Rare Disease Blogs

Note: this is an update to the following blog posts: Part I, Part II

During the week of August 22 2012, the Pompe and Fabry situation in the Netherlands led to more general discussions on first, how to redress the demand on the health care sector and second, what is the reason of the high cost of drugs and in particular medication for rare diseases. Some of the contributions are very helpful because they addressed the issue of what is ‘appropriate and necessary care’ and what not. Certainly in the Netherlands, there is an upward trend to an increasing demand of all kind of medical services. Some people certainly blame the privatization process of health care responsible for that increasing demand. Although there have come forward several good proposals to try to bend this curve, in practice it will not be a very easy task and will need some time to implement it.

The second point, the background of the pricing process for drugs, has brought forward some unrealistic proposals. And more in general, one could say that the level of knowledge about the drug development process is rather low. Specifically, for orphan drugs the situation is even worse. The danger of all this is, that the pharmaceutical companies are scapegoated and become the ‘black sheeps’ in this discussion.

With general elections in the Netherlands on September 12, health care has now become a large topic in the political debates and the rare disease discussion on Pompe and Fabry has accelerated this. Some journals are now asking politicians specific questions on how to lower the cost of health care. So far, most politicians only give very general answers. But when time evolves, I expect that these questions will become much more specific and will also address rare diseases. Although already almost all politicians – from right to left – said that patients with Pompe and Fabry should get a guarantee for their future treatment things can change, especially after the elections. The same can be said for the minister of Health, although her words were chosen rather careful. When the treatment has shown to be effective, then the drug should be reimbursed. ‘To be effective’ and this is exactly where the debate on September 21 will be about. Spetember 21 is the date, where the advice not to reimburse any longer the drugs for Fabry and Pompe (except the classical variant among newborns and babies) will be discussed in the appraisal committee (Adviescommissie pakket of CvZ).

There are at least two occasions the coming weeks, where the rare disease policies will be discussed extensively. The first is a meeting on the Dutch National Plan for Rare Diseases on the 28th of August in Amsterdam. During this meeting the first concept of a national plan will be discussed and it is clear that several of the recommendations of the Fabry and Pompe researchers to go much more European with rare disease research in the Netherlands will be discussed there as well as a clear task division between the several academic hospitals on how to centralize the care for people with rare disease in expert centres. For more information, see www.npzz.nl.

The second occasion will be on September 5 also in Amsterdam, when the VSOP (the Dutch umbrella organisation for people with rare and mostly genetic diseases and a Eurordis member) and the HTA department of the University of Nijmegen will present the results of a one-year study to the effects of stakeholder (among them the patient groups) participation in the insurance health care package of the CvZ (The National Health Insurance Board). In the morning session the results will be discussed with an audience of patient groups and in the afternoon the implications of all this will be discussed with a larger group of stakeholders, among them politicians. So, also here the political debate about Pompe and Fabry will continue. For more information, see www.vsop.nl