Letter to President Macron

Part 3: Legal Analysis and Conclusion

Legal Analysis

The 2011 Directive – highly specialised treatments for rare diseases

Legally speaking, the “same or equally effective treatment” medical standard which L’Assurance Maladie applied to Lysiane’s S2 application, and which they used as the basis for refusing to authorise the TPP treatment, appears nowhere in the 2011 Directive. Rather, this standard is derived from cases which were decided in the European Court of Justice. These were cases in which EU citizens were seeking, or had received, cross-border healthcare in another EU Member State, outside of their Member State of affiliation. The cases generally involved EU citizens who had travelled to another EU Member State in order to obtain conventional medical care, for common medical conditions. One case involved an individual who had purchased eyeglasses in another Member State, and was denied reimbursement back in his home state. Another patient had travelled to another EU Member State to receive dental care. In one case, a patient had arthritis of the hip – and in another dispute, a woman was suffering from pain in her right wrist.

Occasionally, EU citizens were denied authorisation, or refused reimbursement, for experimental, medically unproven treatments; one patient wished to travel to another EU Member State in order to receive “psychosomatic pain treatment”.

In none of these European Court of Justice cases was the patient seeking a medically proven treatment for a rare disease – and certainly none of the claimants happened to be a newborn baby suffering from a rare disease, attached to a ventilator machine in a neonatal intensive care unit. There is a reason such a case has never reached the European Court of Justice. The Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare “aims to establish rules for facilitating access to safe and high-quality cross-border healthcare”. It explains that:

“…the vast majority of patients in the Union receive healthcare in their own country and prefer to do so. However, in certain circumstances patients may seek some forms of healthcare in another Member State. Examples include highly specialised care…”

Patients suffering from a rare disease are exactly the kinds of patients who typically require highly specialised cross-border care. For rare disease patients, cross-border healthcare is not a mere question of personal preferences, nor is it a means of exploring the possibilities or testing the boundaries of Regulation 883 and EU law. Rather, it represents an urgent attempt to obtain a life-changing and possibly life-saving treatment for their rare disease. Often, the highly specialised treatments they seek are only available in certain limited locations, geographically distinct knowledge centres, which serve as “centres of expertise” for their rare disease. Due to the seriousness of their rare disease, and a lack of local expertise, rare disease patients are ready to travel to those highly specialised knowledge centres, wherever they happen to be.

Based on the 2011 Directive’s recognition of the special needs of these rare disease patients, Article 13 (“Rare Diseases”) of the 2011 Directive specifically sets out “the possibilities offered by Regulation (EC) No 883/2004 for referral of patients with rare diseases to other Member States even for diagnosis and treatments which are not available in the Member State of affiliation.”

While the 2011 Directive revolves around the notion of patient choice, nowhere is this privilege – the right to choose a highly specialised treatment in another EU Member State – emphasized more strongly or more clearly than in Article 13, “Rare Diseases”.

CLEISS, the national contact point for France under the 2011 Directive, confirms the special margin of choice which the 2011 Directive grants to EU citizens suffering from a rare disease:

“Medical care and reimbursement

Patients diagnosed with or suspected of having a rare disease have the right to access health care in another Member State of the European Union or the European Economic Area (Iceland, Liechtenstein and Norway) within the framework of Regulations No 883/04 and No 987/09, even if the diagnosis and/or treatment in question is not available in the patient’s State of affiliation.”

Found here: https://www.cleiss.fr/particuliers/venir/soins/ue/maladies-rares.html

Our daughter Lysiane was born with a rare disease; she is a patient “with a medical condition requiring a particular concentration of expertise in medical domains where expertise is rare” (the 2011 Directive, Article 12). Based on this, Lysiane is legally guaranteed a broader margin for selecting appropriate specialised treatment, for her rare disease.

In the context of a rare disease, the question is not the availability in France of existing treatments; in the context of a rare disease, the issue is Lysiane’s legally guaranteed right to choose a highly specialised and medically proven treatment which is not available in France. This is the whole point of the 2011 Directive’s Article 13.

