Proposition 52K0505

Proposition de résolution relative à la mise en oeuvre d'un plan d'action en ce qui concerne les affections rares et les médicaments orphelins.

General information ¶

Authors

Open Vld Yolande Avontroodt, Herman De Croo, Katia della Faille de Leverghem

Submission date

Dec. 5, 2007

Official page

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Status

Adopted

Requirement

Simple

Subjects

EC Regulation medicinal product health policy resolution of parliament illness

Voting ¶

Voted to adopt

Groen CD&V Vooruit Ecolo LE PS | SP Open Vld N-VA LDD MR FN VB

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Discussion ¶

Feb. 19, 2009 | Plenary session (Chamber of representatives)

Full source

Mr Goutry, the rapporteur, is not present. I suppose he refers to his written report.

It is customary for the rapporteur to be present or to wait until he is present.

If you ask that, you will be served on your hints, because here comes the rapporteur. Do you want to explain your report? Of course you can: I assumed that you referred to your written report, but I was a little too quick.

Mr. Speaker, Excellence, colleagues, given the importance of the resolution and its content, we find it more than necessary to be able to present a good report here, followed by a good debate. I will first report, with your approval, Mr. Speaker, on the work of the committee, after which I will briefly present the position of the group.

During its meeting on 3 February, the Committee on Public Health discussed and unanimously adopted a resolution on rare diseases and rare medicines. The basic text came from colleague Avontroodt and colleague della Faille and was eventually thoroughly discussed, but also jointly amended by both the majority and the opposition. Amendments were inspired, among other things, by information from the experts heard during the hearing on 13 January. I will cover it briefly.

First was Mr. Lhoir of the Federal Medicines Agency who explained the European Regulation of 2000 which determines when a medicinal product gets the status of “weather medicine”, i.e. a medicinal product that cures rare diseases. These are medicines that look at the severity and chronic nature of the disease. There must be a link of medical credibility between the molecules, the drug and the disease. The prevalence in the European Union should not exceed 5 out of 10,000. Finally, there is the economic criterion: the firms must be able to demonstrate that the development costs are so large that given the small market, one cannot actually make it without the status of recognition as an orphan medicine.

Because the European Regulation is intended to ensure that patients with a rare disease can receive equally high-quality, safe and effective medication, support measures are sometimes needed. Hence the status of orphan medicine.

Mr Lhoir also emphasizes that in Belgium the Federal Agency plays a prominent role in the early stages of development of especially oncological drugs.

Professor Cassiman, in his capacity as chairman of the Rare Diseases and Weather Medicines Fund of the King Boudewine Foundation, advocated for a formal recognition of the Fund and for the formulation of a national plan for orphan medicines that should be implemented before 2011, a plan such as already exists in France, Spain, Germany and other European neighboring countries.

Currently, the people of the King Buddha Wine Foundation under the leadership of Professor Cassiman do so on a voluntary basis. The hearing indicated that the creation of a recognised fund should be ensured.

The pharmaceutical industry was also discussed. She called for support for a national action plan for orphan medicines.

Then the word was given to the patient associations LUSS and Radior. They witnessed the great problems of people suffering from a rare condition. These disorders are usually of genetic origin, usually very serious and incurable, and have the disadvantage that they occur so rarely that they are sometimes established too late. The treatment is very precarious precisely because there has been less scientific research on it due to the rarity of the disease or because there are fewer treatments.

The floor was then given to an employee of RIZIV who gave explanations on the procedure for reimbursement. It is the Minister of Public Health who decides on the basis of an opinion of the Committee on Medicines. There is also a college of doctors who, at the request of the CTG, provides advice to the medical advisors of the health funds, as most of these medicines fall under Chapter 4, medicines that require prior approval of the counselor.

After the hearing, the cabinet employee of the Minister on behalf of the Minister informed that the patient associations will continue to be involved in the further treatment and discussion of the project. This will also be done within the framework of the Chronic Disease Plan.

During the subsequent debate, everyone emphasized the importance and necessity of an action plan on rare diseases and orphan medicines.

I will briefly outline the various interventions.

Mrs Avontroodt, initiator of the proposal, stressed that it is not sufficient to include bear disease in the existing plan for chronically ill people. On the other hand, in line with what Europe demands, a specific national plan should be established with a 2011 deadline.

