmarking programme (the Globe Programme) and also develops “standard sets”. Within the standard set mechanism, ICHOM not only incorporates the ideas of patients, but also takes into account the views of carers. 

Bettina Ryll, founder of Melanoma Patient Network Europe, stressed that to realise the benefits of healthcare, “we need results”. She stressed that non-patients do not fully comprehend what it is like to be a patient, adding that patients, who under stand the disease which they are living best, are the largest under-used resource of our healthcare systems. “Patients have access to knowledge that can help us target interventions at what matters most,” she said.Nevertheless, she also noted that there is no such thing as “a patient perspective”; there are countless conditions that vary greatly and therefore different groups/people may see different risks and benefits for the same treatment. Dr Ryll said that for a patient, achieving the best outcome meant not only living longer, but also living well: this brings dignity. 

The second session of the Health Collaboration Award looked at how we value medicines in the context of an outcomes-focused healthcare system. The panel suggested five areas where partners in healthcare could collaborate, including: establishing a framework to define value; ensuring a commitment to new commercial models; promoting data infrastructure investment and policy change to support this; and introducing better planning/horizon scanning. There was also a warning that basing drug prices on development costs, would just encourage “inefficient development” rather than rewarding value. "Surely we want to reward new medicines based on what they deliver rather than how much they cost to develop," said Richard Torbett from ABPI. 

Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders, acknowledged that increasingly drug companies are bringing in patients to discuss what the outcomes are that they care about. She conceded that this action would not solve everything, but added that leaving patients out would not solve anything. She noted the difficulties in getting new medicines through reimbursement systems, suggesting that achieving a value-based answer for most drugs that will make health technology assessment systems say “yes”, was unrealistic. She called for a solution to help deal with high prices, but stressed that introducing price controls would not help as this would lead simply to lower development standards. 

Healthcare stakeholders have a mission to bring innovation to the European citizen and the patient, said Luca Pani, CHMP and SAWP Member at the European Medicines Agency (EMA), but added that there would be no value to innovation if patients were unable to access it. He noted that early, joint scientific advice by regulators and HTA bodies, offered an effective solution for access problems, but more effort was needed. Italy, for example, has made huge strides in faster access, with less data for authorisation then using real-life data, Professor Pani said. Links to e-health, real world data and registries would help the value position of medicines in health systems, he added. 

Professor Pani said there was a sense that payers feared that high-priced medicines that work too well could make them unaffordable and health systems unsustainable. He nevertheless pointed out that reduced spending on medicines denies industry return on investment to reinvest in innovation. Outcomes-focused healthcare would only be possible with the same or lower costs across the entire treatment pathway, he added. Professor Pani suggested that changing the system would mean convincing finance ministers to adjust their approach, though, and this would not be an easy task.

Bernard Merkel, Special Advisor at FIPRA, a global public affairs consultancy network, stressed that health systems exist to produce health gain and that this needed to be scrutinised. Agreeing with an earlier presenter, he noted that 99% of conversation in health policy circles is about inputs and processes, with little attention paid to outcomes. Merkel said that in order to address health budget constraints, we would need to have outcomes that are both measureable and standardisable.

Andrew Morris, Scotland’s Chief Scientist for Health, suggested that, unless we prioritise data science in healthcare, in 10 years time, we would all be talking about the same issues in healthcare. “I think the data of ‘collect data once; use it often’ is vital in healthcare,” he said. Professor Morris added that without data to inform decision-making, we would be unable to define value in healthcare. He acknowledged inherent problems in the system, including the scarcity of data scientists in healthcare, which means that the UK was determined to train more of them. Moreover, he stressed that unless data were shared safely and securely, public trust would suffer

Announcing the EFPIA Health Collaboration Awards 2016, Willy Cnops, President of EMEA, UCB, praised the efforts of patient organisations, who he acknowledged were doing tremendous work to get a seat at the table as a recognised and equal partner in healthcare discussions.

The Award for Innovation was won by “The South-West Hepatitis C Partnership”, the Service Delivery Award went to “Cancer Treatment at Home”, the Prevention and Awareness Award was given to “To live with Epilepsy” and the Health Collaboration Award was taken by “Together for Better Health, for us, by us”. 

