miércoles, 16 de octubre de 2013

Positive Dutch decision on Pompe and Fabry disease treatments « Rare Disease Blogs.

Positive Dutch decision on Pompe and Fabry disease treatments « Rare Disease Blogs.

Opinión internacional sobre las enfermedades raras y los medicamentos huérfanos
La reciente decisión de Holanda de seguir reembolsando los tratamientos para Pompe y Fabry.

Positive Dutch decision on Pompe and Fabry disease treatments

On 3 October, 2013, the Dutch Minister of Health, Edith Schippers, announced that the treatment of patients with Pompe disease and Fabry disease can be continued – at least until 2016 for Fabry and 2017 for Pompe. This decision brought to an end a long period of waiting for these patient groups.
In the summer of 2012, a large public debate took place following a negative opinion from the Dutch Health Care Insurance Board (CvZ) to no longer reimburse the treatment of these patients, except for the infantile variant of Pompe disease, because of their unacceptable cost-effectiveness. In a meeting of the appraisal committee (ACP) of the CvZ, this decision was overturned, and the Minister of Health was advised to go on with the treatment. In early 2013, the Minister responded with the answer that she wanted to continue treatment in 2013, but that she would start negotiations with the companies (Genzyme and Shire) producing the biological treatments for the two diseases on the price of their products.
Price arrangements and future treatment evaluations
Price arrangements have now been made with the two companies, which will not be made public. A similar procedure of price negotiations is performed in the United Kingdom as well as the Netherlands when governments decide that price re-negotiations are necessary because of too high prices or too unfavourable cost-effectiveness analyses. The formulated periods until 2016 (for Fabry) and 2017 (Pompe) are chosen, because the Dutch government wants more treatment evaluations of these products to see how patients will respond to treatment in the long run.
The letter from the Dutch Minister of Health is carefully worded and acknowledges the expertise already present in the two reference centers for these diseases, the Erasmus MC in Rotterdam for Pompe disease and the AMC in Amsterdam for Fabry disease. Ms Schippers also expresses gratitude in her letter to the respective patient groups for their patience in waiting for this decision. Both patient groups – as well as the physicians – have responded positively to the letter from the Minister.
Reference is also made to the National Plan for Rare Diseases that will be made public later this month in the Netherlands, followed by a two-day conference (organised by www.vsop.nl), as part of the total European Plan for Rare Diseases. Other important initiatives are also mentioned, including E-Rare, Orphanet, and special databases developed for rare diseases, et cetera.
During the FIGON Dutch Medicine Days on 1-2 October in Ede, there was also a ‘look back’ discussion on the Pompe and Fabry discussion that took place in the Netherlands over the last two years.
Un-transparent price negotiations
In my own presentation for this debate, I discussed the price negotiations. Personally, I’m not that happy with negotiations that are not transparent for the public. It’s quite a strange situation that we had a large public debate on this issue, which now ends with an un-transparent message about the price negotiations. From a competitive point of view this can be understood, but it will lead to the public perception that the prices were indeed too high from the side of the companies.
In my opinion, the price of new medicines coming on the market, and especially those for rare diseases, is also heavily influenced by the ongoing public and governmental requirements for clinical research, authorisation and reimbursement.
Ongoing administrative requirements
When we look at the European situation over the past 10-15 years, we have seen a new European Clinical Trials Directive around the year 2000, a revised European Clinical Trials Regulation being discussed now, a new European Animal Research Directive being implemented now, new transparency requirements for clinical trial data from the EMA being discussed now. In the last ten years we have seen the rise of the HTA hurdle requiring cost-effectiveness analyses in the final reimbursement phases of drugs. Especially, considering that Europe now has 28 Member States, these procedures are not harmonised in a way that would allow us one dossier for all Member States. So, a drug manufacturer or an academic pan-European study project has to deal with different situations in the different Member States and in different languages as well!
All the work that is needed to fulfill these administrative requirements is incorporated in the price of medicinal products coming on the market. For commercial products it is possible to incorporate these costs in the price, but for academic research or spin-off biotech companies from universities it is much more difficult to earn this money back.
So, in my opinion, there is an urgent need for the community of patient groups at large, and for the rare disease community in particular, to start the debate about the large administrative burden that is reflected in the price of orphan drugs.
Especially in a period of economic crisis, it is not acceptable that the cost of regulatory requirements of new drugs is rising, while access to these drugs is becoming more and more difficult because of its often high price.
This Catch-22 situation has to be stopped! Act Up now!
Related blog post: Decision to continue reimbursement for Fabry disease and Pompe disease in the Netherlands is very good news for patients and doctors by Carla E.M. Hollak

about the author

Dr. Cees Smit was born with a rare disease, severe hemophilia. He has been a patient advocate for more than forty years and received an honorary doctorate from the college of deans of the University of Amsterdam in 2003 for his work on hemophilia, patient participation and biotechnology. He is also a Policy Advisor at EGAN (Eur Genetic Alliances’ Network)

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