The number of orphan products in development keeps growing every year. A particular rare disease will be attractive for a drug development company if it matches one of four main business propositions determined by the interplay of market pressure and technology enablers. The degree of maturity of a field will also attract companies and help prioritize which disease to focus on where multiple rare diseases match the needs of the company or the drug that they are already developing
Impatient series #1 – What patient organizations mean when they say they are doing research
Most patient organizations, no matter how small, start with the same mission: to raise awareness and funds for research in their disease. But what research are they doing? are they all starting in the same place? and do they all evolve to run the same type of research? In my experience there are many types of strategy and approaches that patient groups take, in particular when they are starting. What a patient organization means when they say “we are doing research”, therefore, hides many different possibilities. The article describes some of these types, and discusses their advantages and disadvantages.
Impatient series #0 – The impatient series
INTRODUCTION TO THE SERIES - The field of rare diseases, where each disease affects only hundreds or thousands of patients world-wide, is leading this revolution. Their diseases were once orphan to medicine, too rare to receive sufficient attention and investment to move the needle. Today patients take ownership of their research fields, and move the needle themselves. They have become the center of the network and the expert in the room. They are impatient, not wanting to wait for pharma to take an interest in their disease. They are impatient patients and are leading an impatient revolution.
Main lessons from the European Congress on Epilepsy 2018
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.
En Español - Fármacos en camino para el síndrome de Dravet
Dravet syndrome pipeline review 2018 - Summary in Spanish for Dravet families.
Los avances en torno al tratamiento del síndrome de Dravet en los últimos años han seguido pasos agigantados. En los últimos 5 años hemos experimentado una explosión en el número de programas en desarrollo para tratar la enfermedad: de tener solo Diacomit y nada más a tener Diacomit y 14 más en desarrollo. Este texto es un resumen en Español del Dravet syndrome pipeline review 2018 dirigido a familias, con el texto no solo traducido sino también ajustado a los intereses de aquellos que tienen un hijo/a con síndrome de Dravet y su entorno.
DRAVET SYNDROME DRUG DEVELOPMENT PIPELINE REVIEW 2018
The 2018 Dravet Syndrome Pipeline and Opportunities Review provides a review and analysis of 14 drug candidates in development for the treatment of Dravet syndrome, including 9 products that have received orphan drug designations. It also includes an analysis of the competitive landscape and evaluates current and future opportunities of the Dravet syndrome market.