Ms. Burgeon emphasized the importance of the Special Solidarity Fund, which can initially take charge when the medicinal product has not yet been recognised for refund.

I also made a statement during the discussion. I have pointed out that in our country – fortunately – some support measures have already been taken for the orphan medicines. I have mentioned, among other things, the exemption from taxation. While other medicinal products are subject to a tax on their turnover, the orphan medicinal products are exempt from the above tax. I also urged that within the medicines budget, which currently amounts to EUR 3.8 billion, a separate budget for orphan medicines may need to be provided.

During the discussion, the CD&V group also agreed to cooperate on a national plan. We would like such a plan. We also saw interfaces with the discussion later in the committee of the minister’s plan on chronic patients. We believe that at the aforementioned occasion we should again talk about the orphan medicines.

Ms. Salvi spoke about the training of doctors and the importance of doctors’ knowledge in the field of rare diseases and treatments. She found that doctors often intervene too late and that the diagnosis is made too late. Because the diseases are so rare, doctors find it difficult to quickly make a proper diagnosis. If the diagnosis is made late, the treatment can, of course, be initiated late.

The Chairman of the Commission, Ms. Gerkens, raised the question about the responsibility of the pharmaceutical industry in this regard. She also called for at least work on a solidarity scheme between the different European Member States.

The same question was also asked by Mrs. Burgeon. She also asked for reasonability in pricing. Ms. Burgeon pointed out that the previous may be a tear point. We must take care that companies that have the exclusive right to develop only certain, very rare medicines, demonstrate some rationality in pricing and that they do not fall into a kind of monopoly behavior in this regard.

The available part of the resolution actually consists in asking for a policy to be developed together with the patient organisations that recognizes the Belgian Stuurgroup Weesgeneesmiddelen, the so-called group-Cassiman which is now situated within the King Boudewijn Foundation. The resolution also states that a national action plan should be drawn up following the foreign example. Furthermore, the resolution calls for priorities to be set for rare diseases that require new treatment. There must be information development, including at the European level. Finally, there should be a specific exchange network between specialised national and international reference centres.

So far, my report. However, I had been granted permission from Mr. President to hold my own speech thereafter.

I address the applicant, Mrs. Avontroodt, as well as the colleagues who have witnessed the debate and the chairman of the committee to say that this was an example of a good debate. From the concerns present in the committee on public health, we have come to a good resolution that was unanimously adopted. We will of course continue to join in this. Our group is also willing to ensure, through our people in the government, that effective work is done on the various points outlined in the resolution.

We should not fall here into technical aspects, the speech table serves to capture the general political lines. The technical work has already been done in the committee. However, we would like to advocate a number of things. In any case, we would like to support a national plan. It is necessary to draw up a prevalence plan, a need plan, and a supply plan, so that we can link these things together. We can then look at what serious and rare diseases we are talking about. There is statistical and scientific material. We must be able to connect this together. From that national plan, we can then appeal to the European level to reach a streamlined policy. For example, in terms of pricing, orphan medicines are the only medicines that have more or less the same price in Europe. It would be too good to be true if it were so for all medicines. We have been advocating a much more European approach to drug policy and pricing for years. Well, for rare drugs, that’s because, of course, they usually have a unit of production. They are produced by one particular company, they have undergone a general abbreviated registration procedure and they have a general recognition.

Therefore, our measures regarding the pricing of orphan medicines in our country also have an impact on the pricing at the European level.

This brings me to the first point. We note, in particular, that something strange happened with the latest measure that advocated a linear price drop. I will return to that later. We support the National Plan and we will also try to support it.

Second, we naturally also advocate that the work of, among others, Professor Cassiman and his successors, be embedded. That is a reference group that does not survive on a voluntary basis or just because it is taken over by the King Boudewijnstiftung. This group must be recognized within the National Plan. These two must be linked together so that we have a reference group, which, by definition, is an eminent level. The researchers we have in our country – I can say that for a moment – play a very important role on the European and international level. We have top-level scientists. It is precisely the talents and scientific knowledge of those people that we should be able to hold structurally with us; that we must record by recognition of such a committee. This should be linked to the national plan.

