Regulatory agencies continue to recognize that the drug review and approval process benefits from the input received from patients or their carers. On 26 September the European Medicines Agency (EMA) announced the launch of a pilot project to involve patients in the assessment of the benefits and risks of medicines in its Committee for Medicinal Products for Human Use. It is just another step in EMA’s ongoing drive to continuously engage in dialogue with patients and consumers.
In accordance with Arnstein’s ladder of citizen participation, involving patients in the healthcare sector can take many shapes and forms. When we specifically focus on the pharmaceutical R&D process, three main levels can be identified. Patients or their carers can be invited to share their real life experience (Information); to contribute their view and preferences (“Advice”); to participate as a true partner in decision-making (“Co-production”). The highest level of participation is where patients are completely in the driver’s seat, for example as principal or as funder of a project (“Control).
EMA’s mission is to foster scientific excellence in the evaluation and supervision of medicines, for the benefit of public and animal health. It believes that patients can bring value to the regulatory process in relation to their real life experience of the disease and treatment. This stimulated me to better understand 1) at what levels does EMA involve patients in its review and decision-making process, and 2) has the added-value of patient engagement be made tangible?
A quick scan of the EMA website already makes one thing clear: The agency engages with patients in a professional and thorough manner.
I have always thoroughly enjoyed the wealth of information that EMA shares with the outside world. It has allowed me and fellow-researchers to perform several studies on orphan drug development. Also in this instance the Agency did not let me down. Through its website a staggering total of 24 documents concern engagement with patients and consumers were at my disposal. Especially, the 2007-2013 annual reports on EMA’s interaction with patients, consumers, healthcare professionals and their organizations provide a really comprehensive overview of the Agency’s work with patients and consumers and explains how patient engagement has evolved over time. Not unimportantly, the documents provide the necessary descriptive statistics I was looking for.
Although EMA has engaged with patients since its inception, it was not until 2005 that the Agency generated a formal framework on how to engage with patients and consumers. Shortly after the Patients’ and Consumers’ Working Party, a core group of representatives from patients’ and consumers’ organizations, was put in place and since then provides feedback and expert advice to EMA on all matters of interest to patients in relation to medicines.
In those early days engagement could be considered as minor league and basically was limited to input on a couple of official EMA documents, like European Public Assessment Reports (EPARs) and Patient Leaflets, and formal membership of one Scientific Committee and the Management Board. In the following seven years the scope and number of patient & consumer involvement in EMA activities has experienced a huge transition. Nowadays engagement can be considered as major league. Patients have become formal members of various Scientific Committees, Working Groups, and the Management Board. Between 2008 and 2012 on average they provided input on 75-80% of EPARs and package leaflets. In addition, nowadays they frequently provide the patient perspective during various advisory group and scientific (advice) meetings.
The burning question of course is: which activities can be considered as patients sharing their real life experience (“Information”); which as patients contributing their view and preferences (“Advice”), and which as patients participating as a true partner in decision-making (“Co-production”). Since workshops and conferences organized by EMA are attended by multiple stakeholders, patients are one of many who merely provide “Information” during such meetings. Inviting patients as experts to Advisory group and scientific (advice) meetings or to review formal documents is another matter. For me these activities qualify as patients contributing their view and preferences (“Advice”). Finally, as a formal member of a Scientific Committee or the Management Board patients contribute to the formal decision or advice in a similar fashion as other members. Consequently, for me these activities qualify as “Co-Production”. Using these definitions, I reviewed the annual reports 2007-2013 and grouped all reported patient & consumer involvement as “Information”, “Advice” or “Co-Production”.
Between 2007 and 2013 the overall number of patient & consumer involvement in EMA activities increased from 76 and 551.
The result depicted in the Graph below reveals that the engagement of EMA with patients & consumers has increased considerably in the last seven years at all three levels. Since 2011 the increase appears to be leveling of. In the last three years around 60% of total involvement is related to invite patients & consumers as ad-hoc experts for documentation reviews, especially patient leaflets and EPARs (~40%), and to provide the patient perspective during various EMA advisory group and scientific (advice) meetings (~20%). Although it took me some effort to identify them, all patient and consumer representatives that participate in EMA activities can be found in the European Experts list.
Around 10% of the total annual patient & consumer involvement with EMA can be considered as “Co-production”. The Patients’ and Consumers’ Working Party forms an important part of this involvement. This is complemented with patients being a member of the Agency’s Management Board and all its Scientific Committees, except the Committee for Medicinal Products for Human Use (CHMP). Of course, depending on the success of the pilot project to involve patients in the assessment of the benefits and risks of medicines in the CHMP the latter may change in the future.
Figure: Development between 2007 and 2013 of patient & consumer involvement in EMA activities categorized as contributing “Information”, providing “advice” or contribute to formal decision process/”co-production”
What I find important to mention is that going through the annual reports and related documents I did not pick up any signs of tokenism. When it comes to review of EPARs and package leaflets, a steady 40%-50% of patient’s comments results in a change to the text under review. Patient representatives also provide input in the scientific advice process, and a survey by EURORDIS revealed that in around 50% of cases they believed to have made an important contribution to the final outcome. In 2013 patients participated in over 80% of all Scientific Advisory Group and ad-hoc expert meetings. The 2013 Annual report highlights several examples of tangible input by patients that were included in the final advice or report. In 2013 Parents/patients provided valuable input that helped the CHMP reach its final recommendation in relation to a proposed medicine to treat Duchene muscular dystrophy.
EMA also continuously learns from existing experience and to define recommendations and proposals for action through regular consultations with patients & consumers. In 2009 EMA published the outcome of a consultation that focused on how to further develop the interaction with patient & consumer organizations in a more structured way. One aspect that was emphasized was the importance of appropriate support and training for patients/carers involved in the various EMA activities to ensure that “they are fully prepared to participate and know what is expected of them as patient representatives”. Recently, the Agency published a complete training strategy for patients and consumers involved in EMA activities. A 2010 questionnaire by the Agency revealed that at that point in time overall patients & consumers involved in EMA activities were satisfied with the overall process and the opportunity to engage. At this point you may wonder whether there is nothing wrong with the way the Agency engages with patients. Is there any room for improvement? I believe there is.
As mentioned by the Agency in various key documents, Committee meetings are conducted in English. Although this is understandable from a practical point of view, it does limit the pool of patient representatives to those that are fluent enough to allow a meaningful contribution. Occasionally consulting organizations not fulfilling all the eligibility criteria to be involved in EMA activities only partially solves the problem. Moreover, as depicted in the table below, review of the Annual reports 2007-2013 reveals a skewed distribution towards “non-eligible” patient/consumer organizations originating from English speaking countries.
There is a need to invest in novel and innovative ways of engagement that allow EMA to tap into the huge pool of patient experts in the rest of Europe, and thereby also spread the burden for patient experts, many of whom work as volunteer.
There are a couple of other things with room for improvement, like financial support, but overall I am impressed how EMA consistently and increasingly involves patients in its review and decision-making process. Although it remains difficult to make the added-value of patient engagement tangible, the Agency does provide concrete examples where patients’ input has made a difference. A thorough investigation into the added-value of patient engagement would be very welcome. It would provide additional insight as well as a set of valuable teaching cases that can benefit other regulatory agencies and patients.
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