In honour of Rare Disease Day (29th February 2016), we at HRW are joining together to raise awareness and do some fundraising for rare diseases. As part of these efforts, we bring you an article from our own Lucy Jones on her thoughts about patient research in rare disease areas:

Putting up the posters around the HRW office for rare disease day this year, someone came up to me and asked if providing insights on a rare disease is just a drop in the ocean? This made me think. Yes, each rare disease is a drop, but collectively each drop makes up a vast ocean.

A rare disease is even, in some regards, a bit of a misnomer to me, because collectively, rare diseases aren’t rare, they’re everywhere! Over 350 million people globally suffer from a rare disease.

Although these are rare diseases there is still great variety in patient types and patient experiences that can be explored. The rare disease will be a part of a patient’s life, but their attitudes, behaviours and feelings are likely to vary. That’s why it’s so important when researching a rare disease, that what we lack in volume, we make up for in depth.

Therefore, it’s essential with rare disease patient research to consider a number of factors:
1. Be flexible and consider the needs of your patient population; and this is especially true when we think about rare diseases that affect children and adolescents. Taking great care to provide a sensitive approach, we can gain insights whilst maintaining patient wellbeing.
2. Environment: to really understand into the patient experience, context is incredibly important, and in order to see the patient as more than their condition, we prioritise methods that give us a window in to their whole life, day-in and day-out to bring the real patient into the boardroom.
3. Times are changing and often patients with rare diseases connect online. We can access this through research (e.g. methodologies such as online communities and social media listening or through targeted recruitment to capture patient opinion leaders (POLs).

I’m looking forward to this rare disease day. Now more than ever, it’s important to ride the wave, as good research makes the voice of rare diseases heard!


By Lucy Jones

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