• Becky Sarson

All Aboard the ‘Rare Disease’ Band Wagon

So it seems the new agency fad is ‘rare disease.’ Weren’t we all specialists in oncology 5 years ago? Now we’re all specialists in ‘rare disease.’ Give me a break!

With over 7,000 diseases classified as ‘rare’ this new positioning ploy is a blatant attempt for agencies to tap in to what they believe are ‘high budgets’ but with a total disregard of what it is to truly understand and work with rare disease communities. Because that’s what it is in rare disease. It’s not a marketing strategy, a clever poster, gimmick, line or image; it’s about ingratiating and understanding the community, building relationships and working alongside patients and their HCPs to add value to their worlds.

Some of the most rewarding (and overserviced!) projects that I have been privileged enough to work on have been in rare diseases, but I’m no expert. Every rare disease is so different, just like the people who have them and the clinicians supporting and treating the patients. These projects, become passion projects, and I believe that’s what it takes to do it right in rare disease: time, relationships, empathy and understanding. It’s insulting to the people working in these diseases, alongside these small communities, that any agency would have the audacity to say they were an ‘expert in rare diseases’.

The experts are the patients, the HCPs, advocacy groups and pharma: the people pushing to research and develop drugs for these patients, all of whom have been working for years with these communities. The ‘experts’ are certainly not agencies who work for 12 months on a campaign, product or asset. 

Initiating Rare Disease Projects

Approaching a rare disease project should never be done in the same way as a more common condition.

Time should be taken to carefully understand the condition, the people who have it, the caregivers and the HCPs that work with the patients. In the case of rare disease the mindset of all involved is totally different to those who are involved more closely with common conditions e.g. diabetes.

The same goes with the environment in which these people work and are cared for. Everything from supply chain, clinical support, training, diagnostics, patient journeys and disease awareness needs to be understood.


The HCPs involved in the care of patients with rare disease are usually few and far between. The communities are small and everyone tends to know one another on a much more one to one basis than in other conditions.

How many agencies actually spend the time though to go and meet with these HCPs? Work with them to understand what motivates them and how their work could be improved in the treatment of these patients? And whilst agencies are rubbing their hands together thinking about all the billable hours this might mean, I’m not. This should not be charged for. This should be a given in understanding the people you will be working with over the next few months and years. Just like agencies wouldn’t usually charge for a ‘chemistry meeting’ you should not charge for this either. Ultimately, in ‘rare’, the HCP will be as much of a client as the pharmaceutical sponsor.

Finally, there is a tendency for agencies to apply ‘common-condition’ marketing strategy to rare disease. Bonkers… HCPs might not even ever see a rare disease patient in their clinic during their whole career. Yet, the assumption is that we need to develop disease awareness training for HCPs – a crude answer to the problem that comes from a lack of HCP engagement with your project team.

Patients and Caregivers

Like the HCPs, patients and carers in this area will not be your standard patient.

These patients and caregivers are more likely to be highly engaged in their disease and in many cases: more of an expert than the HCPs treating them.

Patients are likely to know why they have their disease, the treatments that are available to them and the trials that are currently underway, the prognosis, the pharma companies specialising in their condition, the elements of their condition that they need to keep an eye on, how to self-manage and what it truly means to be a patient.

These patients are having completely different discussions with their HCPs compared with more routine patients. Often they are suggesting treatment regimens, and trials they may be eligible for. So to patronise them by leaving them out of your project is a recipe for disaster. These patients and their caregivers know far more about what it is to have a rare disease than agencies will ever hope to know; don’t underestimate the power of that.


With limited patients means a limited advocacy and support network. But for the rare diseases that do have patient advocacy groups (PAGs) they are an integral linchpin to the community.

PAGs give a place for patients to connect with other patients across the globe helping them to feel less alone in what otherwise could be a seriously isolating condition. Caregivers equally can find support through these groups whether it be information or peers.

Some of the most powerful insights I have gained working on rare disease projects have come from advocacy. Excluding these groups and the people that run them would again be another big mistake. In my experience involving advocacy has been vital in bridging the gap between understanding the HCPs clinical needs and the patients real-world needs.

So What?

Rare ‘done right’ will never, and should never be profitable. These are much needed medicines and associated projects working to support vastly underserved populations of patients, carers and HCPs – not an agencies next cash cow.

So please, stop beating the ‘expert’ drum and start really understanding what it is you are saying because without substance to your claims you’re just insulting communities out there who desperately need these medicines and your help.

1. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases

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