Jo Balfour
Managing Director, Cambridge Rare Disease Network
Few rare diseases have a treatment, small market size, lack of information and low awareness can hinder progress. Can we build the right networks to contribute to developing new treatments?
Of the thousands of rare diseases currently identified, around 10% have an approved treatment. It is a significant health issue when orphan conditions affect approximately 30 million patients or around one in 17 people in Europe alone. Lives continue to be impacted by illness and unacceptable, untimely death.
The complexity of developing treatments
There are a myriad of challenges in developing treatments for orphan diseases. Low awareness of rare conditions is common amongst the public and health professionals. Additionally, there is an inherent economic conundrum where the market size is small. A company developing a treatment may find it hard to cover its costs unless the drug carries a very high price tag. Low geographically scattered patient numbers can blight clinical trials and regulators still favour endpoints compared to placebo groups.
We believe that the whole drug value chain, from scientific discovery to development to access, should be considered as a whole to ensure that the right treatment gets to the right patient at the right time for the right price.
Collaborating for improved outcomes
As an organistion, we recognised the potential for our networks to collaborate, to drive improved outcomes for rare disease drug development and through this our Companies Forum evolved. This is a progressive group of industry leaders from pharma, biotech and healthcare who meet to exchange ideas and develop a joined-up approach for supporting and influencing drug development strategy with patient groups, for patients.
In rare diseases, there is a wealth of unmet therapeutic needs and limited resources available. We realised the need to avoid duplication, minimise inefficiencies and look beyond the few rare diseases currently receiving the most attention. We believe that the whole drug value chain, from scientific discovery to development and access, should be considered as a whole to ensure that the right treatment gets to the right patient at the right time for the right price.
Capturing the patient perspective
We are great believers in the power of patient voices to help the industry overcome the above challenges – and by this, help make treatments possible for their conditions. We have welcomed advocacy groups to share their unmet needs with our Companies Forum members, recognising that they have become essential in creating transformational therapeutic advances and increasingly they want to be involved in the drug development process earlier and more often.
Together, we can tackle the challenges of developing treatments for the 90% of patients living with rare conditions head on.
