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Home » Rare diseases » Help rare disease patients by involving them at every stage of development

Charl van Zyl

Executive Vice President, Neurology Solutions & EU/International Markets, UCB

Inclusion of the ‘patient voice’ is critical at all stages of research and drug development, starting from clinical trial design, through the regulatory processes — and during access discussions.

A small or rare patient population can affect clinical trial recruitment and slow down the development of medicines.

Data generation

We should explore alternative study designs, developed closely with regulators, involving fewer patients but still delivering data that allows appropriate evaluation.

We are actively exploring new methods of evidence generation with an emphasis on supplementing clinical trial data with external/historical data, as well as pre and post-approval real-world datasets. Regulators are moving in this direction too, however, people living with a rare disease continue to tell us their concerns and stress the need for greater urgency.

Different measures

People often experience difficulties from the disease which extend beyond symptoms that can be measured in traditional clinical trials. These symptoms can be as impactful and relevant to patients, and, therefore, with better Patient Centred Outcome Research (PCOR) we can establish what matters most to people living with a rare disease. 

We must truly respect patients as the experts
in living with their condition, as only they
can tell us what they really need.

Effective listening

We should work closely with patient organisations and Centres of Excellence to listen and learn about the patient experience. We must truly respect patients as the experts in living with their condition, as only they can tell us what they really need.

As part of our commitment to partnering with people living with a rare disease, we provide opportunities for people to speak about their personal realities and describe how their condition impacts life for themselves and their families. These insights can help improve understanding of symptoms and disease management and are invaluable in clinical trial design.

The scientific community, regulators, payers and industry should partner together in listening to and collaborating with patients, to embrace the patient view on what access to the right care would mean for them.

Industry must communicate to healthcare providers and payers the value new options provide above the treatment costs alone. This could include wider societal benefits and less tangible, but equally important, quality of life and mental health benefits for patients, families and caregivers.

By working together, we can create a greater impact for people living with a rare disease.

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