December 4th, 2012
Testimonial from Cees Smit on the advice on the treatment and reimbursement for patients with Fabry and Pompe Disease
Intervention of Cees Smit on behalf of EGAN 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
First of all, words of appreciation for the patients of the Fabry Association for your personal contributions and especially your letters that shows quite clearly the impact of the disease on patients and their families.
At the same time, it also illustrates quite nicely the gap between what QALYs and ICER’s means for health economists and policy makers, but that you as patients totally not recognize yourselves in the words and numbers they connect with your illness.
It means once and again that the contribution of personal and collective experiences, the expertise of patients and their families gives a strong added value to concepts such as burden of disease, quality of life, research outcome measures, etcetera. One cannot exist without the other.
At the same time – and that also applies to the input from the group of patients with Pompe – it also shows that for you it is important to be able to function in a family, a job, a social environment.
Through the arrival of a treatment, this has been made possible for you and these social benefits do not come back in the advice we discuss today from the Health Insurance Board (CvZ). These social benefits are also poorly reflected in QALY calculations, because sick workers in this abstract sketch of reality will be replaced after some time by healthy workers.
Also words of appreciation for the physician / researchers Carla Hollak (Fabry) and Ans van der Ploeg (Pompe) for their evaluation studies. Unlike the previous T = 4 files, we discussed here earlier this year, the CFH (the pharmaceutical commission of CvZ) is content with the way you have conducted this evaluation. And that’s an important difference with the previous cases we discussed here this year.
January 8, 2003
For me, this day, this meeting of the Appraisal Committee (ACP) of CvZ is in sharp contrast with a special event on January 8, 2003 at the Academic Medical Centre (AMC) in Amsterdam, where physician-researchers from the academic hospitals in Amsterdam, Leiden and Rotterdam discussed three rare syndromes with three patients with these diseases. The diseases were:
Haemophilia – a rare disease, for which a treatment became available thirty years ago, around 1965 – 1970.
Gaucher disease, a lysosomal storage disease like Fabry and
Pompe disease.
Both Professor Carla Hollak as well as Professor Ans van de Ploeg told about their work, the launch of a new treatment for two till then untreatable diseases.
On the attendees who were there that day, the various presentations made a big impression. As mentioned in the letter of the VSN (The Dutch Society for patients with neuromuscular disorders) that we received for today’s discussions, the movie of a boy with Pompe disease who could walk again after two years of treatment with the new medication, made a great impression. Even more impressive, was the story about his brother who was denied access to the trial and ended in the wheelchair of his brother. This all happened within one family. I use the slide from this presentation, still in my lectures on the success and problems of the rare disease EU policy making process.
Later that year, some people who were there then, informed cautious how the situation was of that lady in the wheelchair. Also she did not qualify for the first Pompe trials and would probably have not long to live. Fortunately, that changed and Maryze Schoneveld van der Linde is nowadays – in relatively good health – speaking on behalf of the interests of people with Pompe disease everywhere in the world.
In summary, the possibility that you can treat a rare disease or not, makes a world of difference. I have experienced this myself with the arrival of a treatment for hemophilia. A treatment which, according to CvZ standards is also not cost-effective.
The pain in the CvZ advice
is for CvZ clearly the high cost of the medication for Pompe and Fabry.
CvZ could have known this – as many of you noted – also at t = 0, the period where the evaluation studies started. What data of additional research comes available in the coming years, it will not become much cheaper.
Personally, I believe that treatment with these drugs should remain possible. This opinion is based on a number of criteria that CvZ developed for entrance to the basic insurance package, namely:
The treatment is necessary, there is no alternative treatment option, it cannot be paid by someone from its own money and the treatment is given by the physicians from the expert centers slowly and carefully along the lines of the current scientific state of the art. The CFH reports also note that there is a therapeutic value, especially with Fabry disease and partially in Pompe disease, the juvenile variant.
It becomes clear from the letters we received from the stakeholders, the societal organizations that they support my view.
When ongoing treatment of Fabry and Pompe will be stopped because of the poor outcome on cost-effectiveness (first CvZ opinion of 12 June 2012), we certainly will have the costs of a patient population that is slowly but surely medically deteriorating, that is forced to stop working (labor for the coming year is especially desperately needed in the care sector), that will need more medical and social support, that will do a greater appeal on social welfare resources and the like with a slow but awful death at the end of this process.
And of course, it makes a big difference when someone will die because there is no cure for that disease, than to die when you know that there is a treatment for that disease.
