Orphan diseases Geneva

512px-Full_Spectrum_Team_WavingIt seems that there is an acceleration of interest in crowdfunding in the rare disease and orphan drug space. Rare Connect, an online community that connects rare disease patients globally, organizes a webinar this past October, to introduce how crowdfunding can be used by rare disease patient advocacy groups, to help raise funds to support research (Webinar available for viewing).

A introduces the concept of crowdfunding and its relationship to rare diseases and orphan drugs. Crowdfunding is a “collective effort of individuals who network and pool their money, usually via the Internet, to support efforts initiated by other people or organizations.” There are two crowdfunding models :

• Donation model
• Equity-based or investment-based model.

The first type of model, the donation model, posts interesting projects on websites or on a crowdfunding platform, and then, individuals donate money for no return or, at most, access to discounts or early release of products for a particular cause or campaign. The second type of model, the equity-based model involves actual investment in an entity that is pursuing a project and where individuals receive an ownership interest in the entity.

The AKU (Alkaptonuria) Society, a rare disease patient advocacy organization founded in 2003 in the UK, starts a crowdfunding campaign in September 2013. The AKU Society is dedicated to finding a cure for AKU and provides support for patients with AKU. Nick Sireau, the founder and Chairman of the AKU Society, is the father of two boys with AKU. AKU is a rare disease caused by the lack of an enzyme called homogentisic dioxygenase (HGD), which results in patients not being able to fully break down a black pigment called homogentisic acid (HGA). This results in bones turning black and brittle, which can lead to debilitating health problems. AKU is caused by a genetic mutation. AKU has an estimated frequency of one in 250, 000 to one in 500, 000. There is currently no cure for AKU.

The AKU Society initiates a crowdfunding campaign on Indiegogo, a crowdfunding platform, to help raise funds for an international clinical trial for orphan drug Nitisinone, which shows promise for treating AKU. The grand total of the Indiegogo campaign is £ 121, 012 – over the campaign’s target. The October Webinar, including Nick Sireau’s presentation on the identification of the steps and strategies used for his crowdfunding campaign, is available for viewing.

In November at the World Orphan Drug Congress in Geneva, Switzerland, DevelopAKUre, an international research collaboration for AKU, receives three Rare Orphan Advocacy and Research (ROAR) awards :

• Best European Industry-Patient Organization Engagement – DevelopAKUre & AKU Society

• Best Biotech Pipeline – Sobi which supplies Nitisinone

• Best Contract Research Organization – PSR Orphan Experts, which supplies support for the clinical trial.

Please Note: “Full Spectrum Team Waving” by lumaxart [CC-BY-SA-2.0], via Wikimedia Commons.

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