Thirdly, colleagues, we advocate a shared responsibility, including in terms of allocation and proper use of a budget. We need to consider whether a separate budget is needed, with the necessary responsibility. We expect the same responsibility from the industry. We expect the industry, in the broad sense of the word, to pay sufficient attention to that small group of orphan medicines, but we also expect that there will be transparency in pricing so that we can reach a good and transparent pricing policy for those medicines too. This means that we can incorporate this innovative material into our compulsory insurance. Very often these medicines are now in the sphere of so-called compassionate use, as if they are made available out of compassion because there is no refund scheme, or they are in the system of the BSF, the Special Solidarity Fund. Or otherwise – as happened with Herceptin – we shift them under article 56 of the RIZIV law, which is actually not correct because there are administrative costs. There they can be temporarily parked, in the experimental phase, but one would also need to find the right place in the budget for those orphan medicines so that one can come to a good arrangement in that regard.

Mrs. Minister, I may have another last point, namely the fact that orphan medicines are exempt from tax. In the past, we have drawn up a legal provision which exempts orphan medicines, as well as the category of Cx medicines and the category of stable blood derivatives – I refer to Article 191 – from sales taxes.

Well, the minister has issued a linear, modulated price drop of 1.95%. We approved this in the program law of the end of last year. In fact, this measure had a double meaning. This is calculated from the 2007 sales. This is stipulated in Article 191. One must specify this because this is the reference base for the tax. Then we explicitly provided for an exemption for orphan medicines. In the case of the price drop, the turnover of 2007 was taken as the same basis. However, medicines are not included here.

Weed medicines are therefore not exempt from the linear price drop of 1.95% which we actually regret. In the same logic, one can ask why the orphan medicines would be exempted from the tax. Precisely because they have it harder, because they are a less interesting product and a much more rare product. In fact, we had hoped that they would also be exempt from the price decrease. However, this does not appear to be so. I examined it for a moment. The law does not provide for this because the law refers in Article 191 to the turnover of 2007, but does not include the price drop. Therefore, there is no exception for orphan medicines.

In other words, even for the orphan medicines, the price should drop by 1.95%. This, of course, has a repercussion on European prices. If we want to do something for the orphan medicines, I think we should abolish that price drop for the orphan medicines and we should make for them an exception category. Therefore, I would like to submit this proposal to the Minister at the end of my presentation. I would like to hear her response.

Mrs. Speaker, Mrs. Minister, colleagues, I stand here with some humility and great gratitude to all members of the committee, to the minister and to the chairman of the committee, who eventually made this debate possible.

Colleague Goutry, you have made a very comprehensive and serene report. Of course, I am very grateful to you for this, also for the latest deepening and the technique. You did not go to be technical and at the end of the day it is also not a technical debate, because orphan patients do not allow themselves to be housed in boxes and do not allow themselves to be reduced to the technical aspect, on the contrary. It is the great merit of all members of our committee to eventually put especially the orphan patients on the agenda of the plenary session of the Chamber. I know, Mrs. Minister, that the patients and patient associations are very grateful and grateful to you and us for this.

I will not repeat what Mr Goutry has already cited in the full report, such as the definition, the technique and the European regulation, which is now finally transformed into a hopefully true Belgian plan for orphan diseases, with which we follow other European countries, such as France, the Netherlands, Spain, Bulgaria and the Czech Republic. For this purpose, France has offered an auction of 100 million euros. Of course, it is another country. Every beginning is difficult, but I am confident that a substantial part of the Minister’s policy will go there.

Mrs. Minister, I just returned from one of the meetings in the framework of the Czech Presidency of the European Union, where both chronic diseases and the Health Technology Assessment are considered very important. One of the statements there – a clue for us, as members of the committee – is that one should actually do the Health Technology Assessment of everything that isn’t on the agenda and of everything that doesn’t happen. I think this item and this policy domain is exactly one policy domain for which there has not yet been a Health Technology Assessment. One could conclude from this that it is indeed the highest time for it to happen.

Of course, the various elements in the report have been discussed, but I would like to give an example. When I met some people in the press in the corridors and said that the proposal on bees diseases was on the agenda, unfortunately no one knew what it meant. This clearly shows that it is a forgotten group. The term orphan diseases, les médicaments et les maladies orphelines, bear diseases and orphan medicines, is the correct term chosen. We should not be proud of this, on the contrary.

One of the major elements of the resolution is a better awareness, not only to the general public, but also to the healthcare providers and the entire sector of health policy, because there are still insufficient opportunities.

