Patient registries at the World Orphan Drug Summit

I’m just about to leave for Frankfurt, Germany, to participate in the World Orphan Drug Summit. I’ve been invited to help facilitate a workshop on patient registries on Monday afternoon, led by CheckOrphan. It’s going to be an opportunity to discuss many of the issues we tackled in our application to the EC (see yesterday’s post) and encourage businesses to think more creatively about how they build patient registries.

So why are patient registries so important? Basically, a registry is a database of patient information about their condition, their treatments, their health, etc. It allows the registry owner to track how well patients are doing and is particularly useful for clinical trials. Without data on patients, it’s impossible to prove that a treatment is working.

Unfortunately, many patient registries are way too complicated. Clinicians and patients find them difficult to fill in and they’re often clunky and hard to use. That’s why we’re looking at setting up a very user-friendly registry, with online access by patients and clinicians, with all the data then anonymised for clinical trials.

It’s very important for rare disease patient groups to develop registries, as these will give them access to significant data for then working with industry and academia on developing new treatments. I’ll be publishing more about this in subsequent posts.

Plans for an EU-registry for AKU patients

OK – I’ve now submitted our proposal to the European Commission’s Executive Agency for Health and Consumers. We’ll hear by autumn whether it’s been successful. It’s highly competitive, although I’m cautiously optimistic because I think we’ve done a terrific job. The aim is to build an EU-wide network for AKU, based on our existing partners, but also identifying new ones, then to build an EU registry for AKU patients and an online reference network for AKU clinicians to share information about the disease and agree on common treatment guidelines.

If we get the funding, AKU Information Centres based in France, UK, Italy and Slovakia will develop simple mechanisms with health professionals to identify people with AKU, direct them into appropriate treatment and support and develop cooperation on AKU. They will start working in their own countries, and then extend to neighbouring countries.

Our AKU EU Coordination Centre, hosted in the UK, will coordinate the project. We will email patient identification questionnaires and information packs about AKU to a list of all doctors and medical specialists, helping with diagnosis, treatment and care. We will send information to people affected by AKU and their families/carers. We will set up a European network of AKU helplines/email systems. We will set up an EU register of people with AKU. We will develop the online registry into a virtual European AKU reference centre, with care and treatment guidelines and exchange of information. It’s all based on the successful pilot project we did in the UK in 2007-08.

Here are the expected outcomes by the end of the project:

• To set up an EU Information Network for AKU and identify up to 600 new patients in the EU.
• To set up an online registry and collect data on up to 1000 AKU patients (new and existing ones) in the EU.
• To set up a virtual EU Reference Network for AKU that shares information and expertise in order to improve overall quality and management of care for AKU across Europe.

I’ll let you know as soon as we hear back from the European Commission.