EUCOPE Member Spotlight: Q&A with Jane Cooper Senior Vice President and Region Head for Ultragenyx Europe, Middle East and Africa

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 June, we spoke with Jane Cooper Senior Vice President and Region Head for Ultragenyx Europe, Middle East and Africa.

Jane leads Ultragenyx’s regional strategy, development, operations and commercialisation across Europe, The Middle East and Africa region, driving patient access to innovative therapies for rare and ultra-rare diseases.

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

Ultragenyx is 15 years old this year. We were founded to advance innovative medicines for rare and ultra-rare diseases that have never been treated before. We’ve been doing this for 15 years by focusing on diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies. We are proud to deliver transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare diseases. In Europe and the wider EMEA region we drive this innovation by advancing the research – 39% of our clinical trial sites are here and over a third of total patients enrolled or planned to be enrolled in global trials are in our region – and by allowing patient access.

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

Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that’s meaningful for rare disease communities. We are developing and commercializing therapies for serious rare and ultra-rare diseases, which typically have no approved therapies treating the underlying genetic cause of the disease. Since our founding, we’ve launched four innovative medications for five diseases with few or no treatment available before Ultragenyx existed. We’ve engineered our own dynamic development model to meet the unique and difficult challenges of the rare disease space and develop first-ever medicines faster for families who need them. This dynamic development model tells us to work directly with families affected by these diseases because often they are the only ones that can provide that unique insight and inspiration needed to solve the pathway to treatment.

Beyond our pipeline and portfolio, we work with patient advocacy organizations, clinicians, policy makers and other stakeholders across the region to raise awareness of rare diseases and their impact on patients and their families.

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

The biggest challenge we face is that to some other people, rare diseases are too rare to matter. In the rare and ultra-rare disease space, the answers don’t come easy. To go after diseases never treated before we must examine the problems from new angles and discover new insights that move therapy development forward, faster. It means we must be bold enough to sometimes question widely held assumptions about a rare disease and challenge convention, which can uncover—and has uncovered— insights that lead to a much-needed breakthrough. For families that are affected, they often feel alone and forgotten, and treatments for their rare disease can’t come soon enough.

Too often the drug development and approval processes do not share this boldness and urgency. Too often diagnostic pathways like newborn screening lag by years behind disease-modifying treatments. Too often innovative rare disease treatments like gene therapies are expected to follow regulatory and reimbursement pathways designed for small molecule chronic disease products.

What are the major health policy issues and themes that you are most focused on in 2025?

My focus for Europe remains to collaborate with EU policy makers, member states, regions, patient and clinical communities on health policy issues that support diagnosis, drug development, accelerated approval and access to treatment.

We see opportunities to make real strides forward on these policy issues with the EU’s renewed focus on competitiveness and growth. We believe Europe can be more competitive as a place to do rare disease clinical trials with a rare disease 30-day fast track for trial approvals; and by investing in national registries and cross-border data access; and by harmonising Newborn Screening Panels with mutual recognition pilots for ultra-rare diseases.

We believe European rare disease patients and their families can have faster and more equitable access to new treatments with greater regulatory data equivalence in ATMP and OMPs; and with the implementation of EU HTA regulations that reflect the complexities of ATMP and OMP therapies; and if upcoming legislation—including the General Pharmaceutical Legislation, the Critical Medicines Act and the EU Biotech Act —are fully utilized to improve the movement of ultra-rare disease and ATMP patients across Member States.

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

EUCOPE helps us face our biggest challenge – that to some, rare diseases as too rare to matter. The organization, the policy experts that work there and the other members we work with are as committed as us to making sure the challenges of rare disease drug development and patient access to new treatments are part of all health policy discussions at the EU level. EUCOPE is as committed as Ultragenyx to answering those questions that don’t come easily in the rare disease space.