Tanya, the IGA's CEO, reflects on the 1st International Working Group on Gaucher Disease (IWGGD) meeting.

Following the IGA’s biennial members meeting on the 5^th and 6^th May in Rotterdam, the Board plus a few other IGA member association advocates and myself hopped on a minibus and travelled to Leiden for the 1^st International Working Group on Gaucher Disease (IWGGD) meeting.

Biljana, Sara and Kristiyan at the IGA stand

The Sunday evening saw a small reception where delegates from all over the world met, some attending their first face to face meeting since the pandemic, myself included.

Prof Hans Aerts was the meeting host as this is his territory; he works at the Leiden Institute of Chemistry, Leiden University. For the IWGGD board, led by Prof Derralynn Hughes, this was their first face to face IWGGD meeting since the new board was appointed in 2019 in Clermont-Ferrand, France. Since the new board was established in 2019, they have been working hard with the development of 6 working groups, all dedicated to developing guidelines, improving knowledge, setting standards, organising webinars, the establishment of a new website and hosting ethical workshops with the IGA on issues important to the Gaucher community. Members of the IGA and their members are involved in many of the working groups and actively contributing to the work of the IWGGD.

The IWGGD ran from the Monday 9^th to Wednesday 11^th May and covered patients’ perspective, clinical spectrum; Laboratory: Genetics and Biochemistry; New Development and Young Researchers; GBA deficiency beyond Gaucher disease: Parkinson’s disease; Therapies and Clinic: present and future. Each session was chaired by a member of the IWGGD. Approximately 100 delegates attended the meeting in person, with a further 60 participating virtually from all over the world.

Tanya presenting

The Monday morning was dedicated to the IGA session and Patients’ perspective. The IGA kicked off this session with an overview by Chair Biljana Jovanovic on the work of the IGA and then I facilitated a one-hour session on medicines procurement.  The purpose of this session was to highlight the challenges patients are experiencing accessing treatment for Gaucher disease because of increasing pressure on government health budgets, resulting in patients and often doctors having little or no say in what treatment they can receive or prescribe.

The session invited four speakers that represented four stakeholders in the procurement process, the caregiver, the patient advocate, the clinician, and the payer. Each of them shared their experience, the challenges they face in the treatment procurement process and shared some thoughts on possible solutions.

The IGA is becoming increasingly aware that the procurement of the cheapest treatment is becoming more and more of an issue for our community; we are aware of it happening in many countries including Peru, Mexico, Ecuador, Iran, Columbia, and Ukraine to name a few. In some of these cases we are talking about licensed treatments such as Cerezyme, Elelyso and VPRIV, but also the non-comparable Abcertin/Asbroder, which is not a biosimilar as it has not been through the biosimilar regulatory pathway.

Vesna with her volunteering poster

We recognise and it is important to say that just because a     treatment is cheaper, we are not saying that it is not a safe   treatment or efficacious. The issue is that patients are being told   that they will be switching to another treatment without a say, and this is causing anxiety. What will happen if there are any side  effects or an issue with efficacy for individual patients, what will   their options be and at the end of that procurement period, will   they have to switch again to another treatment if it is cheaper?

As expressed by our caregiver and patient advocate, this is having   an impact on their emotional, and psychological   wellbeing.

 Our desired outcome of this session is to seek to agree a strategy   to work as a global community to provide guidance and   information that can empower patients and patient advocates to   communicate with their governments and pharmaceutical   companies to make sure the decision makers have the   information and facts to make the best decision for their community, and the patient voice is heard and involved in treatment decision making. The IGA made a call to action at the end of the session. The IGA working with the IWGGD will seek to:

1. Develop switching and monitoring guidelines that can be used by patients to take to their clinicians to ensure if they are made to switch that this is done in the best and safest way possible for the patient and gives them the information to request a certain level of care, and for the clinician to use as a tool.  
   
   
2. A statement from the IWGGD and IGA regarding best practise for switching to empower patients/patient advocates to be able to engage in a dialogue with the health provider.
   
   
3. The IGA will develop a paper on biosimilars and other non-comparable drugs such as Abcertin that outlines what they are, and an IGA statement on biosimilars, what should be considered by the patient when faced with this situation i.e., monitoring, what standards we expect to be adhered to when Governments are considering procuring biosimilars.

It was great to be around many familiar faces and friends over the 3 days. There were an increasing number of young researchers at the meeting and many of the prizes for the best poster went to these young persons, including the IGA’s award for ‘co-morbidities and a better understanding of the impact on the patient’s quality of life’.

The vibe at the meeting was relaxed, lots of discussions, new ideas developing, questions and issues being discussed, and collaborations being formed.  During the presentations, lots of questions were asked of the presenters and opinions shared, the atmosphere was extremely welcoming, and the IGA and its members were welcomed and included at all stages.

No venue was set for 2024 but the IGA will be there, and we are excited and looking forward to working with the IWGGD and its members over the next two years on multiple projects.

Tanya Collin-Histed

IGA CEO