Meet Hanka Meutgeert: Advocate for Metabolic Disorders in the Netherlands

Annamarie Dillon | June 26, 2012

Annamarie Dillon and Gisela Linthorst of Genzyme speak with Hanka Meutgeert, founder and Executive Director of VKS, a Dutch organization established in 1994 that supports families affected by metabolic disorders. VKS will host the International MPS Symposium in the Netherlands from June 28 to July 1, 2012. VKS, or Volwassenen, Kinderen en Stofwisselingsziekten, is translated to English as Adults, Children and Metabolic Disorders (www.stofwisselingsziekten.nl).


How did you become involved in advocacy work?
In March 1993, our son was diagnosed with a very rare metabolic disease called Aspartylglucosaminuria (AGU), which is a lysosomal storage disease that is biochemically similar to the MPS (mucopolysaccharidoses) diseases. In those days there was no Internet and it was difficult to find information, and it occurred to me that if was difficult for me, then it must also be difficult for others.

I came into to contact with a metabolic patient organization in England, which is now called CLIMB (Children Living with Inherited Metabolic Diseases), that provided information and connected families with metabolic diseases. This inspired us to start VKS—Volwassenen, Kinderen en Stofwisselingsziekten (Adults, Children, and Metabolic disorders)—in 1994, to support 60 families and 40 diseases. Since then, we have now grown to support 160 metabolic diseases and over 1,500 families here in the Netherlands.

What are the biggest challenges your organization faces today?
Staying in existence! Since 1998, we were lucky to have had financial support from the government; however, we are now suffering from cutbacks in funding. This means we are now more dependent on public funding and it's difficult to find new sources of income. In some countries, like in the U.K. and U.S., patient organizations are registered as charities, which can help with fundraising efforts, but this tradition doesn't exist in the Netherlands. While we continue to provide as much support as possible for families, it is less than what we want to do. We cannot ask families to pay, as they often are struggling financially with the associated costs of having a sick child.

What advice would you give to someone who was thinking about establishing a new patient organization?
The most important advice I could give is to be willing to work with existing organizations. What we see today are parents establishing patient groups with the name of the rare disease of their child. I can understand why parents want to do this from an emotional level, however I believe that if you really want patients to profit, then it is better to have a bigger picture of both needs of other families and ways to really stimulate better care and more research.  Working from your own perspective only is  ultimately less effective. We need to work together at both national and international levels in order to continue moving forward. The challenge is to find those goals that connect us with science and industry.

How has the advocacy landscape for rare diseases changed since you first became involved?
A lot! Back then there was no specific interest in rare diseases. Genzyme had just launched the first innovative medicine to treat Gaucher disease. This triggered interest because it was different to the "normal pharma" approach—there was no large patient population, just a small group being treated with a new method to target the disease symptoms. On an EU level, it helped us secure a foothold for specific development of medical products, with the communal action plan on rare disease in 1999.

In 2000, we saw the first ever EU Orphan Drug Act, and we did our best to help bring this into the world. It was a difficult time for lobbying and advocating; however in 2001 we saw the EU approval of the first two orphan products for a metabolic disease. Over the next ten years, under this Act, there have been many new approvals but only 18% have been for metabolic diseases.

In the beginning there was hope, as these regulations were designed to stimulate rare disease research. We now face a new threat at the EU and national level, as there is a growing feeling among policymakers that we have done enough and are now finished with our work.

VKS is advocating for the development of a Dutch National Rare Disease Plan, and we are trying to access the right people in government and other institutes to share our view. A lot has been done but we still have more to do. We think that there is a need for concrete plans to protect the expertise in orphan diseases, by forming centers of excellence, where patients can be sent to the right expert physicians and researchers and to ensure proper long-term data collection.

What do you think is most important in collaboration with an industry partner?
The interest of the patient has to be the priority on both sides. It's natural for patient groups to partner with industry, as they both have the same interest but for different reasons. Early diagnosis, availability and access to reimbursed treatment, and new developments in the field are some of the many shared interests. Partnering will not work if we don't keep patients at heart.

In a few weeks, The 12th International MPS symposium will take place here in the Netherlands. Can you please tell us more about this meeting and your involvement?
This is a unique meeting for not only scientists, but also for families and adult patients. This year it is being organized by VKS working closely with experts for the scientific content. This approach is not common in the scientific world. The scientists, physicians, and families all participate in most sessions, as the patient community is deeply involved in this scientific field. There are a few sessions just for families and a separate children's program.

The meeting is a model for other rare disease communities as it stimulates the active exchange of knowledge on MPS and related diseases. The whole community is involved: patients and families and the medical community, supported by the companies.

The VKS submitted a bid to host this event in 2008, because we had the foresight to understand the importance of 2012, for the development of treatments for MPS diseases and related diseases like Niemann-Pick.

What was your first reaction when you learned that you had won the bid?
Let's make it the best one ever! It was announced 4 years ago at the conference in Canada and the last meeting took place in Australia, so it's been 6 years since the meeting has been in Europe. We certainly feel the pressure to make this year's meeting both international and European and bring added value for all participants—we will soon see if we are successful!

Can you tell us more about the collaboration between the VKS, physicians, and industry in the preparation for this meeting?
We reached out to both centers of expertise in the Netherlands—Erasmus Medical Center, Rotterdam, and the Amsterdam Medical Center—and asked the clinical leaders to co-chair the international scientific committee. The meeting would not be possible without the loyal sponsoring of the three companies active in the MPS field.

Are there any reflections or lessons learned you would like to share with the hosts of the 13th International Symposium in Brazil in 2014?
I am very grateful we can benefit from the GIVE (Genzyme Invests in Volunteer Efforts) program, through which a large number of Genzyme employees have volunteered to support the children's program. Without this support, attending families might not be able to fully focus on the meeting as their children often have more care needs. The Brazilian hosts could learn from this successful collaboration.

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