Mr. Goutry has already mentioned it. I would like to express a word of gratitude to the Board of Directors and the Fund, which was indeed established with the support of the King Boudewijn Foundation, who carried out the preparatory work in all serenity and with the participation of all partners, both scientists and patients. It is a beautiful example of actual patient participation, a bit based on what is happening in France and the Netherlands and where there is a full equivalence in the drafting of the policy.

As always, it is a little sad that our country is lagging behind in terms of data, Mrs. Minister. I have to confirm this in a lot of international literature. We have data from many countries in Europe, but the figures and data from our country are very often missing or are not included in the broader, epidemiological studies. This is, of course, one of the pillars of the resolution. We must at least be able to map out which and how many orphan diseases are already known in our country.

There is another point, which is also very important in other areas: the network of reference centers.

It is a school example of how every country in Europe could contribute to the correct diagnosis and treatment of patients suffering from rare diseases. Our country is fortunately very rich in networks and reference centers, but unfortunately they still work too much side by side. Per ⁇ Professor Van Broeckhoven can testify to this.

An amendment by Ms. Salvi attempted to include the centers for medical heredity in the resolution, but more is of course needed than that. There is a need for effective support for researchers and a need for better coordination in terms of expertise. I would rather talk about reference centers than about expertise centers. The reference centers can then guarantee the quality.

We ask you to make choices. It will of course depend on the data and the capabilities of the networks and the available expertise to make effective choices and to make a long-term plan.

Mrs. Minister, I would like to express a few more concerns. The first concern is that some patient associations of orphan patients have questions when accommodating under the dome of the chronic diseases, because some rare conditions are obviously not chronic. I believe that this sensitivity must be taken into account and that there must be an effective place for the orphan sick in themselves.

Collega Goutry talked about a number of modalities regarding the refund of orphan medicines. Mrs. Minister, I think that you have already anticipated this in the committee and that the discussion on this subject can be traced together with the topics in the steering group, to see what is the most appropriate track for this. In this context, I would like to repeat my request for the recognition of patient associations and to reflect on the role of Europe.

In the committee I have already asked you a question about this to include the topic during the Belgian European Presidency, to which you have indeed replied that this is happening collegially with the other countries. You added that you would take it with you. I ask you here, from the speech desk, again.

The opening that is made to recognize the importance of the theme of the orphan and the orphan medicines is, in my opinion, a step forward towards personalized medicine with individualized diagnoses and individualized therapies, which of course carries other consequences, to which also ethical aspects are linked.

Mrs. Minister, colleagues, I end by thanking you all with a quote from Edith Stein, who put it as follows. “Only those who live in the depths have an eye for the little things in the big whole.”

I think that you, Mrs. Minister, the committee and Parliament have effectively demonstrated in these matters that you also have an eye for the small things in the large whole.

On behalf of the patients, I would like to thank you all.

Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker, Mr. Speaker. If it was adopted unanimously in the Public Health Committee, it is that it helps to address the important challenges posed by rare diseases. The main challenge is ⁇ drug treatments. Despite the volunteer action carried out largely at European level, few “orphan” medicines are currently on the market. Thus, 80% of rare diseases are not treated due to lack of adequate medicines and the price of these medicines remains extremely high overall.

While this challenge of accessibility of treatments does not concern our country alone, it comes up sharply with us due to our modest size. Compared to Germany or France, our population does not represent a very attractive market for the pharmaceutical industry. Based on this finding, it is primary to take the right arrangements to ensure that all our patients have an accurate diagnosis, effective treatment, quickly available and at a reasonable price. In this, I allow myself to call on the firms to collaborate with the government to establish, as provided for in point 3 of the unanimously voted proposal for a resolution, a system of accountability for these firms.

Regarding the central theme, namely patients who, because of the rarity of their illness, are poorly listened, poorly surrounded and sometimes even untreated, fortunately, there are formidable associations of patients who will in the future be even more associated with processes of reflection and decision making. The Minister has already included them in the pilot group of the program "Priority to chronic patients"; they will be even more so tomorrow.

In addition, a large number of actors who have been working together on this subject since 2006 will be officially recognized and benefit from a grant.

Let us assume that this will allow for a positive evolution of knowledge and will ultimately promote understanding between the various stakeholders, for the benefit of patients.

Effective action, of course, involves awareness raising and training of healthcare providers. Today, many of these diseases are not known or are very bad. This misunderstanding endangers the lives of patients and is a source of frustration and unnecessary suffering. Therefore, it is necessary to work especially hard to solve this aspect of the problem.

