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Sept 21, 2012: EURORDIS intervention at Dutch Healthcare Insurance Board public hearing « Rare Disease Blogs.

Sept 21, 2012: EURORDIS intervention at Dutch Healthcare Insurance Board public hearing « Rare Disease Blogs.

October 1st, 2012

Sept 21, 2012: EURORDIS intervention at Dutch Healthcare Insurance Board public hearing

Amsterdam – 21 September 2012: Intervention of Yann Le Cam on behalf of EURORDIS at the Public Hearing by the Package Expert Committee of the Dutch Healthcare Insurance Board (CVZ) on continuing or not to reimburse the Pompe and Fabry Treatments
(full text)
Dear Mr Boer, dear members of the Package Expert Committee,
Thank you for your invitation to present our comments on behalf of the EU patients’ community on the advice of CVZ about the continued reimbursement of the Enzyme Replacement Therapies Fabrazyme, Replagal and Myozyme for treatment of people living with Fabry or Pompe diseases.
I am the Chief Executive Officer of EURORDIS, the European Organisation for Rare Diseases and the father of a child affected by another rare disease, cystic fibrosis. EURORDIS represent 528 patients organisations from 48 countries, so our view is presented on behalf of the 30 million people living with rare diseases in Europe today.  I am also the current Vice Chairman of the EU Committee of Experts on Rare Diseases based in Luxembourg and the past Vice Chairman of the European Committee of Orphan Medicinal Products of the European Medicines Agency based in London.
EURORDIS representatives participate since 12 years in the Scientific Committees of the European Medicines Agency, including the Committee of Orphan Medicinal Products and the Pediatric Committee; we regularly take part into the design of clinical studies or assessment of data through our participation to Protocol Assistance, Risk Management Programmes and Patients’ view on Risk / Benefit Assessments or re-Assessments; we are representing patients in the EUnetHTA Stakeholders’ Forum. We are highly involved in the development and implementation of EU policies to improve access to diagnosis, care and medicinal products for patients through several EU High Level Committees or expert groups, always involving all or most Member States, including The Netherlands.
That is to say that we know the issues, we know the need of the patients, we know the reality of medical experts and of pharmaceutical or biotech companies, we know the requirements of regulatory bodies, we know the importance of data, we know the concerns and constraints of Health Technology Agencies as well as the ones of the national competent authorities responsible of pricing and reimbursement.
Our two key messages are based on this grass root experience as much as on a European and long term vision:
-        First, CVZ is taking a wrong approach to address a real and legitimate issue:
  • CVZ expectations for long term safety and efficacy data is legitimate. You want to have the clinical data, the measurable patients’ health outcomes evidence to base your decision so to do the best possible use of the limited resources of the health care budget in the Netherlands. That is legitimate and good. We are with you. We support you in that.
  • But the requirements of CVZ for national studies are inappropriate. The specificity of the extreme rarity of Fabry and Pompe disease in a population of the size of one country such as The Netherlands, simply doesn’t make it possible to collect sufficient data for a robust analysis and conclusion on long term safety and efficacy or analysis of sub-populations to be treated
  • Furthermore, the potential decision of CVZ to stop reimbursing these treatments or to continue to reimburse only the patients currently under treatments and not the newly diagnosed patients, is simply highly non ethical and not acceptable neither for patients or for society at large. Such selection of patients based on economic or administrative arbitrary criteria rather than on medical criteria reminds me of the dark years of the 30s, when under the pressure of the economic crisis the hygienic institutions created in the 20s for disabled people where closed because some governments around Europe considered they could not afford to pay any more for people who were not contributing to the nation economic growth. Few years later in the early 40s, we all know how that turned out for disabled people. Let’s be clear: when society and decision makers are abandoning the most vulnerable people for economic reasons, all citizens are in danger, including each of you around this table as well as your beloved ones. Social justice is for society to do more efforts for the most vulnerable people so to deal or compensate their vulnerabilities. Today’s decision is about cutting on extremely vulnerable Fabry and Pompe patients. Who will be next tomorrow?
-        Second, the solutions are at hand for CVZ to address its expectations in a satisfactory way and this is through European collaboration based on information exchanged and coordination between Member States :
  • The Netherlands has officially committed twice to provide access of Orphan Medicinal Products to patients in the Netherlands; when it adopted the adopted in 1999 the EU Regulation on Orphan Medicinal Products and more recently in 2009 when it adopted the European Council Recommendation for Actions on Rare Disease. The Netherlands has committed to develop a National Plan on Rare Diseases before the End 2013 as all other Member States, and the Dutch Minister for Health are renewed this commitment in front of the Parliament of Netherlands in June 2012.
  • An EU legislation on Pharmacovigilance has been adopted, including by The Netherlands, and is being implemented since July 2012. This legislation provides the legal tool for regulators to request post-authorisation safety  (PASS) and efficacy (PAES) studies. The long term safety and efficacy of both Fabry and Pompe treatments can be and will be further studied in this European collaborative effort to document more the value based on real life use data.
  • The EU Committee of Experts on Rare Diseases (EUCERD) has adopted this month a EUCERD Recommendation on improving informed decisions based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow to the European Commission and Member States”. This recommendation has been adopted by the representative of The Netherlands in this EUCERD. This recommendation is putting in place a mechanism of exchange of information between MSs on their assessment of the value of orphan medicines at the time of its development, of its approval and few after its introduction on the market. This mechanism will bring together the EMA agencies (and through them the drug agency of The Netherland) with the HTA agencies through the EU network of HTA (EUnetHTA). This is the way to discuss post-marketing European Evidence Generation Plans and few years later to do common assessment report on the effectiveness or relative effectiveness of Fabry and Pompe treatments.
  • In addition, there an ongoing EU Working Group piloted by the Belgium EU Presidency on the creation of a Mechanism of Coordinated Access to Orphan Medicinal Products. Twelve MSs are participating in this initiative. Unfortunately, not The Netherlands, at least not yet, but I hope that after this Public Hearing, CVZ might join in. The short term objective is to put in place a realistic and easy to use mechanism to discuss the value of orphan medicinal products in an agreed European Transparent Value Framework to improve informed appraisal and decision making on pricing & reimbursement at the MSs level. The medium term objective is to get one step further, putting in place a mechanism  for informed negotiation on pricing and reimbursement at European level; A European approach will enable to discuss on the base of a more significant critical mass of patients to be treated, aiming for economy of scale, with prices or budget or cost of treatment per patients based on value and volume.
  • We are convinced that if The Netherlands take an active part into these three new collaborative approaches between MSs, CVZ will have clear answers to its expectations of long term safety and efficacy data together with better targeted patients to be treated + improved medical practice + better prices or budget, so in short, better patients health outcomes and better value for public money.
  • The Netherlands are highly respected across EU for their medicine assessments and health technology assessments. This Expert Committee of CVZ is setting the pace for a higher level of requirements which is echoed in other Member states and in EURORDIS. I am convinced that The Netherlands can play one of the lead role in Europe to improve the system toward more and better evidence generation. What CVZ needs to recognize is that the marketing authorization of an orphan medicine is not a sufficient on-off switch to provide access to patients rather that the specificity of orphan medicines which are intended for rare and highly heterogeneous patients population, is to require a continuum of evidence generation before and after the marketing authorization, and that this can only be obtained through the critical mass at European level.
Europe is watching this meeting today through media, internet and networking.
We respectfully ask you to hold your potential negative decision.

