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#WODC2014: Orphan Drugs – Facing Challenges with Passion

December 09, 2014

by admin

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Having spent the last three days at the World Orphan Drug Congress in Brussels #WODC2014, one can only be impressed by the challenges ahead for patients, companies and authorities. I’d like to mention just a few of them.

One of the challenges are the number of rare diseases in the spotlight. These numbers remain staggering. One of the targets set is to get 6,000 to 8,000 diagnoses by 2020. It was suggested that this was overly ambitious but still half is already an enormous amount. Yet this is the only way forward, as because of the rarity of these diseases, they are often misdiagnosed or missed all together. When it comes to the quest for therapy, as Shire’s Kim Stratton explained, with only 93 therapies approved and with 75 available for to market, this compounds that challenge.

Secondly the preclinical, clinical and related to that, regulatory track, is non-existent in almost all cases, so there’s a need for cooperation with all parties involved. As Alexion’s Dr Stephen Squinto indicated, it is essential to work in close collaboration from the start with the authorities and patients. This is unknown territory and the whole community needs to work together.

The third challenge relates to health economics and market access and is of a different magnitude – certainly in Europe. Whereas the first two challenges are more global, this remains at the national level, as was underlined by Eurordis’ Yann Le Cam. Unfortunately each country sets its own criteria based on national benefits. Although the total budget for rare diseases is only around 3% of the total pharmaceutical budget in Europe, still discussions around cost and price are key. The ultimate goal is to deliver effective treatment whenever a breakthrough is made, wherever the patient lives. Today, as was underlined in the conference, one third of the target population in Europe has no access to orphan drug therapy following market authorisation and pricing.

However, it was gratifying to see that when progress is made around debilitating and life threatening diseases, that these diseases have faces. Being close to the patients who need treatment has a powerful effect on maintaining one’s focus. Helping the patient remains the ultimate goal of all parties involved. It also explains why you find so many passionate people in this industry sector; the proximity and severity of the patient’s condition inspires the industry to give its best.

One of the key soft skills of people in this area is genuine passion. If one wants to be successful in the field of orphan drugs, more than anywhere, you will have to turn a “no” into a “yes” through your unshakable passion for patients with rare diseases.

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