Knowledge goes through research and exchange of information at the Belgian level, of course, but also at the European level. We need to encourage these steps and ensure that our country finally has complete epidemiological data. The text as amended and voted in the committee will help address the main challenges I have just outlined. Knowing the Minister’s commitment in this matter, I can only ask her to ensure that one day, in our country, the majority of rare diseases for which there is treatment are properly treated.

On behalf of the SP-A group, I can announce that we agree to this resolution. We fully support a national plan for rare diseases and orphan medicines.

For the sp.a, and that should be for everyone, it is important that every individual, every person in our society receives the same quality of health care, regardless of the disease that this patient suffers from.

It is known that there is more interest from the industry and, honestly I must say, also from the scientists, because it is often easier and more interesting for scientists to do scientific research for very large groups of patients, because then the chances of success are much greater. For the industry, of course, a larger market is more interesting when one is going to develop a drug or other treatment, because in that situation the revenues from the sale of medicines can of course exceed the investment costs. In other words, the industry makes profits.

This is not the case for rare diseases. For rare diseases – and that is no criticism, though, yet a little – there is less interest in the researchers and also much less interest in the industry.

I have noticed in many people, including in my group, that one does not actually know what a rare disease is, let alone that one knows what an orphan medicine is. We use definitions. These definitions come from the European Commission. From the moment a disease occurs in five people per 10,000, one speaks of a rare disease. If we look at the population in the 27 Member States, that is about 246,000 patients.

In Europe as a whole, this seems to be a lot, but then you should know that most rare diseases occur only at 1 in 100,000. This means that there are almost no patients for these diseases in Belgium. So it is difficult for Belgian researchers to do those studies, to try to find the causes of those diseases. In addition, there are between 5,000 and 8,000 different rare diseases, from different organs and that affect the quality of life of the patient in different ways, both in young people and in adults. In total, it is estimated to be around 6% to 8% of the European population or 27 to 36 million.

In principle, we are talking about a very large group of patients. The major problem is the heterogeneity of that group, which allows this group to unite under the name of rare patients, but does not have the impact power to persuade researchers or the pharmaceutical industry to invest in these diseases. New diseases are still being described. Therefore, the cause of these diseases is not yet known, but one can describe them. Often they can be diagnosed, but the course of the disease is not known. We do not know how life-threatening the disease is. We do not know how long this patient will live. We do not know how the patient should be treated.

I note, however, that the texts and the report often emphasize rare diseases in children. You refer to the medical-genetic centers. These usually work for hereditary diseases or genetic diseases in children. Well, more than 50% of rare diseases affect adults, but not children. These patients are not seen in medical genetic centers; they are seen by neurologists, by psychiatrists and other specialists. It should be taken care not to narrow the patient into a small group of hereditary ill children.

80% of patients are hereditary, but we do not know those patients. It is very strange that we do not know these patients in our society. Why Why ? Before the patient becomes known, both parents are usually not sick. They show a mistake and pass it on to their children, but only if both parents pass on this mistake, the child is diagnosed.

An example is mucoviscidosis or tongue mucus disease.

There is, of course, a method to detect a number of rare diseases. The few women present here who have had children, but also the fathers of those children present here, know what a heelsprick is.

When they are asked what exactly it is, they answer that they take a little bit of blood and do that on a ticket. When I ask why they do this, no one knows. No one knows why the blood of a newborn baby is pricked. It is not even known that children are being tested for some rare metabolic diseases. I thought you were all so mouthy, mouthy when you stand here in front of you...

You know it, but you are one of the few here still present. The others out there should know.

It is not just a lack of available information. There are also few people who ask for information, because they often find it all too difficult.

In adult patients, other diseases are also often inherited, such as peripheral neuropathies, diseases of the musculoskeletal system, which occur in one in one hundred thousand patients. These patients also have the right to a very good health care.

Not all rare diseases are hereditary. There are also those associated with infections or allergies. Anyway, a patient with a rare disease is often chronically ill for several years from the diagnosis. Often there is even no medication, and if it exists, he must take them for life.

Currently, there is too little medical and scientific knowledge about most rare diseases. The simple reason for this is that they are too rare. One must be able to study large numbers before one gets insight into the disease and the disease process.