CvZ did not calculate these costs in their advice and CvZ also not calculated the enormous destruction of capital of the decades of research on both diseases, that was spent in the respective academic centers in the Netherlands. Moreover, today’s advice is also a very poor signal to doctors/researchers, spin-off companies from university hospitals, investors and companies to go in with research in rare diseases in particular and research on chronic diseases in general.
Can the problem of poor cost-effectiveness of orphan drugs be solved?
This is highly unlikely in the short term. It can be if CvZ supports the proposals made by various parties in this discussion to go for larger, European studies. Both Prof. Hollak and Prof. Van der Ploeg pointed on this in their presentation and Yann Le Cam from Eurordis indicated in which direction the expert committee of the European Commission and EMA is looking for solutions.
Also the Dutch National Plan for Rare Diseases (www.npzz.nl) that should be ready by the end of this year, contains enough constructive ideas for CvZ to go on with the development and the reimbursement of orphan drugs. For example, by the appointment of centers of excellence for the treatment of rare diseases, such as is already in place for Pompe and Fabry in the Netherlands. The Dutch Ministry of Health (VWS) could support this policy even further when the treatment of rare diseases and especially those with relatively high cost is brought under the WBMV, the Law on Exceptional Medical Services. CvZ has also brought forward some solutions within the framework of Conditional Reimbursement Advice of the Apollo Network.
Can we afford the more expensive orphan drugs?
I think so. It will therefore be necessary for all parties working together in the health care sector to remove all perverse incentives in the current health care system, so that parties will no longer be reimbursed for unnecessary care.
As the chairman of the KNMG (The Royal Dutch Medical Association) said recently in the discussion about the inability to pay for these expensive treatments, this issue can only be addressed once when all ‘air’ is removed out of the system.
Earlier, we discussed in this appraisal committee the issue of ‘Appropriate Use’. CvZ therefore already signed an agreement with a large number of organizations, among them the KNMG and NPCF (the Dutch Patient and Consumer Federation). Also the report of our former Minister of Health Ab Klink contains a considerable number of examples that can further contribute to the discussion on ‘Appropriate Use’.
At the time, the plans for the ‘t = 4 reviews’ were made around 2006, there were also some considerable changes introduced in the Dutch health care system. The idea of a private market was introduced and also ideas to get away from the system of risk equalization.
The implication of these changes is that meanwhile every Dutchman pays 11 percent of its annual health insurance premium – the premium of say the month of January and the first weeks of February – to maintain the necessary monetary reserves of the health care insurance companies (EU based law Solvency II). This 11 percent is twice as high as in the rest of Europe, where less high reserves are needed because there is no liberalization, privatization of the health care market took place. Alone from the interest of this money – it is about 4 billion Euro – our budget for orphan drugs can be paid.
Can it be cheaper in the long run?
That should be – at least – the objective. It means in my opinion above all that the current knowledge and infrastructure for rare disease research should be maintained and that the Dutch government in cooperation with the whole of Europe, should continue to work on alternative and cheaper treatment methods for rare diseases in the future.
Physician/researchers and patient organizations are already working very hard on this issue. In particular, patient organizations have raised a lot of money for this goal. With the money from the French Téléthon a gene therapy facility was built near Paris. Also patient organizations finance patient registries and biobanks to facilitate research and data on the natural history of rare diseases and to study the effect of new treatments that are coming on the market.
In this context, the Dutch Alpe d’HuZes Foundation collects now annually 30 million Euro’s for research, also for research on rare cancers.
A cheaper product for the treatment of Pompe’s disease of Dutch origin, could have been already there when the research of the Dutch Leiden-based biotech company Pharming with transgenic rabbits could have go on. This technology has been hit or hurt by a political ‘no’ because of the genetic modification process and the objections against this way of working. It’s an ironic example of excellent innovative Dutch research and a lack of political courage to turn the Netherlands into a leading innovative health care country.
The Dutch Ministry of Health (VWS)
Minister Schippers of VWS – in her response to CvZ’s Advisory Package 2012 – already made a number of suggestions to CvZ to start with the operationalization of the concept of cost-effectiveness in the second half of 2012. This also applies to the theme ‘Strict Package Management’ and a follow-up to the report ‘Package management in Practice, part 2’.
It seems to me now, that the CvZ advice we’re discussing today will be put in the refrigerator for a while and that a further analysis will be made around the treshhold for the reimbursement of medical treatments. In the past, this treshhold was set on a QALY value of 80,000 Euro (RVZ-reports of 2006, that made an exception for some expensive orphan drugs). The elements brought up in the discussion of the past 45 days can then all be included. Last year, we examined the treshhold of diseases with a low burden of disease burden and it now seems a good time to do this for diseases with a high burden of disease.
In the follow-up of this process, politicians should decide on the limits that the Dutch society wants to establish for their health care.
