This past Summer was a hot Summer for Pompe patients and everyone else involved in rare diseases in the Netherlands. This was not because of the climate, but because a concept report was leaked to the Dutch News Broadcasting Service. The concept report of the Health Care Insurance Board advised that the enzyme replacement therapy for (newly diagnosed) patients with Pompe Disease (late onset) not longer should be reimbursed. This news was brought into the world on a Sunday evening while everyone was watching the Olympic Games. It doesn't need to be explained that this caused a lot of fear, anger and uncertainty among patients and a lot of disbelief among the general Dutch population and politicians. How is it possible not to reimburse a treatment that has been proven to be effective for a disease and for which no other alternative exists?
Pompe patients responded widely in the media to this advise. The daily news on television was organised by a Pompe patient who in turn was in direct contact with the Dutch patient organisation Spierziekten Nederland (VSN). The topic 'Pompe Disease' was on the news almost every day. On the website of the VSN a lot of information about Pompe Disease was available. This also included the statement "It works!" the VSN and patients kept saying. The VSN also showed short videos of people with Pompe Disease and the results of treatment.
At September 21 2012, three Pompe patients represented the VSN and their members during the public hearing of the Health Care Insurance Board where they provided information based on their experience with the treatment. Again the press paid a lot of attention to the topic. Media attention was also generated by the VSN by sending out a letter signed by the Dutch Neuromuscular Research Centre (ISNO), representing over 200 neuromuscular specialists and researchers in the Netherlands, stating that they do not agree wit the concept advise. They also stated that the method the Health Care Insurance Board has been using to evaluate the effectiveness of Orphan Drugs is not the right one.
Prof. Dr. Ans van der Ploeg advocated strongly that the treatment for Pompe Disease is effective, not only based on experience, but also based on scientific evidence. She informed the Health Care Insurance Board about the effectiveness of the drug, the results of the LOTS study, the follow up study and the Pompe IPA Survey.
Yann Le Camm, CEO of EURORDIS (Rare Diseases Europe) also was invited to present his opinion during the hearing. His statement, covering the European perspective, also contributed to a change in opinion with the Dutch Health Care Insurance Board. The letter he read during that hearing can be read here.
At November 29 2012, the Health Care Insurance Board, did send their final advise to the Minister of Health to keep reimbursing the treatment for Pompe Disease and set up a seperate fund for Orphan Drugs (treatments for rare diseases). The VSN therefore asked the Minister of Health to develop a method to study and evaluate drugs for rare diseases specifically. It is essential that discussions like this one don't need to be repeated and that the development of new effective treatments can be continued.
The Minister of Health will come forward with her answer in the upcoming weeks. You will be informed about further developments in the future.