Currently, there are approximately 1,000 rare diseases. These are the so-called frequent rare diseases. They were invested in them because they had sufficient material to study them.

The text of the resolution presented here has also been presented in the European Parliament, which is always ahead of us.

Europe already has a research programme within the Seventh Framework Programme, which runs from 2007 to 2013. In the aforementioned programme, Europe aims to stimulate scientific research and encourage industry to invest in the development of medicines for the rare diseases in question. This happens at the European level, as the programme exceeds a country or a Member State. It is necessary to work together with the different Member States. Researchers and industry are forced to work together. Europe is doing very well in this area.

Several researchers have enrolled in the program. There have also been various cooperative partnerships. We can therefore expect that in the future we will accumulate more scientific and medical knowledge. We can also expect that the industry, which is very positive about the program, will unite and invest.

The point is that when a dossier is realized at the European level, something must also be realized at the national level. If there is a European plan, there must also be a national plan. Everything that comes from Europe must be translated to the national level and to the peculiarity of the own country, to the peculiarity of the health care and to the peculiarity of the legislation.

We want a national plan. The above-mentioned plan must be seamlessly aligned with the European plan that already works today. The European Union has proposed that every Member State should have a national plan by 2011. I hope that it will not be again as it is always in Belgium, especially that we will begin to think about such a national plan around the second half of 2011. I hope my fear does not come true.

So I make a concrete proposal. I ask the Minister to take the initiative and, in her capacity as Minister, as proof of her support for the national plan, to encourage its development. The national plan is a global plan that addresses all the different aspects of implementation – both legal, economic, social and medical aspects.

I also ask Mrs. Minister to support – now, today, maybe tomorrow but ⁇ not in a year – the establishment of a working group that brings together all actors. They should be supported by the Ministry to prepare the above-mentioned global national plan.

That working group needs to start now, to make sure that we can ⁇ something in the second half of 2011.

Mrs Van Broeckhoven, that steering group, that working group, within that fund or with the support of that fund, works already today. The Minister has also added people from the administration, so that now already those points of attention, which were expressed in the resolution, are being developed further by that working group, that steering group within that fund. The Minister has said that this working group will be substantially supported from the cabinet and from the government. I would like to say that this work group already exists.

Mrs Avontroodt, I am very pleased with that. You know that I could not be present at that hearing because of an exit ceremony in which I had to and wanted to be present. I have read the report.

I can only congratulate that this working group has already been realized. I hope that the working group will therefore have the necessary representation to develop a global national plan.

Of course, I would also like to be informed. I was not aware of the beginning of that working group, because otherwise I would not have mentioned this here. I would like to stay informed about that working group and its activities, rather than being confronted with the results at a later stage.

I think I will keep it, although there is still much to say, especially in relation to the development of the national plan. However, this discussion comes too early. This will be discussed when the national plan is in place. I hope to hear more about it in the future.

From the colleagues in the Chamber, I hope, if that national plan comes up, that they will support it across the House.

I would like to begin this speech by thanking my colleagues, and more ⁇ Mrs Avontroodt, for preparing the work and for insisting that the commission can take action.

This theme met with the concern and interest of all members of the committee and, therefore, we were able to conduct hearings and an interesting work, in collaboration with both representatives of the cabinet, the pharmaceutical sector, INAMI instances, the Reimbursement Commission and some scientists. In particular, we have been able to associate with all these work the representatives of the patients who are at the basis of all our mobilization.

I do not return to all the elements quoted by Ms. Van Broeckhoven, Avontroodt, Lambert or M. and Goutry. I share their concerns about the difficulties we have in mobilizing around those who represent a minority so complex that one feels impoverished. Per ⁇ we find it difficult to mobilize all of our energies. I agree with you on the need for a plan.

I would like to give you a few additional reflections that came to my mind during this work. It is a fact that this type of orphan medicines is not a financially profitable operation for pharmaceutical firms, since one cannot put a sufficient number of them on the market. Nevertheless, they are granted aid at both Belgian and European levels to develop, register and market the medicines they produce.

We need these pharmaceutical companies to conduct research on these rare diseases. It is true that we have a lot of difficulty in determining the difficulties encountered in order to be able to control them with them. However, does research on these orphan drugs not also participate in all the work that allows them to develop new molecules or new processes that will be used in other treatments? It seems to me that they benefit from a return!

