The European Patients’ Academy for Therapeutic Innovation (EUPATI) took its first steps towards the development of pan-European and national infrastructures with its inaugural regional workshop, held in Frankfurt, Germany, on 5 September 2012.
The project, funded by the European Union’s and EFPIA’s Innovative Medicines Initiative and led by patients, was launched in Copenhagen in March 2012 with the aim of providing scientifically reliable, objective and comprehensive information to patients on medicines research and development. The Frankfurt meeting was the first opportunity to discuss its progress with stakeholders and potential collaborators. More than 100 delegates attended from 24 countries.
“Everybody recognises that there are lots of unmet needs for most patients with life-threatening diseases,” said Nicola Bedlington from the European Patients’ Forum. “Patients need therapeutic innovation, and we are helping to address this by involving patients in therapeutic development. We know it can be done.”
The PatientPartner project, Bedlington said, had shown “unequivocally” the key role of patient organisations in building a new environment for the development of new therapies. PatientPartner also acknowledged that patients need training if they are to contribute to scientific projects as equal partners. That’s where the European Patients’ Academy comes in.
“The workshop,” said Matthias Gottwald from Bayer, was “an excellent example of the value of networking. And networking is the basis of the EUPATI project.” Gottwald noted that the training it would provide would help not just patients but all stakeholders, including not just academic research, regulators but also industry, to discover the value of involving patients early in the drug discovery process. “I am really glad that the project is entering reality after all the preparatory work.”
These are early days for the European Patients’ Academy, yet it was clear from the presentations that much work has been done, and the general shape of the project is clear. But nothing is set in stone, Bedlington told the workshop: “We have a framework and basic ideas, but your input as experts from different arenas and geographical areas is absolutely vital to shape what we do. It’s open.”
It’s all about networks, delegates heard. “Without a network, we’ll get nowhere,” said Kim Wever from the European Genetic Alliance Network. “And this is your first network meeting,” she continued, as she laid out the first tasks for the day: to think about how to make EUPATI’s planned National Liaison Teams and National Platforms a reality.
The idea is that the National Liaison Teams – including at least one representative each from patient organisations, academia and industry – should kick-start activity at the national level, leading to the foundation of EUPATI National Platforms, which would, among other things, roll out the Patient Academy’s training and adapt it to national needs.
Session chair Barbara Haake from the German Association of Research-Based Pharmaceutical Companies (vfa) stressed that EUPATI is a collaborative project. “We are at the very first stage in this project,” she said. “The European Patients’ Academy needs your help to make it a success.”
Raising awareness about EUPATI
Taking up the network message, the first breakout session, chaired by Virginie Delwart from Janssen, Belgium, was about how to raise awareness for EUPATI across Europe. Rapporteur Frank Wells, UK, stressed that EUPATI is “not an organisation but a patient-orientated project”.
As a project with many stakeholders, we need to set priorities, he said. But first, as EUPATI is a patient-orientated facility, “the reasons for patients taking part must be clear”, and the project’s events must be coordinated so as not to clash with events that patients themselves might be going to.
We need to talk to each other, said Wells. “Communication is the name of the game.” European patient support groups must use their contact facilities to tell their members about EUPATI and encouraging them to get in touch directly – if they wish. The process should be aided by a “simple, straightforward explanatory template for communication that can be adapted for different groups” as suggested by the audience.
There are many possible methods of communication, and the session took a straw poll about whether the printed word had vanished. The result: two-thirds of those present said we needed print. “The message from all of us is that we need both print and online,” said Wells, with an enhanced EUPATI website. Above all, presentations must be succinct and jargon-free.
Meetings will be valuable in the communications progress, with good scheduling, plenty of notice, and locations with easy access. Influential people should be involved if necessary. “But independence is essential,” said Wells. “Industry is a stakeholder but should not be perceived as dominating, because it doesn’t.”
Engaging on the national level
In parallel with the first breakout session, another group, chaired by Eibhlin Mulroe from IPPOSI, Ireland, looked at how best to establish and organise EUPATI National Platforms. “How do we ensure in EUPATI that there are strong national platforms for the dissemination and extension of the programme?” asked rapporteur Søren Skovlund from Novo Nordisk, Denmark. “How do we ensure that the programme and tools don’t stay in a box in Brussels but become living entities in every country?”
The key take-home message, said Skovlund, was that the three-way partnership between patients, academia and industry was important, and it has to be led by the patients. “But it is a daunting task,” he said. “We must be careful not to put too much on the shoulders of the stakeholders.” That means remembering that EUPATI is not trying to create new organisations in each country, but networks to boost the activity of what is already there.
