Every other year the International League Against Epilepsy organises a major epilepsy medical congress in Europe called the European Congress on Epilepsy (ECE). This year I attended the main three days of the ECE addition in Vienna, looking at the field partly as a drug developer and partly as a patient advocate working on behalf of rare epilepsy patient communities. Here is the list of what I found the most interesting at the ECE 2018 meeting.
Should we talk about syndromes based on the gene that causes them or should we talk about them (and treat them) based on the clinical characteristics that they display? Earlier this month, the epilepsy community gathered in Barcelona for the 32nd International Epilepsy Congress and there was a debate between genetic and symptom-base syndrome classification. This debate goes beyond semantics, and has important regulatory and access implications.
June 23 is a special day for families of people with Dravet syndrome. It is the International Dravet Syndrome Awareness Day, that in 2017 celebrates its 4th edition. That's why today we announce the publication of the 2017 Dravet Syndrome Pipeline and Opportunities Review, a market research publication that provides an overview of the global therapeutic landscape of Dravet syndrome.
February 28 is the Rare Disease Day, and the global theme this year is research.
People from all over the world will come together this month to advocate for more research on rare diseases, and to recognize the critical role that patient organizations play in research.
I am excited to join this year Rare Disease Day and announce the launch of my first eBook: #ImpatientRevolution, a guide for impatient patient organizations.
With 2016 numbers now available, the number of orphan drugs in development for neurological indications is looking quite positive. I have reviewed the numbers of orphan drug designations and approvals by FDA in 2016 to see how popular are neurological orphan drugs today and what the trend is for the near future.