Opening day two, Steinar Aarset, representing the Norwegian MS Society, told the inspiring story of his challenging trip on foot across Norway as an MS sufferer. He told delegates that the key to living a fulfilled life with a disease, is securing emotional support. 

Josep Figueras, Director of the European Observatory on Health Systems and Policies, explored ways of taking into account patient needs and wants in creating health. He call for clearer medical leadership in shared decision making that truly involves patients, noting that while there are some real champions in this area, there remains “an awful lot of lip service. Dr Figueras highlighted the progress made by Sweden’s Esther project, which aims at ensuring the patient’s input is respected and valued, and revealed to attendees a map that detailed a policy framework for person/patient centredness.

He furthermore offered some thought-provoking insights, including the fact that the most effective primary care appears to be in countries where a “gatekeeper” to the specialist exists and that patients in hospital tend to have a more positive view of the services they access than those looking in from the outside. 

An Innovative Medicines Initiative (IMI) project proposal is set to launch in December this year, on patient engagement in research and development processes, EFPIA Science Policy Director Magda Chlebus told delegates. She issued a call to healthcare stakeholders to support this revolutionary IMI project on patient engagement, which received plaudits from the floor. Ms Chlebus pointed out that currently there is no clear alignment across all constituencies that patient engagement really is useful. There is an urgent need to ensure that missing patient engagement tools are developed and that existing tools are actually used, she added.

David Supple, Chair U-BIOPRED (Unbiased BIOmarkers in PREDiction of respiratory disease outcomes) PIP, a project under IMI, presented U-BIOPRED’s “A short guide to successful patient involvement in EU-funded research", a new booklet on how to best involve patients in EU-funded research. On the specific subject of patient engagement, he suggested that stakeholders needed, as a matter of urgency, to reach out to communities that are socially and economically less fortunate. Mr Supple also floated a case for withholding funds aimed at improving patient engagement “until we reach out” to these, deprived communities and social sectors. 

There is a significant difference between the distinct areas of patient involvement, patient participation and patient engagement, Gary Hickey, representing INVOLVE (UK), which is part of, and funded by, the UK’s National Institute for Health Research, told attendees. He highlighted five key challenges to patient involvement: clarity; accessibility; resources; recruitment; and impact/outcomes. Dr Hickey, though, raised a sensitive issue concerning patient engagement. “If we train patients do they become ‘experts in research’ and do we then lose the public perspective?” he asked.

Isabelle Moulon, Head of Public Engagement Department, Stakeholder and Communication Division at the European Medicines Agency, suggested that industry has not yet worked out how to involve patients. By contrast, she noted that the EMA has been involving patients in its work for over 10 years; it is not just a matter of box ticking. She nevertheless added that the EMA was planning increase its transparency in terms of the way it involves patients and how they contribute to the discussion. Ms Moulon maintained that mandating patient engagement in industry processes would not be helpful, but increased focus on stakeholder collaboration and discussion would be more appropriate.

Mary Lynne Van Poelgeest-Pomfret, Secretary to the Board and immediate Past President, EPPOSI, made a plea for the engagement of all as a multi-stakeholder group on patient engagement. She added that when it came to involving patients in any aspect of healthcare, it was necessary to engage a range of people, including patients themselves, their carers/family and patient representatives. 

Zorana Maravic, Director of Group and Project Development at EuropaColon, noted that it had become apparent with the emergence of biomarker discovery, that patient were unaware of their potential For this reason, EuropaColon has produced information to fill in these gaps. Moreover, the association also has developed a pilot project for healthcare professionals to teach patients about patient empowerment in this area.

Mihaela Militaru, Senior Director Patient Advocacy & Strategic Partnerships, Merck, concurred that biomarkers are a game changer in treating cancer and explained how RAS biomarker testing can show which metastatic colorectal cancer in which patient will respond to a particular treatment. She described challenges that lay ahead for expanding work with biomarkers. They include low awareness of their use and purpose, uneven reimbursement regimes, differing test result times and the lack of sanctioned guidance on applying biomarkers.