Recovering the costs of research in rare diseases is a big challenge. Jenni Thorburn shares her experience of crowdfunding in rare diseases and how it can help patient groups such as the AKU Society.

Over 30 million people in Europe are affected by a rare disease. While the word rare gives the impression that only a small number are affected, 6% to 8% of the European population are estimated to have a rare disease.

Despite this, the word rare continues to have an impact. Currently, there are only 84 approved treatments available in Europe for an estimated 6,000 rare diseases. People affected by a rare disease suffer from misdiagnosis, a lack of effective care and often no treatment at all.

Crowdfunding could help change this. It’s a way for the public to help fund projects by collecting small contributions from a large collection of people online. Crowdfunding has the ability to disrupt traditional models of finance, and it could be a breakthrough for medical fundraising.

Facing the Costs

One of the biggest challenges facing rare disease research is cost. Few drug companies conduct research into rare diseases because it’s difficult to recover the cost of developing these treatments.

To help address this issue, orphan designation was introduced in 2000. The biggest financial incentive is market exclusivity for ten years, meaning similar treatments for the same condition cannot be marketed for ten years after the drug goes on sale. However, this does mean companies can charge extremely high prices for orphan drugs.

Whether it’s research into understanding the disease or a potential drug, finding the funds you need can be hard. Once a company has found a drug which may work, they have to conduct clinical trials into its effectiveness. They’re extremely expensive and it’s even more difficult for rare disease trials. Finding interested scientists and doctors can be difficult, and identifying an acceptable number of patients to take part can be even harder.

What Can Crowdfunding Do?

Crowdfunding could help provide funds towards this research. It could even potentially help a rare disease patient gain access to a treatment.

The Rare Genomics Institute in America began a crowdfunding model dedicated to helping rare disease patients through genome sequencing. The project first connects a patient with a research institution, and then develops a budget which can be funded through crowdfunding. As many rare diseases are genetic, genome sequencing helps increase understanding of the disease – the first step in research. Rare Genomics Institute also plan to utilise crowdfunding to develop cures for rare diseases using stem cells. Their aim is to raise more money for rare diseases than the NIH in ten years.

Similarly, the patient group I work for, the AKU Society, recently conducted a crowdfunding campaign to raise funds for the international clinical trials it’s involved in. While we received funding from the European Commission to cover the medical costs, we needed funds to address issues of patient support and travel.

But the rarity of alkaptonuria (AKU) means patients are scattered across the world – often the case for most rare diseases. The society needed funds not only to identify them, but to bring them from across Europe and beyond to the clinical trial centres. Without the patients, the trial would never be a success. The campaign brought in more than $100,000: a brilliant example of how crowdfunding can benefit rare disease research.

Making It Count

Another aspect of crowdfunding is raising awareness. Crowdfunding isn’t just about picking a platform and fundraising on there; people must promote it as much as possible.

Crowdfunding has to be treated like any aspect of modern media. Campaigns must be interactive, wide spread and integrated. Friends, family and supporters all have to be informed. Social media must be utilised. It’s not enough to simply set a campaign up and hope people find it. It’s hard work, like a marathon, and you have to push it until the end.

You have to keep your crowdfunding page as up to date as possible. Posting regular updates about the campaign is crucial, so make sure you choose a platform which allows you to do this. It will remind people to donate and to tell their friends and family. It’s also crucial to post photos and information as regularly as possible. Perks are also important: it provides a way for you to give back and say thank you to your supporters. You just have to make sure you choose perks that don’t cost too much, as well as making sure they’re unique and unavailable elsewhere.

Running an awareness campaign alongside crowdfunding can really help it succeed. Raising awareness is crucial for any rare disease. It could lead the way to making new contacts, and that one new contact may make all the difference. One of the most important things for patient groups, especially rare disease ones, is to form partnerships. With limited funds and manpower, they could provide the help you need.

« Your campaign could offer relief to a patient who previously thought they were completely alone. »

You could also identify new patients. Your campaign could offer relief to a patient who previously thought they were completely alone. Often, people with rare diseases don’t even know they have one. They may face years of misdiagnosis. Or if they are diagnosed, they may not be in contact with you, know about research being carried out or even know a patient group for their rare disease exists. For both the AKU Society and the Rare Genomics Institute, identifying patients was crucial to their projects success.

Why Would They Care?

Crowdfunding can help the causes which won’t get funding elsewhere. It can often be difficult to get attention for the rare disease you want to help, and it’s even harder to compete with large charities for more common conditions. Crowdfunding is an accessible, affordable way of getting your voice heard.

Through crowdfunding, you could find the vital funds needed for rare disease research. It could be the difference between the success and failure of your project. And it offers the public, and your supporters, the chance to potentially help cure a rare disease.

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