BIA webinar recording: Evaluating patient access to rare disease treatments

Watch the recording of Evaluating patient access to rare disease treatments

Approximately 3.5 million people in the UK will be affected by a rare disease at some point in their lifetime. However, approved medicines are available for only 5% of rare diseases. While an area of focus for many healthcare systems, improving access to rare disease treatments is inherently challenging. This means that many patients with rare diseases face high levels of unmet need.

The BIA’s Rare Disease Industry Group (RDIG) has developed a new report that evaluates the current state of play in the UK for access to rare disease treatments, in collaboration with PwC. The report will highlight recent progress that has been made in the UK in improving the lives of people affected by rare diseases, as well as the challenges that remain.

The report will also include an analysis of the approaches taken in other OECD countries to support rare disease treatment access and outline potential learnings for the UK. Watch this webinar to celebrate the launch of the report, and to hear from a panel of experts to discuss the report’s key findings and next steps.

Speakers:

Stephen Aherne

Pharmaceutical and Life Sciences Leader, PwC

Stephen is the Pharmaceutical and Life Sciences sector leader for the PwC UK firm. He is a Partner within the Strategy& Deals practice, specialising in pharmaceutical and life sciences strategy and valuation projects. He has over 20 years of experience within PwC specialising in the pharmaceutical and life sciences industries.

Nick Meade

Director of Policy, Genetic Alliance UK

Nick has worked at Genetic Alliance UK for more than 15 years. Genetic Alliance UK is the largest alliance of organisations supporting people with genetic, rare and undiagnosed conditions in the UK, and is the UK national alliance member of Eurordis. The members and the people they support are at the heart of everything the organisation does. Genetic Alliance UK advocates for fast and accurate diagnosis, good quality care and access to the best treatments; and actively supports progress in research and engages with decision makers and the public about the challenges faced by our community. 

Nick was a member of the NICE Methods Review Working Group. Alongside Finn Willingham of Cell & Gene Therapy Catapult, Nick co-chairs ATMP Engage, a multistakeholder working group focusing on the involvement of patients and the public in the development and delivery of Advanced Therapy Medicinal Products (ATMPs). This year ATMP Engage published 'ATMPs and equity of access', which examined the challenges of delivering ATMPs equitably in the UK.

Sean Richardson

General Manager UK&I at Alexion, AstraZeneca Rare Disease & Chair of BIA Rare Disease Industry Group

Sean has been working in the biopharmaceutical industry for 25 years after attaining a BSc in Biochemistry.  His focus for the past 8 years has been on rare diseases in the UK, working in collaboration with the rare disease community to improve the patient experience of care, from diagnosis to accessing treatment and co-ordination of care.

Tina Taube

Market Access and Orphan Drug Policy Lead , EFPIA

Tina Taube is Director Market Access and Orphan Drug Policy Lead at EFPIA - European Federation of Pharmaceutical Industries and Associations, which represents the pharmaceutical industry in Europe. She holds a Master of Science in International Pharmacoeconomics and Health Economics from the University of Cardiff.