The Synergist

Share4Rare principles for co-creation with patients

A co-creation workshop stock picture
Patients’ participation in biomedical research and medicines development is definitely a safe bet, especially within rare diseases. Share4Rare has implemented co-creation with patients and families to design its digital environment, and the experience could not have been more satisfying

The clue is in the title. Patient engagement (PE) cannot happen without patients, and so it is clear that any materials, initiatives, or projects linked to patient engagement can also not happen without the assistance of patients or indeed their families.

Co-creation with patients is one of the underlying principles of the ethos behind patient engagement. It is now widely accepted that incorporating the expectations of patients from the earliest stages of research will hugely increase the chances of a medicine being successfully developed and brought to market.

Similarly, patients want to feel they have a voice and that they are being heard. By participating in the development of materials and resources and having a tangible impact on outcomes of these processes, they not only feel valued but can be self-assured that they are helping their peers by virtue of their unique insights into the everyday reality of managing their condition. This is even more pertinent when it comes to rare diseases.


Meaningful patient engagement in medicines development requires all stakeholders to have a common purpose and vision and a clear understanding of respective expectations. Ultimately, “co-creation” is about encouraging fair and open participation and quality input based on experience and personal insights of all stakeholders (researchers, clinical developers, regulators, healthcare professionals and patients) to ensure significant and desired outcomes are delivered where the voice of each stakeholder is equally embedded in final outputs. The principle of co-creation should underpin every patient engagement endeavour of this unique initiative; experts now believe we are approaching a tipping point where co-creation with patients is on the verge of becoming the default option throughout the system.

Patient engagement may be especially important for the study of rare diseases in which the number and size of research studies are relatively small, and the added benefit of more targeted outcomes and facilitated recruitment that patient research partners can add is particularly advantageous. By co-creating clinical trials, trial sponsors have reported better recruitment and retainment of patients, as well as a more speedy passage of the drug to market.

PFMD was the first initiative to undertake a comprehensive qualitative survey exploring stakeholders’ expectations from patient engagement in medicines development. The Stakeholder Expectations Matrix was an ambitious survey-based project to understand and clarify each other’s hopes and expectations from patient involvement in drug development.

Despite broad stakeholder categories, clear themes emerged from the endeavour: there was an obvious desire for effective patient engagement but some discord existed in terms of in alignment, structure and clarity.

The underlying sentiment that was consistent across stakeholder groups was that patient-focused medicine development is about involving patients in every step. It was described by an industry interviewee as “… a process through which patients are part of the idea, design, execution and feedback loop of medicines development from pre-discovery to the launch of medicines onto the market.”

However, while this is the ideal scenario, co-creation has its obstacles. Patients may lack confidence in terms of the value of their input. A lack of trust can also impede the process. In addition, buy-in from industry and other stakeholders may not always be a given.

Yet, patients are experts by virtue of living with their disease, as are their families, friends and caregivers. Their input should therefore not simply be viewed as valuable but essential in terms of developing innovative medicines for people with rare diseases. Drugs developed with better patient engagement will mean more relevant treatments and interventions and more meaningful outcomes for patients.

Share4Rare provides you with a platform to share your knowledge and data to advance research in rare diseases and connects you with people based on your disease and symptoms. Register in Share4Rare and help us reach patients with rare diseases all around the world!