Five tips for building a patient-centred rare disease clinical programme

The AKU Society has been incredibly fortunate in its partnership with the Royal Liverpool University Hospital. One of our founders, Dr Ranganath, is a clinician at the hospital and has been instrumental in helping us build a strong relationship with the growing team there.

I asked him what he thought were five tips he could share about building a patient-centred clinical programme for a rare disease, based on his experience with AKU. The reason I emphasise ‘patient-centred’ is because many clinical programmes are unfortunately not very patient centred – they’re more focused on the needs and schedules of the doctors and hospital. But if you want to build a clinical programme that genuinely answers the needs of your patients, you’ll need to make sure that the clinical team puts the patient at the heart of what they do.

Here are Ranga’s five tips:

1. Have patients involved and influencing it: from the start, the AKU Society was about a close collaboration between patients and clinicians, working equally and in partnership. This means that the AKU Society, as a patient group, has a strong input into the design of the clinical programme. In fact, we design it hand in hand with Liverpool hospital. We also consult with our patients to make sure the programme fully responds to their needs.

2. Establish a centre dedicated to researching and treating the condition: this takes time and lots of energy, but it’s crucial. You need to find a centre in your country that will be devoted to your disease. If you live in a big country such as the USA or if your disease is less rare than AKU, you may need more than one centre. We’ve opted for focusing on one centre because we want to maximise the use of our resources.

3. Build a group of clinical experts who can come together and solve problems: this is why you need a clinical champion – a doctor within a hospital who will champion your cause. You need to find someone who is knowledgeable about your disease – or willing to learn fast – and is respected by their peers. That person will then start to form a team of specialists and build the centre of expertise.

4. Have an active fund-raising strategy: in these cash-strapped times, rare diseases struggle to find finance for clinical programmes. Nevertheless, it’s important that you scale up your fundraising drive, working in collaboration with your clinicians to build a proposal and then approach funders. In the UK, you can approach the NHS’s National Specialised Services to apply for funding. You can find out more from the website here: http://www.specialisedservices.nhs.uk/info/agnss. We’ll explain more about National Specialised Services in a future blog.

5. Publicise and inform people about what you are doing and trying to do: again, this is crucial. You need to go out and promote your work and your clinical programme. That’s the only way people will start to support and refer patients. Contact all your patients, ask them to contact their doctors, and spread the word.