And while EUPATI should be seen as a gift to patient organisations, there is an issue of funding that needs to be sorted out – will anyone help patient organisations with the resources required to implement this?
An important issue identified in the breakout session related to clarity and transparency about the benefits of EUPATI – not in general but for each stakeholder group. Is it clear exactly why people are supporting this programme? Such clarity could be important in getting the necessary buy-in at local level, especially as in some countries there is scepticism about getting too close to the pharmaceutical industry.
How could we go about selecting the National Liaison Teams? It was clear from examples from Poland and Malta that a good starting point, where feasible, was umbrella patient organisations. And EUPATI centrally, where anchored in a country, could facilitate by opening doors for new organisations, especially to regulatory bodies. In countries without umbrella patient organisations, that absence in itself could be an opportunity for EUPATI to bring people together.
Encouragingly, delegates from 14 countries signed up to an initial expression of interest in being part of National Liaison Teams or EUPATI National Platforms. “This shows remarkable concrete commitment and interest in being involved in this important next step of defining how EUPATI is to be supported at the next level,” said Skovlund.
Learning from existing educational material
EUPATI has made good progress in collecting training and education information about many aspects of the medicines development process. However, three areas seem stubbornly sparse: personalised and predictive medicine, pharmaco-economics, health economics and health technology assessment; the safety and risk/benefit assessment of medicines. How to remedy that shortage was the subject of the third breakout session, chaired by Suzanne Parsons from Nowgen, University of Manchester , UK.
When it came to detail, there was much to digest, said rapporteur Christine Mullan-Jensen from Novo Nordisk, Denmark, particularly as regards the overlap between personalised and predictive medicine on the one hand and rare diseases on the other. And with pharmaco-economics, it seemed that much training is for experts in the area, so there is not much for patients – and it can certainly be hard to find patient-friendly material. One piece of advice from the breakout session was to look more closely at the UK, where the area is seen as more developed.
When it comes to patients’ roles and responsibilities, it was suggested that there is material that is buried in documents with more general titles – a suitable case, perhaps, for data mining. “Patients”, it was stressed, means both patients and patient organisations. One suggestion was to include carers in the definition. Many patient organisations also have codes of practice, which could be made available.
And, said Mullan-Jensen, the breakout session identified several topics currently missing – considered, certainly, but not included in the original call for the submission of educational material to the project. These included patient registries, patient surveys, the process of obtaining marketing authorisation, partnership models of patients and physicians, as well as quality of life measurement in research.
More general suggestions on enhancing the material included approaching research centres in universities – there may be quantities of unpublished material. Languages are an issue as well: most materials received are in English. Given that the European Patients’ Academy will develop educational resources in seven languages, it will be important for translators to have specific expertise in the areas covered by the material. In addition, EUPATI Network members in different countries might be asked to help in creating abstracts in local languages.
The next steps are to contact low-response patient organisations, and send out another call for educational materials to the workshop’s participants. EUPATI might categorise the individuals to be approached with “perhaps a more tailored call”, said Mullan-Jensen. Also potentially helpful would be a standard text to outline the benefits of contributing material, and a structured way of organising that information. Here, again, networking will be vital. “Each and every one of us is embedded in an organisation where we could approach other members of the organisation to see how they could be involved or submit resources,” she said.
Delivering the training
Once sufficient material is collected and the lists of topics on which patients should be educated are drawn up, how should training and education be delivered? That was the topic for the fourth and final breakout session, chaired by Nancy Hamilton from EURORDIS, the European Patient Organisation for Rare Diseases.
Rapporteur Liuska Sanna from the European Patients’ Forum described how the session had split into three groups, each charged with considering training for one of the three core audiences of experts, patient advocates and the lay public.
In terms of training the patient experts, there were suggestions but also plenty of questions. What will be the selection criteria for admission to the Patients’ Academy’s courses? Sanna reported the group felt that motivation would be “really key” for this audience, given that the diploma course would last several months and might prove difficult to combine with other life engagements. Training, thought the delegates, should be hands-on, and provided by trainers with a mix of profiles. It should be thorough, though not to the extent of creating patient experts on a level with doctors. EUPATI was also advised to use existing courses provided by, for example, the EORTC and EURORDIS as part of the education.
Other questions remained. Would it be realistic for these experts to be trained as “trainers of trainers”, cascading information down? And there was a question about sustainability: What happens after training – will there be any follow-up?
Information for patient advocates should be provided through a variety of means, the session thought. One methodology is not enough. Participants should be able to choose from among different sources and methods. Different stakeholders, including patients talking about their concrete experiences, should provide information. It will also be important to adapt material to the context and culture of the individual countries. There was a suggestion, too, that in some cases patient advocates would benefit from individual coaching and mentoring.