In the committee, I wondered how to hold pharmaceutical firms accountable. Indeed, Mrs. Minister, a member of your cabinet responded to study, with the pharmaceutical firms, the possibility of helping them and encouraging them in the production of this type of medicines, while negotiating accountability mechanisms.

Currently, they restore to the community the budget planned for spending on medicines, when it is exceeded. I agree with the proposal of Mr. Goutry: You could ask them for a gesture in favor of orphan medicines, understood to be assisted for registration rights and exemptions.

I would like to point out that it is in medicine that the problem is the most acute. However, very significant intervention efforts are provided for these medicines. I have the figures: from 2004 to 2008, we are moving from a budget of 25 million euros for 5 specialties to a budget of 77 million euros for 30 specialties. In 2009, it will result in expenses of the order of 110 million euros.

For those who have not been able to follow the debate in the committee, the prices of medicines are overpriced. Some examples of the number of patients treated clearly identify the problem: for Hunter’s disease, Elaprase for 8 patients costs 300,000 euros per year per patient. Among the new drugs, I am indicated, among other things, that, to treat enzyme deficiencies, Naglasyme costs 375,700 euros per patient and per year for an average weight of 25 kg, therefore more than 1 million per year for a patient of 75 kg. You can imagine the magnitude of the problem: these figures are hallucinating.

We are already intervening in different ways. Orphan medicinal products are fully reimbursed to patients; producing companies are exempt from the tax on the turnover made on these medicinal products; firms have the possibility to introduce price and refund requests upon obtaining the positive opinion of the Committee for Medicinal Products for Human Use, without waiting for the official approval of the European Commission. This is a three-month gain that is a famous advantage.

Everything is done to improve the protection of the patient, the help that is given to him and the research on orphan drugs.

by Mr. Goutry and others gave some indications. We will examine all possible ways to support research and reduce the cost of medicines, but I am convinced that without a large European initiative, marginal results can only be achieved.

You introduce the second point that, for me, emerged from the work, namely the fact that an organization and collaboration are absolutely necessary at the European level.

There are eight patients. For other diseases, no patients were mentioned in Belgium but a few in other states. If you do not consider the number of patients who may benefit from treatment and how to treat them – in other words, if you do not evaluate the cost of manufacture and the price of the drug at European level – it will become untreatable and unpaid by each state, and ⁇ ⁇ ⁇ by Belgium, which is a small country.

It is well known that it is difficult for pharmaceutical firms to lower the price of ordinary medicines on our small market, knowing the influence that a decrease will have on the larger markets, which are obviously much more important for them. Certainly, discussions are ongoing at the European level but, in my opinion, it is on this point that we must be able to manage the collaboration with pharmaceutical firms.

The third element on which I am speaking is related to other issues, in particular that of taking into account the pain we talked about this morning at the congress organized by Mr. From Germany on the centers of pain.

It is difficult for doctors to make a diagnosis within a reasonable time. Sometimes it takes several years to ⁇ this. It is highlighted the fact that first-line physicians, but also a whole series of specialists, would not be sufficiently trained or would not have the ability to treat differently a patient who consults them and whose symptoms they do not understand. This emphasizes the fact that while these patients are in a critical state of health – because they have pathologies not only rare but often serious and incurable – the legitimacy of their complaints is not recognized.

It is therefore important to raise awareness of all health actors about this problem when a “strange” patient comes to them.

I recall what the patient representatives told us in the committee and which also appears in other issues: let us try to take into account the capacities of the patients and the mobilization of their surroundings, essentially the family. Indeed, once the diagnosis is made, one cannot merely administer a medication to the patient; one must install around him a network of care, which must integrate relatives and psychosocial support.

We therefore join those famous bio-psychosocial models requested by other patients, such as those overwhelmed by diffuse and permanent pain, which we also have difficulty recognizing and for which there is no effective drug treatment.

We also join the need to organize health care actors around these different issues. We are talking about national plans, we are also talking about reference centers that would allow for better targeting things. We will also be led to reflect on a reorganization of healthcare in relation to each other and to develop the support role of the reference centers, to reflect also on the availability of other health actors, at home or specialized in hospitals.

Even if we dedicate ourselves to rare diseases and orphan medicines, therefore to a very limited number of people, we realize that the necessary consideration of their particularity can occur through a general reflection that we have already had the opportunity to talk about other difficult pathologies.

