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Swii.ch Health - Patient centricity for real thoughts from ecrd

Patient Centricity for Real – thoughts from ECRD

A couple of weeks ago, I stood in the large, vacuous lobby of an exhibition centre in Vienna, waiting to receive my standard issue hessian bag and free pen. I was about to enter a conference for the next couple of days of my life. To all of us who are seasoned conference attendees we know the score; we scan the horizon and take in the tables awash with glossy brochures. These are usually manned by individuals who are hopeful for human engagement during what can be, a lonely time, alone, on an exhibition stand whilst the sun shines brightly outside. We eagerly down load our conference app hopeful that new ideas and insights will bring change and vigour to our careers.

We could be anywhere, in any city, in any country and let’s be honest, in such a sanitised environment and often removed from real life, belong to any discipline.

But this time it was different. This experience felt real and congruent from the start, and I share below my brief thoughts and observations from the European Conference on Rare Diseases and Orphan Products (ECRD) held in May of this year in Vienna.

As I entered the hall I was immediately immersed in conversation with people affected by rare disorders, their families, those who support and treat them and representatives from drug and technology companies all working to bring solutions to a fragmented and often hard to define area of medicine.

Vinciane Pirard from Sanofi Belgium highlights this in her opening message, “The ECRD meeting is a special moment for the rare disease community as it fosters collaboration between all stakeholders in the area” The conference was described as “Rare Diseases 360 degrees, collaborative strategies that “leave no one behind” and it was immediately clear that this collective of people “walked their talk”.

The rare disease landscape has undergone dramatic changes in the last two decades in terms of recognition as a public health issue in both the EU and US and even further afield. It was clear to me that this spirit of collaboration between stakeholders at a Global level has driven this movement whilst ensuring the patient and the family are at the heart of the research and care agenda – definitely not as a peripheral after thought as in some other therapeutic areas.

Whilst the conference was divided up into several work streams, my time was primarily spent engaging with the digital patient stream. It is obvious that not only do the rare disease community heavily embrace digital communication (since many individuals and their families and connected researchers are scattered across the globe) but that they may actually be the catalyst for the acceleration of digital disruption – collaborative groups of people can bring embryonic solutions to life and scale them quickly. This is born from real human need and an ability to collaborate across boundaries.

This is an area of medicine where knowledge is initially scarce and where patients and their families frequently drive the research agenda and collaborate with researchers as “co-scientists” in their disease journey in true co-creation fashion. Here, I witnessed genuine patient centricity at work at its most intuitive; in the rare disease area there is no time for hype and in some conditions, every month and year of waiting for a correct diagnosis and potential treatment is precious lost time for people and their families.

But without the pioneers of joined up thinking, medicine and innovation, better care cannot develop to enhance the lives for all concerned and a few of the presentations and people stood out for me. A tweet from a leading Duchenne advocate about her potential assistance in helping psychologists truly understand the changing needs and feelings of people with Duchenne and their families resonated as it reminded me of similar discourse initiatives carried out in different areas of chronic care. In these, the gap between Physician, patient and family was anecdotally measured in terms of stigma, and understanding, leading me to think that any “around the pill” services should indeed focus on a narrowing of this gap.

Also of note is Maria Weigl who provided a patient testimonial related to living with mucopolysaccharidosis and for reminding delegates that even within rare conditions each person is still individual with unique presentations, challenges and aspirations; so perhaps a truly personalised approach within rare disease support should be what we all strive for?

Christina Waters from Rare Science stimulated thought and debate for her reinforcement of the essential need for collaboration at many levels bringing science and scientists closer to people via research collaborations to enable people to “own their own data”.

Although a frequent challenge to all communities is harmonisation across countries to allow for digital collaboration, shared records and equality of access to care, rare disease organisations frequently demonstrate their ability to innovate and think in an agile manner. Innovations of interest that packed the congress centre (even on a sultry, summer day) were the use of conversational interfaces to identify patient relevant outcome measures (PROMS) and early stage leading deep learning initiatives to help identify and accelerate the diagnosis of rare conditions; potentially decreasing the time to presentation and ultimately the time to diagnosis.

Several overlapping themes emerged; the need for digital innovation and collaboration and large-scale data sharing and scientific evidence gathering, conversely the challenge of small groups of people in terms of small data sets and evidence gathering which could prove a challenge to areas of artificial intelligence where large data sets “ultimately drive the machine”.

To sum up, the rare disease community which gathered in Vienna demonstrated a very real practice of patient centricity and has much to teach more mainstream leaders in this area of participatory medicine.

The words of Ted Coine* sum up for me the behaviours of this community; “Connect enthusiastically, give relentlessly, and lead bravely. “Do this and the future is yours”

ECRD community, you bravely do this (and more) – the future is yours to shape for us all!

Rachel Jones is a Pharmacist and Behavioural Scientist. With over 20 years as a Health Care Professional and within the Pharmaceutical Industry she now works as a consultant to Healthcare and Technology companies in the area of digital communication and patient engagement. 


She runs the Rare Disease Series** with Rob Wyer from Swii.ch Health that explores the before, during and after of patients and their families who live with rare conditions.

  • * Ted Coine Writer and public speaker, author of A World Gone Social:How companies Must Adapt to Survive
  • ** The Rare Disease Series is a video, blog and interview series that aims to bring together the view points and insights from ALL involved in living with, supporting and treating rare conditions.