Spotlight on… Cath Lawrence, Rare Disease Franchise Lead, Early Access Programs.

Cath joined the Bionical Emas Early Access Program division following extensive experience from over 20 years working in the pharmaceutical industry, predominantly in medical affairs in rare and ultra-rare diseases.

Early Access Programs are a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational product for treatment outside of clinical trials when no alternative therapy options are available.

The rare disease landscape is challenging. There are over 7,000 known rare diseases affecting over 350 million people worldwide. People living with rare conditions often wait years for a diagnosis only to find that little is known about the natural history of their disease and that there are no approved treatments. Currently, only 5%of rare diseases have an FDA approved treatment, therefore many people with a rare disease face living with lifelong, often life-threatening conditions and a need for access to therapies.

As Rare Disease Franchise Lead Cath is accountable for ensuring that all rare disease Early Access Programs are carefully designed to meet the nuances and complex requirements that rare conditions can present. For example, rare disease patients and their families often live with a high level of disease-specific burden and so the rare program team take time to understand each condition at the start of the program in order to minimise patient burden where possible. Consideration may also need to be given to the geographical spread of clinical expertise which may be limited in some countries giving rise to potential inequalities in access and cross border travel.

Leading on rare disease Early Access Programs allows Cath to use her passion for providing patients appropriate access to treatments in this area of high unmet need on a daily basis, something she finds very rewarding.

To find out more about Bionical Emas, our Early Access Programs or Rare Disease Franchise, please email cath.lawrence@bionical-emas.com.