Mr. Speaker, Mrs. Minister, dear colleagues, after these brilliant interventions to which I agree, I will endeavour to be as concise as possible. First of all, I would like to thank in particular Mrs. Avontroodt for initiating this interesting debate as well as Mr. Goutry for the excellent report he presented to us.

Without going back to the statistics, I would like to remind you that in Belgium, the number of people with orphan diseases is estimated at 65,000. The majority of these diseases are still not sufficiently known to health professionals. This situation can lead to misdiagnosis that can not only cause suffering for the sick and their families, but also dangerously push away their care.

Additionally, adequate treatment is often not available and when effective treatment appears on the market, its availability is often uncertain or even very expensive. In this debate, the pharmaceutical industry has a key role to play because it is the main source of innovation. Unfortunately, the development of orphan medicines faces the complexity and heterogeneity of diseases as well as the limited number of patients.

Furthermore, orphan medicines represent an unattractive market because they do not allow amortisation of research costs by sufficient production. In 2000, the European Union adopted the Regulation on the recognition of orphan medicinal products. It provides that the industry can benefit from European and national incentives for the development of this type of medicines. In addition, orphan medicines are recognized by an exceptional status.

A number of countries, including the Netherlands, Italy, Spain and France, have already put in place a specific policy on rare diseases and have taken measures to stimulate the development of orphan medicines.

In France, rare diseases were considered as one of the five major priorities of the Act on Public Health Policy of 9 August 2004. Translating this strong political commitment, the National Plan for Rare Diseases 2005-2008 proposes a series of concrete, coherent and structuring measures for the organization of the French health care system.

The President of the French Republic has also announced a new national plan for the coming years. In our country, there is still a long way to go for a health policy on rare diseases. Only 31 medicines are reimbursed, while we are hosting many early-phase or first-time human clinical trials. The chronic diseases plan of the Minister of Health, for which we demand a quick implementation, will have to take into account the peculiarity of orphan diseases.

Like other European countries, we need to develop a specific approach to the care of patients suffering from a rare disease. Healthcare providers and the general public should be better informed and healthcare professionals should be trained to better identify these diseases. The dynamics of the development of new orphan medicines and their access to reimbursement as quickly as possible must be clearly continued by appropriate measures. Finally, mechanisms at national and European levels must be established and supported to improve and streamline the exchange of information on these diseases.

In formulating these requests, we only demand the implementation of the Government Agreement which expressly stipulates that it is necessary to provide an answer to specific questions in the field of health such as, for example, orphan diseases but I trust the Government and Ms. Minister in particular to examine all possible pistes to promote the research and financial support of these diseases for patients.

Mr. Speaker, after the excellent intervention of my colleague, Jacques Otlet, I would also like to thank our colleague, Mrs. Avontroodt, for her very positive initiative.

While reading the resolution, I will allow myself to insist on one point that is not addressed and which, despite all, seems to me fundamental. It is true that we need to develop medicines that can treat people who have the disease, but we must also try to prevent the transmission of this disease. Since it is known that 80% of orphan diseases are of genetic order, it seems to me that it is also necessary to support genetic research in this sector, in particular everything that is pre-implantable diagnosis that allows to evolve, in a very positive way, in the care of these diseases. In addition to pharmaceutical research and drug research, we need to anticipate more, act and develop genetic research and everything that is pre-implantable diagnosis to ensure that these diseases do not pass from generation to generation.

Mr. Speaker, it was Ms. Salvi who followed this issue in a committee for our group, but she is now sick, not of a rare disease but of a simple flu.

I would like to say all the importance that we attach to these so-called rare diseases, but which affect several tens of thousands of people in Belgium. In some cases, rare diseases are diseases that are not yet sufficiently known. Some diseases now well known or beginning to be, such as fibromyalgia, were considered rare diseases in the past.

Therefore, it is worth insisting on research, on improving our knowledge in these diseases. For taking into account medicines, it is important to create a fund. It was also said the importance of improving international cooperation on these diseases: some rare diseases here are common in Asia or Africa.

It can therefore be interesting not only to benefit from existing research in these countries, in terms of knowledge of these rare diseases, their treatment and their diagnosis, but also to create an international fund related to them. It is important not to work on these issues within the limited framework of our borders.

I will further insist, like the resolution, on the fact that, since these are essentially long-term diseases, the follow-up of the health path, the fact that the patient is at the heart of taking care of the treatment and its improvement are essential.