The Four Fundamental Freedoms of the EU

The laws and rights involved in this dispute go beyond the 2011 Directive; they arise out of the central purpose of the EU itself. At the core of the European project is the effort to create a single EU market. The EU’s four fundamental freedoms – the free movement of goods, services, people and capital – provide the foundation for this single market.

In joined cases 286/82 and 26/83, Luisi et Carbone, the European Court of Justice specifically confirmed that the fundamental EU freedom, the freedom to provide services, also includes the freedom to receive services:

“…the freedom to provide services includes the freedom, for the recipients of services, to go to another member state in order to receive a service there, without being obstructed by restrictions…”

In the same set of joined cases, the European Court of Justice also declared that:

“persons receiving medical treatment… are to be regarded as recipients of services.”

In addition to the European Court of Justice’s case law, the 2011 Directive itself reminds Member States that healthcare services are covered by the fundamental freedom to provide and to receive services in the EU:

“As confirmed by the Court of Justice, neither its special nature nor the way in which it is organised or financed removes healthcare from the ambit of the fundamental principle of the freedom to provide services.”

Thus, based on the fundamental freedom to provide and to receive services, which is enshrined in the Treaty on the Functioning of the European Union, Article 56 – and as confirmed repeatedly by the European Court of Justice in multiple decisions which are binding on all EU Member States – EU citizens enjoy a fundamental legal right to receive services, including medical services, in other EU Member States, without facing arbitrary, unjustified, undue obstruction.

The four fundamental freedoms hold a very special place in EU law. Peter Altmaier, a German Minister, and Angela Merkel’s top adviser in the Brexit negotiations, recently explained it the following way:

“These four fundamental freedoms are at the heart of the single market… That means that any country that would like to participate in the single market, basically has to accept the single market as it exists.”

Accepting the single market as it exists means fully respecting the four fundamental freedoms, of every EU citizen.

Because the four fundamental freedoms are so critical for achieving the single EU market, the four fundamental freedoms are treated, legally, as a kind of red line. When an EU Member State is brought before the European Court of Justice for obstructing one of the four fundamental freedoms, the Court subjects the Member State’s actions to strict legal scrutiny. The EU Member State has to explain to the European Court of Justice exactly how and why the EU Member State’s obstruction was objectively justified. The EU Member State also has to demonstrate, convincingly, that when the EU Member State obstructed one of the four fundamental freedoms, that the EU Member State did not exceed what was objectively necessary to achieve a certain, specific goal. The goal cannot be any vague, random goal, chosen at whim; the goal has to be a specific, legitimate goal. The Member State will even be required to convince the Court that the Member State could not have achieved that same, important, legitimate goal, using less restrictive measures.

In brief, when an EU Member State obstructs one of the EU’s four fundamental freedoms, the EU Member State will have a great deal of explaining to do.

Large bureaucracies tend to dislike when constraints are placed upon their power and their discretion. This principle is universal, and it holds true when dealing with the social security systems of the various EU Member States. On several occasions, EU social security systems brought before the European Court of Justice have attempted, unsuccessfully, to justify their obstructive behaviour, using the following argument: “each EU Member State is responsible for organising its own social security system; how we carry out that responsibility is our business, and our business alone.”

For EU citizens, it is very fortunate that this argument does not work. Every single EU Member State, in managing its social security system, must manage it in conformity with EU law. This means that the social security system’s decisions cannot violate EU law, and in particular, they cannot violate the EU’s four fundamental freedoms. This was articulated very clearly by the European Court of Justice in the Watts case, Case C­372/04:

“Whilst… it is for the legislation of each Member State to determine the conditions in which social security benefits are granted, when exercising that power Member States must comply with Community law, in particular the provisions on the freedom to provide services… Those provisions prohibit the Member States from introducing or maintaining unjustified restrictions on the exercise of that freedom in the healthcare sector.”

