Being a patient representative (ePAG) in ERN BOND

Interview with Rebecca Tvedt Skarberg, member of European Patient Advocacy Group (ePAG)

The European Reference Networks (ERNs) are 24 virtual networks involving healthcareERN BOND logo providers across Europe. They aim to facilitate discussion on rare diseases that require highly specialised treatment. One of these networks is the European Reference Network for Rare Bone Diseases (ERN-BOND).

ERN-BOND has selected osteogenesis imperfecta, as an area of focus, to understand the common challenges in diagnosing rare bone diseases and to provide recommendations for improving referrals, reducing diagnostic errors and shortening diagnostic delays. In February 2018 a White Paper on OI was presented at the EU Commission to present the existing challenges of diagnostics in OI.

Rebecca Tvedt Skarberg from Norway is a patient representative in ERN-BOND. She was suggested to BOND’s European Patient Advocacy Group (ePAG) from the OIFE and is now a member of BOND’s Steering Committee. She is also represented in many of the 10 working groups of ERN-BOND.

Who are you and what is your relationship to OI?

My name is Rebecca Tvedt Skarberg. I am 46 years old and live in Oslo, Norway. I have OI type 3 and I have had about 100 fractures. I have a small stature and use a wheelchair to get around.

How long have you been ePAG in ERN BOND?

I actually became involved in ERNs before they were «born». I took part in writing the application to form the network and was part of the establishment process. Inês Alves, from the achondroplasia community, and I worked together with rare bone specialists in Europe on setting up the network. EURORDIS held an ePag election in 2016 where I was nominated by OIFE and eventually elected ePag of ERN BOND. The ERNs were launched in 2017 at a memorable kick-off meeting in Vilnius, Lithuania. I was honoured to hold a plenary talk at that meeting. And looking back now, I do not think I understood what a giant step for rare conditions the ERNs were until afterwards. I sat on that stage looking out at health ministers, prime ministers, members of parliament (MEPs), medical experts, patient representatives, organizations and other stakeholders, talking about my hopes and dreams for the advancements the networks could bring. Everyone had come together to work towards creating better care for people with rare conditions. That day still gives me goosebumps.

What do you do as an ePAG in ERN BOND?

I think it is safe to say I have done a little bit of everything. From the get-go, the learning process has been steep. The networks were brand new and had to find their form and function. That alone feels like rowing a boat, while you are still building it. Our coordinator Luca Sangiorgi often compares ERN BOND to a newborn baby. Everything is new, it is still very dependent on TLC (tender, loving care) and people being hands on putting in the extra effort just to survive. It takes a while to learn how to crawl and eventually run! I am a member of the steering committee and have been in and out of several of the working groups. I attend video meetings where we map out next steps for building educational resources, for creating guidelines, for clinical trials etc. The last year I have invested a lot of time in understanding how registries work and how we best can build EuRR-Bone, our European registry on rare bone disorders. I have always advocated for patient representation and always looking for ways to make the patient voice heard.

How much time do you invest?

This is almost impossible to answer. If I wanted to, I could easily let BOND become my full-time job. However, for obvious reasons I still need my paid job, I do have family and friends, I have health issues and a life. Therefore, I try to balance my days as best I can. Sometimes, I must admit I am quite off balance, and find myself up half the night reading emails or polishing presentations. Luckily my husband is very supportive and never complains. He also has a bone condition, much rarer than OI, so we share the drive for more information and knowledge.

What kind of skills do you need to have to become an ePAG?

I think it is important to be interested in rare bone conditions, both your own and others. You should know the basics and be ok with having OI. If talking about OI puts you on an emotional roller coaster, it might be too tough becoming a patient representative. To help others, you need to help yourself first. Secondly, I think it is good to be able to speak, read and write English. The language barrier in Europe is real and to be able to contribute to discussions, English is essential. I wish this wasn’t that important and that translation technology was further developed, but this is where we are at today.

