The Voice of People with OI

February’s OIFE Magazine main topic is research and I could have called this editorial “The patients’ voice”. But to be honest, I’m not too fond of the term patients. Patients belong in hospitals. The OIFE represents people with OI and families, who most of the time live active and meaningful lives outside hospital walls.

There are however many ongoing initiatives that include patients in one way or another: Patient involvement in research, patient centred outcome measures, patient reported data, patient priorities in research and I could go on and on. Many stakeholders want to hear our voice these days. Both because they are interested, but also to secure funding or to get approval from authorities.

Some studies are putting the patients to work, by using data that patients are providing themselves. Both the RUDY study and Rare Commons are mostly based on patient reported data. And, why not? We are the real experts, right? Yes, but not always. If terminology is not understood before the questions are answered online, results can be imprecise and sometimes completely misleading. The Adult History Study from the US showed for instance, that many don’t know or understand their own type of OI. And using severity doesn’t necessarily help. A person with mild OI who has severe pain, or who has become deaf, might regard his OI as more severe than an otherwise healthy wheelchair user with OI type III.

“By engaging the end users of study results throughout the research process, we are more likely to focus on asking the right questions, study the outcomes that matter most to patients, and produce the useful and relevant results that are more likely to be used in practice.”

Patient-Centred Outcome Research Institute (PCORI)


So why is our voice important? The most known impact of OI is fractures. But there can also be wide-ranging systemic, psychological, emotional and social effects of both the diagnosis and its treatment. Objective tests or examinations might be inadequate to understand the impact of OI on a person’s health, function or quality of life. This can lead to a mismatch between the person’s priorities and perspective and the efforts of clinicians and other stakeholders.

The ‘patient voice’ can contribute in priority setting as well as influencing health policy in a wider perspective. The patient organisations can encourage clinicians and researchers to do more research in OI. And some foundations provide grants, like Care 4 Brittle Bones and Fundacion Ahuce. As an umbrella, OIFE can contribute with advice, because of our overview and big network. We can also create arenas for connecting and getting different stakeholders together.

So what does the voice of the patients say? I’m pretty sure it depends on whom you ask. The father of three years old Marko with OI type III from Croatia, will give you a totally different answer than Olga 54 with type V from Estonia. And Peter with type I who has recently become deaf, will have other priorities than Martha, with a femur fracture that won’t heal.

How do we make sure that the organisations really represent the voice of people with OI? The simple answer is that we have to work together. The OIFE member organizations need to be informed about the new trends regarding patient involvement. Tools must be developed to gather opinions from the community, like focus groups, queries, panels and webinars. We also need to educate patient experts with OI to meet the constantly increasing need for patient voices in research projects, in European Reference Networks (ERNs) and in consultations with health authorities and policy makers on both national and international level.

“Patient involvement in research needs to be taken to the next level. Rare Disease Day 2018 offers participants the opportunity to be part of a global call on policy makers, researchers, companies and healthcare professionals to increasingly and more effectively involve patients in rare disease research.” – EURORDIS

Rare Disease Day 2018 took place one month ago and the topic was research. Some of you were perhaps attending national RDD events. OIFE was represented at the European Parliament in Brussels where BOND-ERN’s launched a report on diagnostic challenges in OI. No matter what you did – I hope you had an interesting Rare Disease Day. As you all know – it’s only a warm up to Wishbone Day!


Rare greetings from Ingunn

OIFE president


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