EU Member State social security systems are not free to simply do whatever they want with their power; they must exercise their power in accordance with EU law. Once again, as explained by the European Court of Justice:

“It is settled case­law that a system of prior authorisation cannot legitimise discretionary decisions taken by the national authorities which are liable to negate the effectiveness of provisions of Community law, in particular those relating to a fundamental freedom such as that at issue in the main proceedings”.

EU Member State social security systems must, at all times, comply with, answer to, and respect, EU law, especially the four fundamental freedoms.

The 2011 Directive, Article 8, reminds Member States of the inviolability of the EU’s four fundamental freedoms. Article 8 also reminds them of the legally binding nature of the Court’s decisions regarding cross-border healthcare, by using the same phrases and terms which the Court itself used when deciding these cases:

“…individual decisions of refusal to grant prior authorisation, shall be restricted to what is necessary and proportionate to the objective to be achieved, and may not constitute a means of arbitrary discrimination or an unjustified obstacle to the free movement of patients.”

In the case of our daughter Lysiane, the treatment which is used in France to treat our baby’s rare disease consists of keeping the baby in a hospital, attached to a ventilator machine. Lysiane was stuck in a French intensive care unit for five straight weeks, with no release date in sight. Since ventilation assistance generally requires long term hospitalisation, the French treatment creates colossal healthcare costs. Germany’s highly specialised TPP treatment, on the other hand, is medically proven to resolve the upper airway obstruction associated with this rare disease, and instantly liberate the baby from the ventilator machine – thereby safely and effectively eliminating the need for long term hospitalization. This makes the TPP treatment a cost-effective medical breakthrough in the treatment of this rare disease.

Based on these facts, and considering the law, what crucial objective was L’Assurance Maladie trying to achieve when it refused Lysiane’s request for the TPP treatment? The goal L’Assurance Maladie was trying to achieve has to be legitimate; therefore, L’Assurance Maladie cannot say, “our objective was to waste money; forcing Lysiane to remain connected to a ventilator machine in France, and endure expensive long term hospitalisation, was necessary and proportionate for achieving our objective, to waste money.”

Once again, the goal has to be legitimate; since the goal has to be legitimate, L’Assurance Maladie cannot say, “our objective was to use up excess space in France’s neonatal intensive care units, expose Lysiane to the unnecessary risk of hospital borne illnesses, and separate Lysiane from her parents for as long as possible. Forcing Lysiane to remain connected to a ventilator machine in the neonatal intensive care unit was necessary and proportionate for achieving our objective, to use up excess space in France’s neonatal intensive care units, expose Lysiane to the unnecessary risk of hospital borne illnesses, and separate Lysiane from her parents for as long as possible.” By putting into words the actual consequences which L’Assurance Maladie’s position entails, it becomes clear how irrational it was for L’Assurance Maladie to refuse Lysiane’s S2 application for the TPP treatment.

L’Assurance Maladie’s irrational rejection of Lysiane’s request to obtain the highly specialised, medically proven and cost-effective TPP treatment for her rare disease is a clear example of the type of unjustified obstacle to the fundamental freedom to provide and receive services, and the free movement of patients, which the Treaty on the Functioning of the European Union forbids, and which the 2011 Directive forbids, and which the European Court of Justice, in case after case dating back many years, has militated so strongly against.

Indirect discriminatory effect

In order to achieve a single EU market, state actors in the EU are prohibited from engaging in market interference. If, hypothetically speaking, the UK government, seeking to steer UK consumers to drink more domestically produced English beer, rather than imported French wine, suddenly imposed a high import tax on all wine entering the UK – this, in practice, would produce what EU law specialists call an “indirect discriminatory effect”. The tax on imported wine, even though it does not single out French producers, would unfairly prejudice French vineyards, because French wine would suddenly increase in price relative to domestically produced English beer. This type of market interference, even though it is subtle, and indirect, is illegal in the EU.