Also, it is good to have some insight into the eco system of rare disorders; research, pharma, data, authorities, structures, networks and organizations. How do they all fit in as pieces in the puzzle? Although it is good to have a clue about these topics, these are things you will learn as you go along. You don’t have to be an expert to get involved, you just have to be willing to get onboard. It is far more important that the organization you represent has your back and people you can turn to for advice and guidance. Being a patient representative is about representing a group or organization. It is not about private agendas and advocating for yourself. You must want to make a difference for your group and not be afraid to speak up about it.

What is the biggest challenge of being ePAG in ERN BOND?

Difficult question…. One challenge is the feeling that you never have enough time or that you don’t understand enough about the different topics or projects. In the beginning I had to read the same documents numerous times before I felt I understood anything. Being asked questions about research tools, about clinical pathways, PROMS and CROMS can almost wipe you out. Especially if you have just come home from work, or you are recovering from a fracture. Sometimes I wonder how I got myself into all of this. But then I remember all the people I get to meet and discuss rare bone conditions with. The world is becoming smaller and together we can make a difference for people with rare conditions. The thought of that is refuelling in itself.

Can you mention an example where input from an ePAG changed the course of action in ERN BOND?

I try not to look at it like a «battle of wills» between patient representatives and health care professionals. It’s not a war, it’s a collaboration. My experience is that all members of BOND are very eager to hear what we have to say. Many times, there are no rights or wrongs. It’s more about putting priorities on the agenda. We all know that paediatric care is far better than adult care. Therefore it can be important to foster the adult perspective. OI is about more that broken bones, measuring height and straightening legs. Adults with OI face various challenges previously neglected in health care services or when new projects are funded. Lung dysfunction seems to be the main cause of death in OI, so why do we not have more focus on lung and pulmonary care? Hearing loss is a major contributor to feeling isolated and depressed, so why are there not more audiologists and psychologists onboard amongst our medical advisors?

There has been a lot of focus on bisphosphonates as if it has revolutionized OI, but the jury is still out on the long term effects of OI. Is counting fractures really a good measurement on how effective a drug is? And while talking about medical treatments, we still do not have access to treatment that affects the root cause of OI. We are still putting band aids on symptoms while in other disease areas they have come much further. Most importantly, I don’t think all OIers are on a hunt for The Cure. They value their lives just as they are, but want to help manage care, pain and everyday life as they grow older. These are some of the messages we try to get across.

What can a patient with a very complex case do to get access to expert advice from ERN BOND?

ERN BOND discusses complex cases through an IT platform called the clinical patient management system (CPMS). It is a form of second opinion you can ask for through your health care provider. Individuals cannot access CPMS themselves, but have to go through the doctor in charge of care. That doctor, if not already a member of BOND, has to access the ERN through a clinic in your country that is a part of the ERN structure. In CPMS the doctors can share x-rays or test results and discuss the findings and treatment options. The idea is that instead of the patient traveling to find the expertise the information «travels» through the secure IT structure. The system meets the requirements of GPDR and your data is shared only after you have given written consent. Please note that each country has specific rules and arrangements for the referral of patient cases to ERNs. (Editor’s note: Some countries are for instance not connected to ERN BOND via the CPMS system). See more info on ERN BOND’s website!

What are the most important activities ERN BOND is working on at the moment?

We will be seeing a major expansion of BOND from 2022 as we will be welcoming many more members onboard when the second call from the EU is finalized. We might even double in size if all applicants are accepted. This means a new boost, new people, establishing new working groups and distributing new roles. We are constantly talking about how to prepare and set the stage for this next step in develop-ments. We also need more ePags onboard. We are constantly trying to reach out to rare bone patient organizations to spread the word.

Do you have any messages for the readers of OIFE Magazine?

I think my main message is to get engaged through your OI national organization and ask them how you can become involved. Progress does not happen by accident, and nothing is ever given. We have voices, our experiences are real and together OIFE will continue to create change in the field of OI.

Ingunn and Rebecca won the prestigious Black Pearl Award for their OI-work in 2021

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