RARE DISEASES - To support patient-centricity in clinical trials, look to rare disease communities

Rare diseases by nature affect a very small percentage of people. When looking at the clinical trial landscape, there are simply not as many patients to participate in studies, resulting in fewer companies focused on developing solutions to support the needs of these communities.

But patients cannot afford to wait for solutions – every day that they are left without answers means that their quality of life suffers. Instead, patients are actively rolling up their sleeves and partnering with drug companies to seek out new treatment options that will advance the care of their communities, changing the way all stakeholders collaborate to solve critical issues and bringing hope to people everywhere. This is a textbook example of patient-centricity in action.

While we are making strides across the life sciences industry to become more patient-centric, there remain many competing and contradictory ideas on what this truly means. I believe Guy Yeoman and his fellow researchers put it best in their article, Defining Patient Centricity with Patients for Patients and Caregivers: A Collaborative Endeavour, stating: ‘Putting the patient first in an open and sustained engagement of the patient to respectfully and compassionately achieve the best experience and outcome for that person and their family.’

To advance clinical trials, we must follow this guidance by putting patients first in all that we do. With this approach, we have an opportunity to examine the unique obstacles the rare disease community faces and learn from its successes.

The process of designing a patient-centric clinical trial starts well before it is executed. In the rare disease community, patients and their families have become deeply involved from the start of product design through screening, recruiting and participation – even discussing potential endpoints with researchers to ensure trials meet their ultimate needs. Sustaining this relationship beyond the completion of the trial is critical as well. By following up with participants to understand their experiences, we can not only design products that patients will ultimately use and benefit from but apply these learnings to our future strategies.

Across all therapeutic areas, companies must start to recognise and involve the personal experience of patients to change our approaches. Organisations like NORD, Global Genes and Rare Patient Voice work to amplify the patient perspective and push for regulatory change to help deliver the best patient experience to those seeking new treatments and clinical trial opportunities. The more that organisations adopt this philosophy, the more we will succeed.

With significantly limited resources and a small set of patients that can participate in research, each step of a rare disease clinical trial is critical. When the stakes are this high, inefficiency is not an option. All actions taken by providers, clinical research organisations and sponsors need to consider patients from every angle – not simply because they are working toward a blockbuster drug, but because it’s the right thing to do. By adopting this same level of critical thought and velocity across all clinical trials, we can develop better solutions in both the fastest and safest way possible.

While all companies hope that our clinical trials will provide the answers to restore people’s health, the reality is that patients are doing whatever they can – even participating in multiple trials – to get the answers they need. To them, clinical trials are a continuum of care, so we need to recognise that we are not the singular entity that will solve every patient’s problem. There’s not one sponsor in this world running all clinical trials. Instead, we must figure out ways to build collaborative frameworks that put patients first.

The Castleman Disease Collaborative Network works to build an entire ecosystem around the advancement of new therapies. This collaborative framework includes researchers, physicians, patients and caregivers who are all working together to unlock the complexities of Castleman’s Disease.

This is truly an innovative and sustainable way to quickly develop better treatment options for all therapeutic areas.

The rare disease community has not only figured out how to work alongside patients, families and advocacy groups to solve some of healthcare’s greatest challenges – it has excelled. As we look ahead to the future of our industry, we have a responsibility to operationalise patient-centricity in clinical trials in the same way. Just as the rare disease community has embraced the ideas and experiences of patients as part of its trial design and product development, we too must seek its guidance and integrate this thinking into our approaches.

The ultimate test of patient-centricity will be seen in the evolution of our traditional models, and the companies that come to the forefront will be those willing to rewrite the rules – not around profits but the patient populations they serve. By committing to these shifts in patient-centricity, success will follow.

To support patient-centricity in clinical trials, look to rare disease communities

Adopting patient-centricity as part of clinical trial development

Balancing urgency with patient safety

Viewing clinical trials as a true care continuum, not transactions

A new vision for patient-centricity