For at least 5 years now, the Pierre Robin Reference Centre at Necker Hospital in Paris has been maintaining a wall between Pierre Robin Sequence patients in France, and qualified healthcare providers in Germany. This policy does not explicitly target German providers of medical devices, or German providers of healthcare services. However, in practice, the policy has an indirect discriminatory effect upon them. The policy, and its indirect discriminatory effect, can be demonstrated by presenting the Pierre Robin Reference Centre in Paris with the following questions:

  • Over the past 5 years, a number of French babies have been diagnosed with Pierre Robin Sequence. How many times were the parents of these babies informed that the TPP treatment was a safe and medically proven treatment option for their baby’s rare disease, and that it can be obtained in multiple treatment centres just next door in Germany?
  • How many times has the Reference Centre in Paris recommended the medically proven TPP treatment for Pierre Robin Sequence?
  • How many times has the Pierre Robin Reference Centre prepared a medical certificate in support of a French patient’s S2 application to receive the TPP treatment in Germany?
  • How many French patients suffering from the Pierre Robin Sequence have received the TPP treatment in Germany?

The answers to all of these questions – zero, or close to zero, over a period of more than 5 years – will demonstrate that a pattern exists which can only be the result of a deliberate policy. The goal of this policy is to restrict French patients’ access to the TPP medical device – a good – and to restrict French patients’ access to Pierre Robin Sequence healthcare treatment in Germany – a service. This policy not only turns the spirit of European cooperation upside down, to the detriment of a particularly vulnerable population – babies suffering from a rare disease – but due to its verifiable, indirect discriminatory effect, it also violates Article 34 of the Treaty on the Functioning of the European Union.

This policy also raises broader questions which transcend EU law: the question of basic medical ethics, and the physician’s obligation to obtain informed consent, as explained in the following section.

Dr. Veronique Abadie and The Pierre Robin Reference Centre in Paris

Dr. Veronique Abadie, the director of France’s Reference Centre for the rare disease, Pierre Robin Sequence, refused to provide us with the medical certificate we needed for Lysiane’s S2 application to receive the TPP treatment in Germany. Instead of providing us with a medical certificate, Dr. Abadie wrote a letter suggesting that the same or equally effective treatment is available in France. Dr. Abadie is the director of France’s Reference Centre for the rare disease, Pierre Robin Sequence. This means that in France, her medical opinion regarding this rare disease is definitive. Therefore, one cannot be surprised that after L’Assurance Maladie received Dr. Abadie’s letter, L’Assurance Maladie rejected Lysiane’s S2 application.

In Germany, Dr. Christian F. Poets is the Chairman of The Interdisciplinary Centre for Craniofacial Malformations at Tübingen University Hospital. It was at this Centre in Tübingen University Hospital that our daughter Lysiane received the TPP treatment which resolved her upper airway obstruction, liberated her from the ventilator machine, got her out of the intensive care unit, and changed her life. Dr. Poets was in charge of Lysiane’s medical care when Lysiane received the TPP treatment in Tübingen. Dr. Poets was genuinely surprised that L’Assurance Maladie rejected Lysiane’s S2 application. He was surprised because his Centre in Tübingen has provided the TPP treatment to patients from various other EU Member States, and those patients from various other EU Member States had always received their S2 documents from their respective EU Member State governments without any problems whatsoever.

Dr. Poets in Tübingen, and Dr. Abadie in Paris, are professional colleagues; they have known one another for years. Dr. Poets and Dr. Abadie address one another on a first name basis: “Christian”, and “Veronique”. On 7 May 2017, a conference was held in Toronto Canada. The subject of the conference was the rare disease, Pierre Robin Sequence. Experts on Pierre Robin Sequence came from all over the world to attend the conference, and to discuss treatment strategies: https://robin-sequence.com/

Dr. Poets and Dr. Abadie both attended this 7 May 2017 conference in Toronto. During the conference, Dr. Poets and Dr. Abadie spoke with one another. Dr. Poets asked Dr. Abadie why she would not support Lysiane’s S2 application.

Dr. Abadie told Dr. Poets that if she did, the result would then be that other French patients suffering from Pierre Robin Sequence would also go to Germany for treatment, instead of going to her.