Cees Smit, Diemen / Hoofddorp, September 21, 2012
First of all, words of appreciation for the patients of the Fabry Association for your personal contributions and especially your letters that shows quite clearly the impact of the disease on patients and their families.
At the same time, it also illustrates quite nicely the gap between what QALYs and ICER’s means for health economists and policy makers, but that you as patients totally not recognize yourselves in the words and numbers they connect with your illness.
It means once and again that the contribution of personal and collective experiences, the expertise of patients and their families gives a strong added value to concepts such as burden of disease, quality of life, research outcome measures, etcetera. One cannot exist without the other.
At the same time – and that also applies to the input from the group of patients with Pompe – it also shows that for you it is important to be able to function in a family, a job, a social environment.
Through the arrival of a treatment, this has been made possible for you and these social benefits do not come back in the advice we discuss today from the Health Insurance Board (CvZ). These social benefits are also poorly reflected in QALY calculations, because sick workers in this abstract sketch of reality will be replaced after some time by healthy workers.
Also words of appreciation for the physician / researchers Carla Hollak (Fabry) and Ans van der Ploeg (Pompe) for their evaluation studies. Unlike the previous T = 4 files, we discussed here earlier this year, the CFH (the pharmaceutical commission of CvZ) is content with the way you have conducted this evaluation. And that’s an important difference with the previous cases we discussed here this year.
January 8, 2003
For me, this day, this meeting of the Appraisal Committee (ACP) of CvZ is in sharp contrast with a special event on January 8, 2003 at the Academic Medical Centre (AMC) in Amsterdam, where physician-researchers from the academic hospitals in Amsterdam, Leiden and Rotterdam discussed three rare syndromes with three patients with these diseases. The diseases were:
Haemophilia – a rare disease, for which a treatment became available thirty years ago, around 1965 – 1970.
Gaucher disease, a lysosomal storage disease like Fabry and
Pompe disease.
Both Professor Carla Hollak as well as Professor Ans van de Ploeg told about their work, the launch of a new treatment for two till then untreatable diseases.
On the attendees who were there that day, the various presentations made a big impression. As mentioned in the letter of the VSN (The Dutch Society for patients with neuromuscular disorders) that we received for today’s discussions, the movie of a boy with Pompe disease who could walk again after two years of treatment with the new medication, made a great impression. Even more impressive, was the story about his brother who was denied access to the trial and ended in the wheelchair of his brother. This all happened within one family. I use the slide from this presentation, still in my lectures on the success and problems of the rare disease EU policy making process.
Later that year, some people who were there then, informed cautious how the situation was of that lady in the wheelchair. Also she did not qualify for the first Pompe trials and would probably have not long to live. Fortunately, that changed and Maryze Schoneveld van der Linde is nowadays – in relatively good health – speaking on behalf of the interests of people with Pompe disease everywhere in the world.
In summary, the possibility that you can treat a rare disease or not, makes a world of difference. I have experienced this myself with the arrival of a treatment for hemophilia. A treatment which, according to CvZ standards is also not cost-effective.
The pain in the CvZ advice
is for CvZ clearly the high cost of the medication for Pompe and Fabry.
CvZ could have known this – as many of you noted – also at t = 0, the period where the evaluation studies started. What data of additional research comes available in the coming years, it will not become much cheaper.
Personally, I believe that treatment with these drugs should remain possible. This opinion is based on a number of criteria that CvZ developed for entrance to the basic insurance package, namely:
The treatment is necessary, there is no alternative treatment option, it cannot be paid by someone from its own money and the treatment is given by the physicians from the expert centers slowly and carefully along the lines of the current scientific state of the art. The CFH reports also note that there is a therapeutic value, especially with Fabry disease and partially in Pompe disease, the juvenile variant.
It becomes clear from the letters we received from the stakeholders, the societal organizations that they support my view.
When ongoing treatment of Fabry and Pompe will be stopped because of the poor outcome on cost-effectiveness (first CvZ opinion of 12 June 2012), we certainly will have the costs of a patient population that is slowly but surely medically deteriorating, that is forced to stop working (labor for the coming year is especially desperately needed in the care sector), that will need more medical and social support, that will do a greater appeal on social welfare resources and the like with a slow but awful death at the end of this process.
And of course, it makes a big difference when someone will die because there is no cure for that disease, than to die when you know that there is a treatment for that disease.
CvZ did not calculate these costs in their advice and CvZ also not calculated the enormous destruction of capital of the decades of research on both diseases, that was spent in the respective academic centers in the Netherlands. Moreover, today’s advice is also a very poor signal to doctors/researchers, spin-off companies from university hospitals, investors and companies to go in with research in rare diseases in particular and research on chronic diseases in general.
