Patient Relevant Outcome Measures and Meaningful Patient Engagement – for the good of all: Interview with Nicola Bedlington

Nicola Bedlington

Nicola Bedlington is European Patients’ Forum’s Secretary General since September 2014 and was previously the Executive Director since the setting up of the EPF Secretariat in June 2006.

TB: Mrs. Bedlington, can you please tell us what is your position and main occupation?

NB: My name is Nicola Bedlington and I am the Secretary General of the European Patients Forum (EPF), an organisation that works with patients’ groups on public health and health advocacy across Europe uniting the interests of and voice of patients. EPF helps to empower patients’ organiations through educational programmes, policy initiatives and projects. The European Patients Forum works with all the EU Institution and also in close collaboration with other stakeholders in Brussels. My own background is in the disability field at European level, and I have been with EPF since 2006 when I set up the secretariat in Brussels.

OM: Based on what we heard in the session this morning, we would like to hear your opinion on early patient engagement in medicine development.

NB: We are currently co-leading a specific project called PARADIGM , a Public Private Partnership, funded under the Innovative Medicines Initiative. PARADIGM aims to create a framework for meaningful patient engagement in the entire lifecycle of medicines, collaborating with the industry, the regulators, the HTA community and healthcare professionals, patients, and so on. We have three focal points within the project. One of them is prioritization of medicines research, another one is clinical trial design and the third one is the very crucial early dialogue between industries, regulators, the HTA community and patients on looking at the evidence requirements for specific product technologies. This project started only six months ago, and a strong focus has also been on the matrix of patient engagement, concretely why it matters and the difference it can make.

TB: Could you highlight any major challenges for the project so far?

NB: I think that one of the advantages is the good teamwork – we are a thirty-four-member consortium made up of industry, academia, patients’ organisations, NGOs and ethics panels, and the collaborative spirit is really there. One of the challenges is that it is a very short project, it is only thirty months, so we need to achieve a lot in a very short period. Thus, it is crucial that there is an alignment with other organisations working on patient engagement to avoid any duplication. Rather than looking at it from a competitive perspective, it is important to look at the complementarity and synergies, so that we can move forward as effectively as possible and create durable change in mindset and practice.

OM: From a regulatory framework perspective, is there anything that would support you to better advocate for patients and support them in accessing medicines?

NB: I think we could model what has happened within the European Medicines Agency (EMA), which has a very strong patient engagement strategy. So, patients are involved at all levels: on the management board, in the scientific advisory committees. There is an ongoing patients and consumers working party bringing together patient groups and consumer groups, and the Pharmacovigilance Risk Assessment committee (PRAC) has patients fully on board. These are all examples where patient engagement has really been embedded and it would be nice to see similar approaches in other areas, for example the HTA environment.

TB: How can we support patient engagement in the development of healthcare services?

NB: In my opinion, this is really relevant, because it brings us back to the question “Do we ask the right questions?”. Therefore, patients need to be involved in this discussion – they are the ultimate recipients of healthcare services and what the healthcare system stands for. The right approach is therefore to involve them in the dialogue, rather than have them tick boxes. We must involve them in the discussion around what endpoints we are looking at, and what matters for them as patients in terms of outcomes. We need to move from patient reported outcomes, to patient designed and patient relevant outcome measures. We are collaborating with OECD on this issue through their PaRIS initiative.

OM: Is transparency something that is challenging between different countries and bodies in your point of view?

NB: Transparency can be all sort of things to different people. We have developed transparency guidelines with and for our members. The aim of these guidelines is not about policing member organisations, it is about encouraging them. The role of transparency is discussed not only in relation to funding received from industry – it is also about the way of how we do advocacy and consultancy work, the way our governance is structured, the way we communicate and how we undertake projects. So, in short, our entire area of activity. These guidelines have been very well received by our members. Another example of transparency links to clinical trials. During the Clinical Trial Regulation legislative pathway, we did a lot to make sure that there were appropriate references to transparent publication of clinical trials’ results, also in layperson’s terms, even if these trials go wrong. Obviously, transparency in relation to pricing is also a big theme, we have mixed feedback from our members: some of them wish for complete transparency, others prefer to get an idea how prices are actually arrived at. We will be publishing an updated paper on value and pricing where we call for a fair framework to ensure optimal access to valuable innovation by patients across Europe. We need to find that important balance between innovation and solidarity.  

TB: Is there also a need for patient engagement in the area of orphan drugs?

NB: I think there are a lot of unmet needs in the area of rare diseases relating mostly to children. Big efforts are being made to address some of the bottlenecks in the system. EURORDIS is playing a critical role here as the representative voice of patients with rare diseases, and EPF member.  

This intervierw was conducted by Young Gasteiners Theresa Bengough and Oana Motea

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