"We are delighted to have been recognised by EURORDIS and the rare disease community for our work in advancing the treatment of rare diseases in Europe and around the world. As a company committed to investment in research and development, Novartis is proud to be part of the solution to make these orphan medicines more accessible to patients," said Dr Susanne Schaffert, Head of Region Europe and President of Advanced Accelerator Applications (AAA), Novartis Oncology. "We will continue to forge strategic partnerships that help to make a difference to the rare disease community."

EURORDIS-Rare Diseases Europe is a non-profit alliance of over 700 rare disease patient organisations from more than 60 countries. Together, they work to improve the lives of the 30 million people living with rare diseases in Europe. The EURORDIS Black Pearl Awards are presented to the stars of the rare disease community for their major achievements and their outstanding commitment to the rare disease cause. Nominations are submitted by the general public, shortlisted by a nominations committee and then selected by the EURORDIS Board of Directors.

In announcing Novartis as the winner of this award, EURORDIS drew attention to the collaborative style of working we proudly embody, highlighting our "effective collaborations with the scientific, medical and patient communities." At a strategic level, we promote collaboration as a way to develop emerging fields of biomedicine and build strong partnerships that aim to deliver optimal patient outcomes. As an example, we were the first major pharmaceutical company to announce an alliance to develop chimeric antigen receptor (CAR)-T cell therapies for cancer and in 2017, this treatment received the first ever FDA approval for gene therapy to treat cancer in children and young adults. To have our science-based innovation recognised by EURORDIS as "the hallmark of the rare disease community" is a huge honour and marks a new age in rare disease therapies.

The rare disease landscape has seen enormous progress since 2000, when orphan drug legislation was first adopted in Europe. As an early investor in orphan medicines, Novartis has been able to strengthen our knowledge and capabilities through experience. As a result, our rare disease research and development uses the latest science and tools to bring much-needed medicines to the community.

This prestigious award from EURORDIS recognises that we have set the bar high, both for the rare disease community and for ourselves. Our continued common goal is clear - we must, and will, aim to achieve better outcomes and care for people living with rare diseases.