Can the problem of poor cost-effectiveness of orphan drugs be solved?
This is highly unlikely in the short term. It can be if CvZ supports the proposals made by various parties in this discussion to go for larger, European studies. Both Prof. Hollak and Prof. Van der Ploeg pointed on this in their presentation and Yann Le Cam from Eurordis indicated in which direction the expert committee of the European Commission and EMA is looking for solutions.
Also the Dutch National Plan for Rare Diseases (www.npzz.nl) that should be ready by the end of this year, contains enough constructive ideas for CvZ to go on with the development and the reimbursement of orphan drugs. For example, by the appointment of centers of excellence for the treatment of rare diseases, such as is already in place for Pompe and Fabry in the Netherlands. The Dutch Ministry of Health (VWS) could support this policy even further when the treatment of rare diseases and especially those with relatively high cost is brought under the WBMV, the Law on Exceptional Medical Services. CvZ has also brought forward some solutions within the framework of Conditional Reimbursement Advice of the Apollo Network.
Can we afford the more expensive orphan drugs?
I think so. It will therefore be necessary for all parties working together in the health care sector to remove all perverse incentives in the current health care system, so that parties will no longer be reimbursed for unnecessary care.
As the chairman of the KNMG (The Royal Dutch Medical Association) said recently in the discussion about the inability to pay for these expensive treatments, this issue can only be addressed once when all ‘air’ is removed out of the system.
Earlier, we discussed in this appraisal committee the issue of ‘Appropriate Use’. CvZ therefore already signed an agreement with a large number of organizations, among them the KNMG and NPCF (the Dutch Patient and Consumer Federation). Also the report of our former Minister of Health Ab Klink contains a considerable number of examples that can further contribute to the discussion on ‘Appropriate Use’.
At the time, the plans for the ‘t = 4 reviews’ were made around 2006, there were also some considerable changes introduced in the Dutch health care system. The idea of a private market was introduced and also ideas to get away from the system of risk equalization.
The implication of these changes is that meanwhile every Dutchman pays 11 percent of its annual health insurance premium – the premium of say the month of January and the first weeks of February – to maintain the necessary monetary reserves of the health care insurance companies (EU based law Solvency II). This 11 percent is twice as high as in the rest of Europe, where less high reserves are needed because there is no liberalization, privatization of the health care market took place. Alone from the interest of this money – it is about 4 billion Euro – our budget for orphan drugs can be paid.
Can it be cheaper in the long run?
That should be – at least – the objective. It means in my opinion above all that the current knowledge and infrastructure for rare disease research should be maintained and that the Dutch government in cooperation with the whole of Europe, should continue to work on alternative and cheaper treatment methods for rare diseases in the future.
Physician/researchers and patient organizations are already working very hard on this issue. In particular, patient organizations have raised a lot of money for this goal. With the money from the French Téléthon a gene therapy facility was built near Paris. Also patient organizations finance patient registries and biobanks to facilitate research and data on the natural history of rare diseases and to study the effect of new treatments that are coming on the market.
In this context, the Dutch Alpe d’HuZes Foundation collects now annually 30 million Euro’s for research, also for research on rare cancers.
A cheaper product for the treatment of Pompe’s disease of Dutch origin, could have been already there when the research of the Dutch Leiden-based biotech company Pharming with transgenic rabbits could have go on. This technology has been hit or hurt by a political ‘no’ because of the genetic modification process and the objections against this way of working. It’s an ironic example of excellent innovative Dutch research and a lack of political courage to turn the Netherlands into a leading innovative health care country.
The Dutch Ministry of Health (VWS)
Minister Schippers of VWS – in her response to CvZ’s Advisory Package 2012 – already made a number of suggestions to CvZ to start with the operationalization of the concept of cost-effectiveness in the second half of 2012. This also applies to the theme ‘Strict Package Management’ and a follow-up to the report ‘Package management in Practice, part 2’.
It seems to me now, that the CvZ advice we’re discussing today will be put in the refrigerator for a while and that a further analysis will be made around the treshhold for the reimbursement of medical treatments. In the past, this treshhold was set on a QALY value of 80,000 Euro (RVZ-reports of 2006, that made an exception for some expensive orphan drugs). The elements brought up in the discussion of the past 45 days can then all be included. Last year, we examined the treshhold of diseases with a low burden of disease burden and it now seems a good time to do this for diseases with a high burden of disease.
In the follow-up of this process, politicians should decide on the limits that the Dutch society wants to establish for their health care.
Cees Smit, Diemen / Hoofddorp, September 21, 2012
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