President Macron: as Lysiane’s father, there are many strong words I could use right now to describe this situation in general, and Dr. Abadie’s conduct in particular. However, I will refrain from doing so. What I think, and how I feel, is ultimately far less important than the full and open investigation which this situation calls for, and the actions your government takes as a result of this full and open investigation.

We as parents saw first-hand the dramatic advantages of Germany’s TPP treatment over France’s conventional treatment, ventilator machines, because our daughter Lysiane received both treatments. Thus we understand Dr. Abadie’s concern that the Reference Centre which she directs at Necker Hospital could in the future lose some French patients to Germany, since the Reference Centre at Necker Hospital does not offer the TPP treatment. However, Dr. Abadie’s wish to protect the client base of the Reference Center at Necker Hospital is not an acceptable justification for obstructing Lysiane’s S2 application.

Dr. Abadie’s conduct raises several disturbing issues, including the following. First of all, Dr. Abadie is the director of an EU recognized Orphanet Reference Centre for the rare disease, Pierre Robin Sequence. The EU’s European reference networks, “especially for rare diseases“, are supposed to “help realise the potential of European cooperation regarding highly specialised healthcare for patients and for healthcare systems by exploiting innovations in medical science and health technologies” (the 2011 Directive, Article 12). The safety and effectiveness of the highly specialised and innovative TPP treatment has been definitively proven in peer-reviewed medical studies dating back over ten years – but throughout all of France, a country of 65 million people, our daughter Lysiane may be the first French baby to ever receive this breakthrough German treatment. This is striking – but Dr. Abadie’s explanation to Dr. Poets at the 2017 Pierre Robin Sequence conference in Toronto explains why. Dr. Abadie’s intentional obstruction and non-cooperation completely defeats the purpose of the 2011 Directive’s European reference networks. If Dr. Abadie is unwilling to help realise the potential of European cooperation regarding highly specialised healthcare for rare diseases by exploiting innovations in medical science and health technologies – such as the TPP treatment – then she should not be directing a European Reference Centre.

Second of all, by obstructing French babies from obtaining the highly specialised TPP treatment in Germany, the Pierre Robin Reference Centre at Necker Hospital, under the leadership of Dr. Abadie, not only violated EU law through intentional market interference, and illegitimate obstruction of the EU’s four fundamental freedoms; the Pierre Robin Reference Centre at Necker Hospital also violated its own mission statement. The mission statement of the Pierre Robin Reference Centre at Necker Hospital includes “Facilitation of patients’ access to care” (“Facilitation de l’accès aux soins des patients”). Any definition of “care” must in any case include all highly effective and medically proven devices and techniques, even if they were not developed by Dr. Abadie and her team of physicians in Paris, but rather by her colleagues and counterparts in Germany. According to her Reference Centre’s mission statement, Dr. Abadie should be facilitating patients’ access to care. If Dr. Abadie believes it is acceptable to obstruct patients’ access to care – specifically, to a safe, highly effective and medically proven treatment for a rare disease – then once again, she should not be directing the Pierre Robin Reference Centre at Necker Hospital.

Third of all, and most important, Dr. Abadie’s wish to protect the client base of the Reference Center at Necker Hospital, as indicated by her discussion with Dr. Poets at the Pierre Robin Sequence conference in Toronto on 7 May 2017, is not an acceptable justification for obstructing Lysiane’s access to a safe and medically proven treatment for Lysiane’s rare disease. No doctor should place professional interests above the health interests of patients. Such conduct raises serious questions of basic medical ethics.

What makes this even more disturbing is that this question concerning basic medical ethics may very well be part of a broader, systematic pattern. As we have already indicated earlier in this document’s “Background Information” section, we spoke with Dr. Abadie by telephone regarding our daughter Lysiane’s health, and the TPP treatment, on Monday 10 April 2017. During this telephone call, Dr. Abadie confirmed that she knows Dr. Poets in Tübingen, and she knows his work. Regarding the TPP treatment, Dr. Abadie told us:

“I know this technique and I know Professor Poets. It’s a technique which works. It works, it’s undisputable.”

