Member Spotlight / News - February 22, 2023

EUCOPE Member Spotlight: Q&A with BioCryst

Every month, EUCOPE spotlights a member company and the great work they’re doing to advance the life sciences industry and drive innovation to serve patients better. In February, we spoke with Sandra Nestler-Parr, Vice President, Value & Access, BioCryst Pharmaceuticals. 

Sandra Nestler-Parr is Vice President Value & Access at BioCryst Pharmaceuticals and responsible for pricing, market access and HEOR evidence generation strategy for the company’s product portfolio. Over the past fifteen years, she has held senior market access and HEOR positions in the biotechnology and pharmaceutical industry and in consulting. Prior to joining industry, Sandra spent several years in academic research. Throughout her career, her focus has been on rare diseases, market access of innovative orphan drugs, the evidence and engagement strategies necessary for patients to receive these life-changing therapies, and healthcare policy. Sandra has served as co-chair of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) Rare Disease Special Interest Group. She is also a passionate patient advocate and trustee of a charity concerned with a rare genetic condition. Sandra holds post-graduate degrees in biochemistry from the University of Cambridge, toxicology from the University of London, and health economics, management and policy from the London School of Economics and Political Science.


Tell us about your organisation and its mission and how you drive innovation internally?

BioCryst is committed to improving the lives of patients with rare diseases, a shared mission that unites our 550+ employees. Established in 1986 as a spin-off at the University of Alabama at Birmingham in the United States, BioCryst discovers, develops and globally commercialises oral therapies for patients living with rare disorders with significant unmet medical need.

Driven by world-class expertise in structure-guided drug design, BioCryst has, over the past three decades, created and perfected a drug development platform that integrates crystallography with traditional biology and medicinal chemistry. This unique approach enables the development of new molecules that are potent, selective and bioavailable – all important qualities of any effective oral therapy.

Our drug development platform has yielded a targeted oral treatment for hereditary angioedema (HAE), a chronic, rare, genetic disorder characterised by unpredictable and potentially life-threatening attacks of soft tissue swelling.

We also continue to advance a rich pipeline of investigational molecules that includes compounds, such as an oral Factor D inhibitor, for the treatment of multiple rare complement-mediated diseases.

Despite BioCryst’s recent growth, our unique transparent and supportive ‘ownership’ culture empowers our people to continually achieve and push the boundaries of what is possible. We unite across all fronts in the organisation to serve patients and maintain our mantra ‘go fast, patients are waiting’ at the heart of everything we do. It is the combination of ground-breaking science and our people that drive our success.

How do your organisation’s activities help patients now and into the future?

We look for areas where our science can improve the lives of patients with rare diseases. HAE is a great example. We heard from patients that, although they were grateful for the therapeutic advances over the last 10 to 15 years that helped them prevent and treat HAE attacks, they wanted something more – something that would alleviate the burden of injectable and infused therapies.

Developing an in-depth understanding of what matters to patients and their families is at the core of our drug development journey. We, therefore, involve and incorporate the perspectives of patient communities along with expert clinicians at every stage of our drugs’ lifecycle. Building these long-term, collaborative relationships has, for example, enabled us to improve clinical trial recruitment, which poses a significant obstacle in rare disease research.

At BioCryst, we are committed to providing wide-ranging, timely and sustainable patient access to our therapies. Continuous growth of the evidence base enables us to demonstrate their value to relevant stakeholders across diverse markets. In addition, we also adopt a pragmatic approach towards identifying solutions with healthcare providers that accelerate access.

Patients living with HAE experience many challenges and limitations in their day-to-day life due to the impact of their disease. The partnerships we have established with the patient community, healthcare professionals and healthcare providers around the world help us to understand their broader unmet needs and provide tailored support programmes.

Continuous improvement of our structure-guided platform technology and advancement of our pipeline is another key focus for BioCryst. We anticipate further growth in our portfolio of oral medicines that help address the unmet needs of patients living with a rare condition, and we continue to expand our presence globally to bring these treatments to as many in-need patients as possible.

What do you see as the biggest challenge facing the life sciences industry today?

Never before have there been such rapid advances in health technologies, such as gene therapies, nanotechnology, tissue engineering and digital health technologies. Although many new innovations are expected to revolutionise healthcare and significantly improve population health, they pose historic challenges. I see the most pressing of these challenges for our industry (and society at large) to be the fostering of continued innovation and effective and accessible healthcare solutions, whilst maintaining affordability against a backdrop of growing pressure on healthcare budgets.

It will require multi-stakeholder partnerships and significant evolution of existing policies to identify and agree on pragmatic, sustainable solutions that address rising healthcare costs without compromising innovation or equitable patient access.

The Biotech Social Pact is a promising example of an industry-led initiative that speaks to the above objectives. Progress towards a holistic, viable solution requires all key parties in the healthcare ecosystem to join the dialogue.

What are the major health policy issues and themes that you focused most on in 2022 and focusing on in 2023?

The EU HTA Regulation is a key focus. There remains considerable uncertainty about the specific methodologies which are being developed for the joint clinical assessments for rare disease therapies. Similarly, the operationalisation of the Regulation is still subject to significant debate. We are undertaking a forward-looking project focussed on understanding how the Regulation is likely to impact our internal processes and how we may need to adapt our resources and infrastructure to be prepared when the policy comes into effect.

The integration of real-world data and real-world evidence in regulatory decision-making, as anchored in the European Medicines Agency’s Network Strategy to 2025, is also at the centre of our attention.   Owing to the inherent challenges of research in rare disease populations, the available clinical evidence for rare disease medicines is often limited at the time of license application. Furthermore, healthcare authorities’ concerns about the clinical efficacy and real-life value of rare disease innovations often result in delayed reimbursement and restricted patient access. We have revised and strengthened our real-world evidence strategy to build an extensive and continuously growing body of evidence to demonstrate our therapies’ value that resonates with all relevant stakeholders. It is hoped that an integrated approach to utilising real-world evidence between regulatory and health technology authorities will be forthcoming in the not-too-distant future.

What attracted you to join EUCOPE and how can we help you achieve your business goals?

BioCryst joined EUCOPE long before our recent transition from an R&D to a commercial-stage company. With a modest footprint still in Europe, BioCryst recognises the importance of partnering with organisations that share the goal of improving and saving the lives of patients through innovative health solutions, and that have the ability and weight to amplify our voice on relevant policy topics.

EUCOPE offers a unique platform for its diverse membership to network, collaborate, share experiences and best-practice and join forces to drive advocacy and relevant policy initiatives. Given the typical challenges around patient access inherent to rare disease therapies, we highly value the focus and rich expertise of EUCOPE in country and market access policy, and its well-established relationships with national and EU decision makers that provide a conduit for our perspectives. In addition, utilising its portfolio of membership services and tools has helped us to stay current on relevant European and national policy.

The opportunity to leverage the EUCOPE network has been invaluable in building on existing and developing new working relationships at the EU, regional and national level. Ultimately, this helps us to deliver on our business goals and better serve patients.

For more information about EUCOPE membership

Visit our Benefits of Membership Page, or schedule an introductory call with our Business Development Manager Dante Di Iulio