Julie Matthews
CEO, TMC Pharma
What makes a company successful in orphan drug development?
When orphan drugs first came to notice in the late 1990s, pharma companies saw insufficient commercial potential. Small inexperienced companies, on occasion born out of desperation of families afflicted by a rare disease, have led the way in orphan drug development. These small companies therefore require substantial, focussed support by a multi-skilled company such as ours.
After 20 years’ working with many small companies and a diverse range of rare diseases, we have the broad and highly specialised experience necessary to provide innovative approaches to orphan drug development.
How has the orphan drug landscape changed in the last 5-10 years?
Historically, orphan diseases have been neglected by drug developers. Most are serious and life-threatening diseases, patients and their families affected now want to see medicines developed as fast as possible.
Big pharma companies are now involved in the orphan drug arena, but do not generally develop the products themselves. Instead, they wait for small biotechs to develop the drugs and purchase them after marketing approval has been obtained.
Another big area of change has been the emergence of complex advanced medicinal therapeutic products (ATMPs) such as cell and gene therapies. The range of medicinal products to treat rare diseases is now at an all-time high.
Historically, orphan diseases have been neglected by drug developers. Most are serious and life-threatening diseases, families affected now want to see medicines developed as fast as possible.
What are the key success factors for developing new treatments in rare/orphan disease areas?
In orphan drug development, it is important to have a team with not only in-depth but also wide experience. While a small biotech company may have only one or two products in development, our teams are working with around 60 clients, developing over 60 medicines at any given time.
Clearly defined, effective partnerships between the sponsor company, the clinical trials team and the investigational sites recruiting patients ensures high-quality trials. Finding appropriate patients to recruit quickly is often helped by working with energetic patient support groups.
How are you accelerating development in this area?
We ensure that companies developing orphan drugs apply for incentives such as orphan drug designation (ODD) which gives expedited processing and less costly regulatory fees.
We help with early definition of the best product development plan, often advising how to run clinical studies in innovative ways to make them more efficient. Our global team then seamlessly puts plans into action, supporting and driving all development activities through to marketing approval and beyond.