French original:
“Je connais cette technique et je connais le Professor Poets. C’est une technique qui marche – ça marche, c’est indiscutable.”

Before we ever contacted Dr. Abadie, we, Lysiane’s parents, were desperate to find the best possible medical treatment for our daughter’s rare disease. After exhaustive research, which we carried out on our own, we located several medical studies providing details of the TPP treatment. For us as parents – not doctors, but parents – the TPP treatment was extremely promising news. Learning about the TPP treatment gave us hope for our baby, who was struggling to breathe, and who suffered repeated instances of oxygen desaturation in the French hospital. For Dr. Abadie, on the other hand, the TPP treatment was not news at all; Dr. Abadie has known about the TPP treatment for years. She knows the head physician leading the German medical team which administers the TPP treatment in Tübingen, she reads their medical studies, and she knows very well that the medical effectiveness of the TPP treatment is “indisputable”.

Dr. Abadie also knows that the treatment she has been proposing for French babies in France – connecting the baby to a ventilator machine with a CPAP mask – is a treatment associated with real medical risks. A newborn baby wearing a CPAP mask is exposed to the serious and known risk of facial deformities. As explained earlier, in the Medical Analysis, Dr. Brigitte Fauroux, a colleague of Dr. Abadie at the Pierre Robin Reference Centre at Necker Hospital in Paris, studied the risks of ventilation masks, and the facial deformities caused by these masks. Dr. Fauroux’s peer reviewed medical study indicates that the serious risk of maxillary retrusion is triggered not by months and months of the baby wearing the ventilation mask, but from the baby wearing the mask for more than 10 hours per day, for any period of time – months, weeks, possibly even days.

This leads to the question: were French parents informed of the serious risk that these CPAP ventilation masks could cause their baby to suffer from facial deformities? If French parents were not clearly informed about the known risks of facial deformities, then how can those French parents possibly have provided informed consent for their baby to receive this particular treatment? For a parent’s consent to qualify as informed consent, the physicians would first need to explain to the parents all of the relevant and material facts associated with the proposed treatment, including the benefits, the risks, and the alternatives.

The French parents who gave consent for their baby to receive one of the French palatal plates which Dr. Abadie described in her letter to L’Assurance Maladie – were those French parents informed that the French palatal plate which their baby was receiving had “no effect on ventilation”, as Dr. Abadie admitted in writing? Were those French parents informed that the French palatal plates lacked peer-reviewed medical studies to demonstrate what benefits they could offer, and what risks they might create? Were those French parents informed that just next door, in Germany, this exact same type of treatment, a palatal plate, is used to treat babies suffering from Pierre Robin Sequence? Were those French parents informed that unlike the French plates, the German palatal plates do successfully resolve the potentially life-threatening breathing difficulties faced by these babies? Were they told that unlike the French plates, the German palatal plates are backed by over ten years’ worth of peer-reviewed medical studies, which prove that the German plates are not only highly effective, but also safe for babies to use?

As for the French parents who gave consent for their baby to undergo invasive surgery, including mandibular distraction osteogenesis, and even labioglossopexy – sewing the baby’s tongue to the baby’s lip, a shameful practice which civilised countries should ban – were those French parents informed of less invasive alternative treatments? Were those French parents informed that the TPP treatment in Germany might allow their baby to avoid these painful, invasive, and risky surgical procedures altogether, and yet still fully resolve their baby’s breathing difficulties, safely, and effectively?

Regarding risks: the Pierre Robin Reference Centre at Necker Hospital in Paris cannot claim that they did not know about the risks of ventilation masks and facial deformities; one of the physicians at this Reference Centre wrote a definitive study on these risks.

Regarding benefits: the Pierre Robin Reference Centre at Necker Hospital in Paris cannot claim that they did not know about the safety and remarkable effectiveness of the TPP treatment in Germany; Dr. Abadie, the director of the Reference Centre, knows Dr. Poets personally, knows his work, and admitted that the medical effectiveness of the TPP treatment is “indisputable.”

