Say and do it well, each and every time: rare disease communications and conduct to be #ProudOfPharma

Whether you’re a newbie or seasoned rare disease stalwart, we must never forget the unique close-knit nature of every rare disease community. It’s important to understand the concerns that people feel and voice, when anyone new, especially a global corporation, appears on the scene. As a pharmaceutical company puts developmental and commercial muscle behind a medicine, the hopes and dreams of families are on the table. It is important to get the communications and conduct right. Following seven years working in rare diseases, Aurora shares our #ProudOfPharma 3Cs framework to ensure authenticity in this important area of medicine.

In a 2013 paper (1), Ian Phillips, Norris Professor of Applied Life Sciences, Director of the Center for Rare Disease Therapies, Keck Graduate Institute, US concluded: “The rare disease community has been built on the sensitivities of mothers, fathers and families with their sick loved ones so often going through a difficult and prolonged diagnostic odyssey. The community is largely made up of patient advocate groups and their specific clinicians, and often academic researchers, as the stakeholders. Biotech companies specialising in orphan drugs have built up credibility, compassion and sensitivity to the rare disease community. The community is an asset to work with. The concern is that big pharma companies may be bullish on rare diseases and enter like a bull in a china shop.”

So let’s look at some numbers and the impact… more than 450 medicines for rare diseases are in clinical development, and many more in earlier stage research. Companies are bringing medicines to market for small numbers of patients that could transform the experience of a disease – from overpowered to in control; a better life.

To name just a few, J&J, Merck, Novartis and BMS have established products in the rare diseases market. GSK and Pfizer have created units to increase partnerships with rare disease biotechs, as well as their own R&D in rare diseases.

So big pharma is starting to make an impact in the rare disease space – more drugs, often at high price, are coming to market (I won’t dive into the commercial arguments, they are important and I cover the necessity of the drug development model in my July #ProudOfPharma blog post here). I want to focus on the need for ‘human’ communications and conduct.

What our experience has taught us…

The Aurora team has worked in rare diseases with both the ‘specialised biotechs’ and the ‘big pharma’ at the UK, European and global level for a long while now. The thing to remember is that uniquely in rare diseases, compared to other areas, the success of a medicine can lie in the hands of just a few people – so the way pharma company employees communicate and conduct themselves is important and will always go noticed. More often than not, the words and the way it is delivered will ripple out to others in that community. I’ve seen this happen time and time again – a team’s conduct, and the communications attached to them, can become the global discussion points within just hours of responding to one person, in one country, in response to one question. That doesn’t mean ‘say nothing’ – quite the contrary, the mantra is: ‘say and do it well, each and every time’.

The Prof’s paper is excellent and one that anyone entering this area of medicine can learn from and I’m pleased to report that to date, having worked at the cutting-edge of corporate, franchise and compound-level communications in rare diseases I’ve not seen any fine porcelain trodden under hoof. In fact in my clients I’ve seen great care, attention and a meticulous focus to do the right thing. So the three guiding principles to operate successfully in rare diseases, born out of Aurora’s experience are:

1. Character: Adopt a consultative approach to engage with stakeholders
2. Contribution: Communicate what your company uniquely brings to rare diseases
3. Compass: Demonstrate compassion and sensitivity at every step

It is important to appreciate the sensitivities of this space. Collaboration is key in rare diseases. The community is close knit and the process of drug development and making them ultimately available has to include regulatory-appropriate and open interaction with patients, researchers and clinicians. I’ve seen the 3Cs work in practice, especially when embedded into the culture of the rare disease team. Continually reminding your whole team is key, this mitigates for the inevitable team turnover that happens in all businesses.

Applying the three principles creates a jump-off point for a communications strategy:

  • Character: Include patients and other stakeholders at every stage of compound development (pre through to post launch) – understand how this could, or does already, fit into their care and the local healthcare system that delivers it to them
  • Contribution: Demonstrate your company’s capabilities and approach in rare diseases – show your investment and expertise in trying to bring a medicine to market successfully
  • Compass: Ensure every member of your company shows compassion and is sensitive in all interactions with every rare diseases stakeholder – no slip ups allowed, ever

Getting it right is a joy and makes us #ProudOfPharma in rare diseases.

Give me a shout if you’d like help applying the 3Cs to your communications in rare diseases. It’s not ‘rocket science’, but it is diligent work to do this day in, day out and that is what it takes.

Will we see you at WODC?

If you are in Brussels during the World Orphan Drug Congress Europe in a couple of weeks and fancy a chat then let’s meet up. Two of my team are speakers on the conference programme:

  • Dr Chris Hall: will present on working together, our ‘innovator’ mind set approach to medical education and the importance of engaging the public
  • Stephen O’Farrell: will run a roundtable on collaborating with people with rare diseases to understand their healthcare challenges and to co-create solutions to overcome them

Aurora will be at the World Orphan Drug Congress for rare diseases


  1. Phillips M I. Big Pharma’s new model in orphan drugs and rare diseases. Expert Opin Orphan Drugs; 1 (1): 1-3

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