Medical devices for rare diseases: the unmet need

"On 28 February, patient advocates across the world will be looking to raise awareness of Rare Disease Day. A globally co-ordinated movement for people with rare diseases, the day will highlight the challenges they face when it comes to accessing life-saving treatments.

Drugs are a particular sticking point. Since patient groups are small by definition, sometimes comprising just tens to hundreds of people, pharma companies have often been reluctant to work on new therapies. The Orphan Drugs Act, which was passed in the US in 1983 and in the EU in 2000, has improved this situation significantly, by incentivising the pharma industry to develop ‘orphan’ medications. However, many patient groups remain under-served.

The situation is even worse in the field of medical technology. According to a survey conducted by the FDA in 2016, involving 588 clinicians, there is a dire need for new medical devices for diagnosing and treating rare diseases.

Around two thirds (64%) of respondents said they were dissatisfied with existing devices, while three quarters (74%) saw the lack of profitability to industry as a major impediment. Four fifths (79%) reported diagnostic devices for genetic diseases as an unmet need – a red flag when you consider 80% of rare diseases are genetic in origin..." Lire la suite