If French parents of babies suffering from Pierre Robin Sequence were not given sufficient information to form reasoned decisions about the medical treatments which were being recommended and administered to their baby – including all relevant and material facts about the proposed treatment’s benefits, risks, and alternatives – then once again, serious questions of medical ethics are raised.

Setting aside EU law, these serious questions of medical ethics warrant a full and open investigation, all by themselves. We ask you, President Macron, to order such an investigation, and we ask you to allow those of us here in France whose children suffer from Pierre Robin Sequence to know the results of the investigation.

Violations of fundamental EU law: justifications and derogations

In 2010, the European Court of Justice issued a ruling in a case concerning a Bulgarian citizen suffering from cancer in his right eye, Case C-173/09. In this dispute, Bulgaria’s national healthcare system actually demonstrated that it was capable of successfully treating the cancer in this patient – however, the treatment proposed by Bulgaria would result in the loss of the patient’s eyesight in his right eye.

The patient learned that fellow EU Member State Germany not only offered a medical treatment which would achieve the goal of eliminating his cancer, but that the German treatment would eliminate the cancer in such a way that he would be able to keep his eyesight in his right eye.

The patient asked the Bulgarian national healthcare system for prior authorisation to obtain this medical treatment in Germany; the Bulgarian national healthcare system rejected his request. The matter went before the European Court of Justice.

To Bulgaria’s credit, the Bulgarian national healthcare system refrained from presenting the shameless and embarrassing fiction that the proposed treatment in Bulgaria, and the requested treatment in Germany, were “the same or equally effective” – even though both treatments would indeed successfully eliminate the patient’s cancer. The Bulgarian government was honest enough to admit that the German treatment was superior.

However, the Bulgarian government requested a derogation from Article 56 and from the obligations of EU cross-border healthcare law based, essentially, on a claim of poverty. The Bulgarian government argued that if a sufficient number of Bulgarian patients sought cross-border healthcare treatments in other EU Member States, and if the Bulgarian government had to cover the costs of such cross-border treatments, then it could potentially complicate Bulgaria’s ability to engage in healthcare planning, and it might even undermine the financial balance of Bulgaria’s entire social security system.

This attempt by Bulgaria to escape the obligations of EU law failed.

The truth is that the dire warnings of a hypothetical exodus of EU patients to other EU Member States, to obtain planned cross-border healthcare, has absolutely no basis in reality, and this is why the European Court of Justice rejected Bulgaria’s argument – in spite of the fact that Bulgaria is undoubtedly one of the poorer EU member states.

As the European Commission explained:

“Patients prefer to receive healthcare in their own country. That is why the demand for cross-border healthcare represents only around 1% of public spending on healthcare, which is currently around €10 billion. This estimate includes cross-border healthcare which patients had not planned in advance (such as emergency care for tourists). This means that, at present, considerably less of that 1% of the expenditure and movement of patients is for planned cross-border healthcare, like hip and knee operations or cataract surgery.”

Found here: http://europa.eu/rapid/press-release_MEMO-13-918_en.htm

Cross-border healthcare represents approximately 1% of public healthcare spending. This 1% figure includes two distinct components. The first component is planned cross-border healthcare; planned cross-border healthcare is simply healthcare which the patient has planned out in advance. The second component is unplanned cross-border healthcare – for example, when a German skier breaks his leg while skiing in the Austrian Alps, and receives emergency medical treatment in an Austrian hospital. Since the sum total figure for cross-border healthcare expenditure, i.e. both planned and unplanned added together, amounts to approximately 1% of public healthcare spending, planned cross-border healthcare represents just a fraction of this 1%. And since rare diseases are, by definition, rare, planned cross-border healthcare for EU citizens suffering from a rare disease represents literally a fraction of a fraction of 1% of public healthcare spending. Based on these miniscule figures, L’Assurance Maladie cannot possibly argue that refusing to provide Lysiane with authorisation to receive the TPP treatment in Germany was a necessary and proportionate measure to preserve the financial integrity of the French social security system.

