Charitable access success

Atif Taha, Qatar

Six years ago, my family received devastating news. Two of my three children were diagnosed with Gaucher disease, a very rare condition that affects 1 in 50,000 people around the world.

The fact that the disease has viable, albeit lifelong, treatment is a great blessing. The big problem, however, was the exorbitantly expensive cost. Figures that are out of reach of any family in the world except, perhaps, for the richest 0.1%. And we have not one, but two children requiring this medication. As far as we know, no Gaucher patient in the world is paying for treatment out of pocket. During these very testing 6 years we managed to secure intermittent doses of the medication for our eldest child who was showing more advanced symptoms. The other child was left untreated. A decision no family should have to make.

We got in touch with Mrs. Tanya Collin-Histed from the IGA who lobbied on our behalf and sped up our application to Sanofi, the drug manufacturer, to receive the medication. Unfortunately, our application was rejected multiple times on the grounds that it did not meet the criteria per Sanofi’s policy at that time. But Mrs. Collin-Histed, Tanya, did not give up. She set up meetings for us with the lead Gaucher specialist in the UK who wrote medical reports to support our case. Throughout these 6 years, Tanya never stopped advocating for our kids. They were much more than just case files or data points to her. They were two promising young children that deserved a chance for a healthy life.

One exceptionally hot and humid late June afternoon, we received the long awaited news. We were informed that, following a change of policy, Sanofi is now offering the drug, free of charge, to our children as long as their conditions require. Apparently, Tanya’s relentless advocacy, through the IGA’s channels, finally managed to move the mountain of impossibility. She was instrumental in Sanofi’s policy update.

No words can express our gratitude for what Tanya and the IGA team has done for our family. The six years of tireless campaigning on our behalf. They believed that our story could have a happy ending, and in doing so gave us hope.

The IGA always say "Rare but not alone", they mean it genuinely and we lived it. The IGA inspired us to start a new chapter in our life. Supporting ones in need and lending a helping hand is our ultimate goal. We started with our home country Sudan with the IGA full arsenal assisting us.

Tanya adds:

I have been privileged to be a member of the Gaucher community for 27 years as mum to Maddie and 25 years as a patient advocate. Every day there are highs and lows, and you meet amazing and strong people. Some days are more special and 30th June was one of those days. Working in collaboration with Sanofi and in particular with Bonnie and Jasmina these two siblings from Qatar were approved for treatment through Sanofi’s charitable access programme. These cases have been a huge challenge but the first rule of advocacy is don’t give up and never take the first answer given if it’s NO.

The IGA and Sanofi have worked together on many cases where access to treatment has been a challenge. Sanofi’s International Cerezyme programme has treated and continues to treat hundreds and hundreds of Gaucher patient around the world. I would like to congratulate Sanofi for recognising that due to demand and a constantly changing world, they have been open and responsive to reviewing their charitable programmes and we are delighted that these two siblings were able to meet their criteria and be approved for treatment.

Sadly there are still hundreds of Gaucher patients around the world without treatment and as a community we still have so much to do.

 Visit our website for more information on charitable access 

 

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

Reflections on my first IGA biennial members meeting

By Harry Albright

Communications and Campaigns Officer

Photos by Francisco Carreiro

On the weekend of 6-8 May, I attended my first in-person IGA board meeting since I joined the organisation in July last year. This was followed by the biennial members meeting,

IGA board and staff

We travelled to Rotterdam in the Netherlands where the one-day board meeting was held on the Friday. It was a great experience to finally meet some of the people that I have been working with so closely over the past 10 months. It was also good to connect with the new board members who joined us. Unfortunately, due to visa issues, a few board members had to join online.

The board reviewed the work of the past few months, including the newly launched GARDIAN Registry, and prepared for the biennial meeting that was to happen over the next two days. The dedication of the volunteer board members is impressive. They all work hard and are committed to making the world better for Gaucher patients and their families. Without their input, the IGA would be able to do only a fraction of the work it currently does.

In-person attendees of the biennial meeting

On Saturday and Sunday we were joined by over 40 people from around 30 countries, most in-person with a few online, for the biennial members meeting. This is an opportunity for members of the IGA to connect, give and receive updates on their work and the work of the IGA, take part in workshops and hear presentations from pharma companies and others working in the rare disease field. This time we heard from Dr Lucy McKay from Medics for Rare Diseases and Barbara Sjouke MD PhD from the Amsterdam University Medical Centre, who spoke about gene therapy. Takeda, Sanofi, Freeline and Prevail shared the industry perspectives.

We also took time to recognise the many volunteers who serve the IGA in many capacities, including the Regional Managers and others who work so hard on our behalf. Volunteering with the IGA is a great way to give something back to the Gaucher community, and it doesn’t have to be a huge or long-lasting commitment. There are often one-off jobs, like serving on a time-limited working group (planning International Gaucher Day for example), as well as the more substantial roles like serving on the board.

IGA CEO Tanya Collin-Histed
giving a presentation on GARDIAN

Again, it was clear how happy people were to once again be able to meet face-to-face, something that hadn’t happened since 2018. For my part, it was good to meet so many people who are doing amazing work supporting Gaucher patients in their own countries, and to hear about their experiences and concerns, especially in the field of communications and campaigns. I had many great one-to-one conversations, and I certainly learned a lot. It was the first time I was really able to experience the sense of community amongst Gaucher patients and their families, and the willingness of so many to support and encourage others, especially those who are newly diagnosed and who are looking for answers and treatments.

The people who attended the board and biennial meetings are amongst the most engaged in doing this work, but it is clear that many people don’t have the time or energy to be more active in the IGA’s work, or they have other priorities in their lives. Not everyone needs to be actively engaged. If you are one of those people, do consider at least staying in touch through newsletters, social media or other means, so that your local Gaucher group knows about you and you can contribute to discussions or requests for input to help serve everyone better.

Together we are stronger.