A negative decision will have wide implications for Europe and even abroad and for all RD patients.
Patients cannot be the one to be blamed for the current lack of data. The one to be accountable are the companies and the Member States.
These drugs for Fabry and Pompe patients are working, they are saving lives for the one affected by the most severe forms and making their life longer, they are improving the quality of life of all affected.
It is morally and politically wrong to condemn the patients and their families just to save money while The Netherlands, the other MSs in EU, the EMA and the two companies who are the marketing holder of these three products have not explored and used all the possibilities, most of which are new possibilities, of generating long term data on safety and efficacy.
By doing so, you would pre-empt the possibility to generate such data. And your decision will be interpreted not as a tough and wise decision but as a shamed injustice, based only on lack of willingness to find acceptable solutions and to collaborate at European level, a lack of care for patients and only focus on economy and saving money at the expense of patients’ lives.
Yann Le Cam
Chief Executive Officer
EURORDIS – The European Organisation for Rare Diseases
Question & Answer:
-        Q: I resent your reference to the 30s as the situation is not comparable
A: I understand your resentment, my analogy is strong. My point is to say that when decision makers are deciding to deprive part of its citizens among the most vulnerables in their population, like Fabry and Pompe patients are in The Netherlands, from reimbursement of care without exploring all possible options only to save money, the whole population can really worry because each citizen is in danger of similar unfair decision made on the ground of economy
-        Why should we provide access to medicines for Fabry and Pompe patients at the expense of providing access to treatments for more common diseases?
The rare disease patient movement in Europe defend a fair and balance solidarity. I never oppose the need of one patient to another, I lost half of my family from different cancers, my friends have been affected by AIDS, we have diabetis and Alzheimer in the family. We cannot oppose the suffering of cancer patients to Alzheimer patients, of lysosomal storage disease to diabetes. All patients affected by frequent or rare diseases both deserve a fair access to treatments when they are safe, effective and affordable. The only question is how to make it sustainable with the right approach based on good medical practice so to do the best use of existing resources.

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