Skip to main content
Home » Rare diseases » Faster treatment access: why putting patients first leads to better outcomes

Luke Robinson

Vice President and General Manager, UK, Ireland, Nordics,
BioCryst Pharmaceuticals, Inc.

Collaboration with regulators, physicians and pharmaceutical firms can improve access to medication for those living with a rare disease.

Embracing the rare disease community, as well as an effective reimbursement and healthcare system can help speed up access to care. BioCryst Pharmaceuticals explains how committed relationships with patient groups, health care professionals and regulators can result in improved outcomes for those living with rare disease in the UK. 

Prompt response to patient needs 

Vice President and General Manager, Luke Robinson explains what ‘patient first’ means at BioCryst. “As a company, we look at ways we can enable patients to access new treatments more speedily, particularly in rare diseases where there can be limited options and a significant impact on quality of life and often life expectancy. In this space, we have a closeness to patients and physician groups, and because of that, we can see a direct positive impact on outcomes.” 

Putting patients first and caring for people with
a rare disease requires a holistic approach.

Standing with the rare disease community 

The focus at BioCryst is rare diseases — from hereditary angioedema, a disorder characterised by recurrent episodes of severe swelling to the limbs, face and intestinal tract to research focused on complement deficiency disorders and many other rare diseases.

“We chose to invest in the UK because it has a developed healthcare system, a world-class regulator in the MHRA, a well-respected reimbursement body in NICE and active patient groups,” explains Robinson. “It is a good test bed for how you can bring a product to market and address unmet needs in the context of limited treatment options.” He particularly points to the Early Access to Medicines Scheme (EAMS), which complements the company’s ethos of keeping no patient waiting. 

“This enables us to gather ‘real-world’ experience and data outside a clinical trial,” Robinson adds. “While it is a learning experience for physicians and us, in terms of how to use products in the best way, it also supports our collaborative approach, which focuses on working with groups that can support efficient delivery of treatments to those in need.” 

 Supporting better outcomes for all 

“Putting patients first and caring for people with a rare disease requires a holistic approach,” advises Robinson. An increased number of options is essential as living with a rare disease can have a significant psychological and physical impact. While the UK has shown that it is a place where patient-centric companies can work collaboratively with regulators and others, it is important, as we progress, that this engagement continues while always keeping patient need and choice at the centre.  


Next article