When it comes to lay patients and the general public, said Sanna, “clearly the information needs to be simple”. Video seemed to be an important form of channelling the information, preferably via YouTube, which is simple to access and widely used. Information should be split by topic or by perspective (for example, of doctors, patients or industry). The key to reaching people will lie in good communication and making the information visible. It was suggested to have magazine articles, and posters in hospitals. The group also discussed collaborating with schools to reach children.
“There is already a lot of knowledge out there,” said Sanna. “We need to pull it together and channel it to EUPATI.” Above all (and this was a recurring theme of the workshop), the project should avoid reinventing the wheel. A final recommendation is to make the content lively – it needs to be interactive, and give concrete life situations. Drug development, said Sanna, is complex, with many grey zones, and the material should reflect that.
Developing the tools to reach out to patients
The initial presentations at the workshop briefed delegates on progress so far. Outlining the project’s strategy, Nicola Bedlington hailed EUPATI as “a paradigm shift in involving patients in research and development”. She agreed that much work has already been done by patient organisations, academics and industry, but it is fragmented and ad hoc. “The role of the Patients’ Academy is to bring it all together, coherently, and reach out to more patients.”
The project will focus on 12 European countries, with materials in the seven most frequently spoken European languages. It will have six core areas: the medicines development process, from start to finish; personalised and predictive medicine; drug safety and risk/benefit assessments; pharmaco-economics, health economics and health technology assessment; the design and objectives of clinical trials; and patients’ roles and responsibilities in the development of innovative medicines.
Project members have already identified three distinct training audiences, as Niels Westergaard from Biopeople, University of Copenhagen, explained. “We are talking about an unmet informational need of patients and public about medicines development, from A to Z. There is a potential for patients to be a part of this. It is very important for us to build a training programme at different levels so patients can play a role in clinical trials at various levels.”
At the most specialised level, EUPATI will offer a certificated diploma course, aiming to train at least 100 patient experts to become fully confident and capable in all aspects of the medicines development process. EUPATI’s own budget will cover the costs of training these “key players”, as Bedlington described them, including travel costs.
In parallel, EUPATI will develop an Education Toolbox – primarily online – offering patients a choice of subjects to learn about; the target is to have at least 12,000 patients using the toolbox by the end of the project’s five years. And finally, at the most general level, EUPATI will offer an online portal for use by individuals or patient organisations that will not just have all the available material online and searchable, but will also be combined with the ability to ask questions.
“We will need to adapt the material to audiences to address the right level of audience participation and knowledge,” said Matthew May from DIA Europe. “We will be coming back to you to check that it is right and in line with your expectations.”
Clearly, web technology is going to be crucial. Here there are just two goals, said John Lenehan from Hibernia College, Ireland, which specialises in online learning: “To create a website that is easy for patient users to access and navigate, and easy for members of the consortium to populate with a variety of content.” That also means providing forums to allow collaboration across any of EUPATI’s audiences.
Quality of content is key
The accent will be on quality, underpinned by independence and transparency. “We want to deliver education and information programmes on new medicines development that are high quality, patient centric and up to date,” said Liuska Sanna.
Outlining the project’s plans for quality planning, quality assurance and quality control, Sanna described plans for an editorial plan to prevent problems in the development of educational materials, as well as user testing, feedback mechanisms and focus groups. The whole approach will be summed up in a quality management manual which, it is hoped, will contribute to the project’s sustainability by laying down guidelines for future courses.
Bella Starling from Nowgen, part of the University of Manchester and the Central Manchester Trust, gave a snapshot of initial findings about the state of patient information on pharmaceutical development in Europe, based on 300 resources submitted by stakeholders. One clear anomaly has already emerged: there seem to be relatively fewer resources in three of EUPATI’s core areas – personalised and predictive medicine, pharmaco-economics, and patients’ roles and responsibilities. “Findings like this will really help EUPATI to focus on its priorities,” she said.
Another interesting result, said Starling, was that relatively few of the resources were produced by public-private partnerships or collaboration between experts from different areas. That is a gap EUPATI, with its collaborative approach, can fill, she said.
Exciting times ahead
EUPATI will run for five years, and has a sustainability strategy to ensure that the resources and training it sets up will be able to continue after its core funding ceases at the beginning of 2017. A lot has to be achieved until then – but the first EUPATI Workshop in Frankfurt has demonstrated that EUPATI is “up, running and real”, ready to take on the challenges and take patient engagement in research to a new level.