The World Orphan Drug Congress took place in Barcelona, Spain, late last year.  Several PARADIGM project partners attended the Congress, where they presented the project to the rare diseases community. Given the project’s emphasis on building patient engagement, particularly within vulnerable populations, PARADIGM’s presence was of paramount importance. On Rare Disease Day 2019, February 28, the PARADIGM members who attended the conference reflect on the role patient engagement has to play in this tight-knit but pro-active community.

Mathieu Boudes, PARADIGM Coordinator, explains that it was important for members of PARADIGM to be present at the Congress.

When it comes to patient engagement in medicines R&D, [the area of] rare diseases is ahead of the game. Indeed, the patient groups in rare diseases have a history and a fantastic track record in engaging with regulators and industry to develop new therapies for their members. The community of medicine developers have so much to learn from them.

Boudes highlights EURORDIS-Rare Diseases Europe, that with their new programme the European Community Advisory Boards (also featured in a previous blog article here) as well as their long term presence including activities in diverse procedures and committees at the EMA, helped shape the patient engagement landscape in Europe.

It is very important also to mention that the HIV community, also present in PARADIGM through EATG is an historic player and force driver in patient engagement with industry. Learning from the two communities is a fantastic opportunity for PARADIGM, and we do intend to harvest the fruit of these learning and progresses to make patient engagement even greater.

Paul Robinson of Merck further builds on the value of patient engagement, which he admits can be subjective depending on the stakeholder involved. He says that one of the critical work packages for PARADIGM is the metrics group.

Defining what each participant values, and then coming up with a measurement tool has been a huge challenge, but unless we all see patient engagement as a valuable exercise it is likely to be seen as a hurdle rather than an added- quality step.

He says a literature review has given the group a good feel for what is considered valuable – a framework for measuring this value will be designed, and then tested and refined as the project continues.

Operational metrics are easy – how many patients, how many drop-outs, how fast recruitment etc. but, measuring quality, satisfaction and impact is harder… that is the challenge.

The long-term legacy of PARADIGM was elaborated on by Eva Molero of Synapse, who says the project focused on sustainability “since day one”.

We want to promote the usage and impact of our results. We are looking for strategies that will contribute to making patient engagement the ‘norm’. We have started this trajectory by looking at initiatives that are “sustainable” in patient engagement and beyond.

According to Molero, they have learned that successful initiatives are driven by a clear vision and mission. The importance of being inclusive and involve all stakeholders is highlighted, while the framework for collaboration should enable win-win situations for all groups involved.

It is paramount to lay out a solid governance model where everyone is represented, permeated with transparency, openness and fluid communication. On the processes to bring all stakeholders together and build trust, flexibility and agility have been underscored as key features, as well as the ability to identify early on any changing needs. PARADIGM should look for cooperation and alliances rather than competition.

According to Nicholas Brooke of The Synergist, patient engagement especially applies to rare diseases.

The health ecosystem is shifting to a model that’s going to be segmented on the patients and what matters to them because it’s going to drive costs and outcomes. I am quite optimistic this paradigm shift will benefit medical solution development in rare diseases as the system will tend to shift towards unmet needs and value to the patients to drive research and business decisions.

In terms of patient voices, the rare disease community has already proved to be one of the most active and best organized, adds Brooke.

Rare disease specifics will remain, but systematic patient engagement combined with real world evidence will lead to better patient outcomes in a more efficient way, creating value for the patients and the stakeholders working with them.

Kirsty Reid, EFPIA, was also present at the Congress. She says there remains significant unmet medical need in Europe and across the globe for rare disease; the pharmaceutical industry has a role to play in helping to find effective solutions.

To achieve better health outcomes for patients with rare diseases and to have more treatment options for these patients, companies actively carry out medicine development programs which involve difficulties linked to the unique characteristics of rare diseases, including the limited number of patients available for clinical trials and treatment and there is often limited information on epidemiology and disease mechanisms of the condition being investigated. There is therefore a strong need for effective, meaningful and sustainable patient engagement between the patients, the extended stakeholder community and industry, according to Reid, who adds PARADIGM is an “essential driver” in achieving this. 

There are orphan disease incentives in Europe that have seen investment in rare disease R&D grow considerably and the availability of safe and effective orphan medicines has increased including a substantial increase in new clinical trials for drugs treating rare diseases being registered in the EU. However there remains a need of support for global medicine development through engagement with patients that allows it to be tailored to population needs that will benefit rare disease patients. And at EFPIA, #WeWontRest until all people with rare diseases get faster access to medicines.

PARADIGM members not only got to bring the project’s goals and objectives to the rare diseases community but also gained new insight into the needs of those with rare diseases, as well as those working in the area, while at the Congress. The collaborative nature of PARADIGM means that this was an opportunity for several stakeholders from different project partners to deliver their individual perspective and interact with a community already deeply versed in patient engagement.