The World Orphan Drug Conference in Brussels

It was 300 attendees at the World Orphan Drug Conference in Brussels from the 16th to 17th of November. Among them was the European Huntington Association’s President Astri Arnesen, the Board Members Svein Olaf Olsen and Bea De Schepper.

The pharma industry, authorities and patient organizations gathered in Brussels to discuss how drugs for rare diseases can be facilitated. It’s a fact that a small patient group and subsequently a small market, makes drugs for rare diseases hard to economic benefit of. Developing drugs costs money and it is time consuming.

Often it takes more than 10 years to provide a drug into the market. Gerro Stroebbe, the Global market Access Director of Novartis, emphasized how important Enroll-HD is in making this process faster:

EHA Astri Arnesen and Gerro Struebbe

Astri Arnesen and Gerro Struebbe in a conversation during the conference

“First, in clinical trials we have a problem to find patients.  Second, many patients step down from the trial. Third, the patient group is so small that it is hard to find participants. Enroll-HD is therefore very important.  Here thousands of patients are ready for entering a trial – this means a huge difference compared to starting from zero.”

Further, several of the speakers pointed out that medical treatment in the future most likely will narrow in to the specific individual. This means that the patient voice will be more and more important.”

In the world today, it is estimated that we have about 7000 rare diseases – and several thousands more will be identified in the future. As a consequence, you see a patient group that are not big and homogenous but rather individualistic.

Stroebbe gave this prediction of the future: “You will probably be able to prescript a drug for all diseases”. He further stated that patients maybe will deliver their gene map when entering the hospital. Judging from that map the doctor will know how to treat the disease.

The EHA President Astri Arnesen, the Board Members Svein Olaf Olsen and Bea De Schepper at the Orphan Drug conference.

Astri Arnesen, Bea De Schepper and Svein Olaf Olsen at the Orphan Drug conference.

The President Astri Arnesen is confident that the patient voice of Huntington’s Disease (HD) affected will be heard. “I have noticed that everyone wants the patient voice to be loud and strong,” she stated.

Arnesen looks forward to continue the work in making the voice of HD heard. “In the HD field we have so many fantastic collaborators. EHA has to make sure we play an important role with the resources we have.  It’s about being smart and strategic – and as long as we work together I see success down the road,” Astri Arnesen says and smile.