Furthermore, unlike Bulgaria, France is one of the richest countries in the European Union. France’s healthcare system is well-funded, and provides its citizens with a balanced medical and hospital service which is open to all. In France there is no immediate shortage of medical resources, nor any structural or prolonged deficiencies in hospital facilities. France faces no problems maintaining treatment capacity, and the viability of France’s welfare system is not at risk.

Also, when one calculates the high cost of France’s ventilation assistance, which generally requires long-term hospitalization, and compares it to the cost of sending French Pierre Robin Sequence patients to Germany to obtain the TPP treatment – which typically requires two to three weeks of inpatient care – then it becomes clear that prescribing the TPP treatment would substantially reduce the expenses incurred by the French national healthcare system.

The European Court of Justice acknowledged Bulgaria’s legitimate need to engage in healthcare planning “to control costs and to prevent, as far as possible, any wastage of financial, technical and human resources”. However, L’Assurance Maladie, by preventing French patients from obtaining the TPP treatment, and instead forcing them to endure extremely costly long-term hospitalization, is directly violating all of these legitimate planning objectives.

L’Assurance Maladie’s rejection of Lysiane’s request for a medically proven and cost-effective treatment for her rare disease violates EU law – both primary law and secondary legislation – and represents the kind of arbitrary, irrational, unnecessary and unreasonable exercise of national discretion which the European Court of Justice has repeatedly struck down.

Conclusion

The legally binding Directive 2011/24/EU on the application of patients’ rights in cross-border healthcare aims to “foster developments of the diagnosis and treatment of rare diseases”, and “to facilitate improvements in diagnosis and the delivery of high-quality, accessible and cost-effective healthcare for all patients with a medical condition requiring a particular concentration of expertise in medical domains where expertise is rare”. The TPP represents exactly the type of high-quality cost-effective rare disease treatment which the 2011 Directive describes.

The TPP treatment corrects the underlying anatomical problem, Pierre Robin Sequence’s upper airway obstruction, allowing the baby to breathe naturally and independently, on her own – and the TPP treatment achieves this without any surgery. Ventilation assistance on the other hand simply forces air down the baby’s blocked airway; the baby’s airway, however, remains obstructed, and when the baby is disconnected from the ventilator machine, the breathing difficulties return. Furthermore with the TPP treatment there is no need for any external equipment. CPAP on the other hand does require equipment, a ventilator machine; this radically limits patient mobility, since the baby has to be connected to the ventilator machine almost around the clock. The TPP treatment also eliminates the need for long term hospitalisation, which CPAP generally requires, and which comes at a tremendous financial and human cost.

No objective and impartial medical comparison can reasonably conclude that CPAP, which requires the baby to be connected to a ventilator machine, and the TPP treatment, which achieves crucial medical objectives that CPAP cannot and does not achieve, are “the same or equally effective”. Medically, economically, and in terms of quality of life, the TPP treatment provides fundamental advantages over alternative Pierre Robin Sequence treatments, including CPAP. This is why babies from other EU Member States, and even babies coming all the way from Russia, have been transferred to Germany to receive the TPP treatment.

Article 13 (“Rare Diseases”) of the 2011 Directive sets out “the possibilities offered by Regulation (EC) No 883/2004 for referral of patients with rare diseases to other Member States even for diagnosis and treatments which are not available in the Member State of affiliation.” By email, CLEISS confirmed for us that the 2011 Directive grants EU citizens suffering from a rare disease the right to obtain a highly specialised treatment in another Member State, if it is not available in their Member State of affiliation.

L’Assurance Maladie’s refusal to grant prior authorisation for the highly specialised and medically proven TPP treatment for our child’s rare disease violates the 2011 Directive, Regulation (EC) No 883, and the fundamental principle of the freedom to provide and receive services under Article 56 of the Treaty on the Functioning of the European Union. It represents the kind of arbitrary, irrational, and unreasonable exercise of national discretion which the European Court of Justice has repeatedly struck down. It cannot be justified on any public policy grounds, and was neither necessary nor proportionate. This unfounded rejection should be reversed.

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