A panel of experts in rare disease discussed some of the latest trends at Labiotech Refresh, focusing on the growing patient involvement and how it is benefiting this field.
Rare diseases are attracting biotech and pharma more and more since they are typically less crowded areas with unmet clinical needs. However, the lower numbers of patients usually lead the companies to give their treatments a high price, which has been the focus of polemic discussions for a long time.
“When you have a very severe rare disease; you have no other choice than trying very innovative therapies, and we are bringing innovation,” said Karen Aiach, Founder & CEO of Lysogene. She started the company back in 2009 to save her daughter, suffering from Sanfilippo A. She’s confident that despite the high prices, new therapies can address the current costs and burden that these conditions represent for health systems and the family of patients.
Karen remarked that patients are becoming more and more involved in the whole process: “Rare disease advocates are more and more listened to“. She mentioned the recent launch of the first committee for rare disease in the United Nations as an example. “We’re taking their perspective into account now, on a very objective basis, to include the expectations of patients and their families into consideration and make sure that the endpoints we will pursue (…) will make sense to them“.
Christian Deleuze, President of Sanofi Genzyme France, agreed: “Without patient support, patient advocating… there would not be Genzyme“. His company pioneered a treatment for Gaucher disease back in 1984. Christian sees involvement as a positive sign that could help solve problems like data collection, which can be a challenge when developing 2nd generation treatments since the trials require high patient numbers.
Rafaèle Tordjman, Managing partner at Sofinnova, brought in the investor’s perspective: “There’s a special interest in life-threatening, very severe diseases that can justify the high price“. Her company does not have funds allocated to rare diseases but rather evaluates each condition separately.
If you want to hear the experts themselves discuss the latest trends in this field, watch our panel video!