Letter to President Macron

Part 1: Background Information

23 February 2018

Monsieur le Président de la République Française
Palais de l’Elysée
55 Rue du Faubourg Saint-Honoré
75008 Paris
France

Re: Lysiane Pakter – French baby with a rare disease

If a newborn French baby suffering from a rare disease, immobilized in an intensive care unit and connected to a ventilator machine, doesn’t have the right to obtain a highly specialised, medically proven and cost-effective treatment for her rare disease in another EU Member State – then who in France does have the right to obtain cross-border medical care?

Dear President Macron,

My partner and I are writing to you to urgently request your intervention in a matter involving our baby, Lysiane, who suffers from a rare disease, Pierre Robin Sequence, and our continuing struggle to obtain an S2, L’Assurance Maladie’s administrative approval, for a highly specialised, medically proven and cost-effective treatment, the “TPP” treatment in Germany, for her rare disease. Our intervention request is for your assistance obtaining this S2, which we were wrongfully denied.

France’s Reference Centre for this rare disease confirmed that the TPP treatment is not available in France, but that it is available in fellow EU Member State, Germany. Nevertheless, L’Assurance Maladie rejected our request for this treatment, and denied us the S2. In spite of our efforts, L’Assurance Maladie continues to maintain its unfounded rejection; this is why we have finally decided to make this heartfelt appeal to you. We as parents want this administrative ordeal with L’Assurance Maladie to finally be resolved, so that we can focus our energy where it belongs: on our baby Lysiane, who suffers from a rare disease.

L’Assurance Maladie’s rejection of our request for this highly specialised rare disease treatment violates the 2011/24/EU Directive on Cross-border Healthcare, Regulation (EC) No 883/2004 on Social Security Systems, 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 infringes upon the EU’s four fundamental freedoms – the free movement of goods, services, people and capital; it undermines a key EU policy objective at the heart of the European project – the effort to create a single EU market; and it cannot possibly be justified as “necessary and proportionate” on any public policy grounds. We have a growing list of individuals and international organizations who have carefully analysed our case, and who openly agree with our position. Attached please find several letters of support, beginning on page 4. Our allies include:

  1. the European Commission’s SOLVIT network, which agrees with our legal position, Case Number 2569/17/DE;
  2. the Member of Parliament representing our district in France, Mr. Bruno Bonnell, of the Assemblée Nationale; Mr. Bonnell is an active defender of patients’ rights, and people with disabilities; he is supporting us in our appeal;
  3. EURORDIS, the largest rare disease patient organization in the EU; EURORDIS has pledged to accompany us in court, all the way up to the European Court of Justice; EURORDIS has sent a formal letter of support to L’Assurance Maladie;
  4. a Member of European Parliament, Ms. Françoise Grossetête, who has extensive experience working with the 2011/24/EU Directive on Cross-border Healthcare; Ms. Grossetête has sent a formal letter of support to France’s Minister of Health, Ms. Agnès Buzyn;
  5. the European Patients’ Forum, the largest patient advocacy umbrella organization in the EU, and a key stakeholder in the drafting of the 2011 Directive; the European Patients’ Forum has sent a formal letter of support directly to you, President Macron;
  6. an international network of law professors who specialise in EU cross-border healthcare and social security law, who agree that L’Assurance Maladie’s rejection was unfounded.

As we explain in this document, the treatment which is used in France to treat our baby’s rare disease, Pierre Robin Sequence, consists of keeping the baby in a hospital, attached to a ventilator machine. Since ventilation assistance generally requires long term hospitalisation, the French treatment creates colossal healthcare costs, and it uses up precious space in neonatal intensive care units. Germany’s highly specialised TPP treatment, on the other hand, is medically proven to resolve the upper airway obstruction associated with this rare disease. The TPP treatment liberates the baby from the ventilator machine without surgery, and without long term hospitalization, making it a cost-effective medical breakthrough in the treatment of this rare disease.

Thus, not only does the French treatment have the disadvantage of keeping the baby attached to a ventilator machine, substantially reducing mobility, and quality of life – but in addition to this, it doesn’t even make financial sense. For L’Assurance Maladie in France to deliberately obstruct a French baby’s access to the medically proven German treatment, which dramatically improves the baby’s ability to breathe, and quality of life – and which reduces healthcare costs, by eliminating the need for long term hospitalisation – is as irrational as refusing to adopt email, and stubbornly insisting on using expensive and cumbersome fax machines instead.

Since the lives and suffering of newborn babies is at stake, L’Assurance Maladie’s rejection doesn’t just defy logic; it is also unconscionable. No ethical doctor or civil servant should force a newborn baby suffering from a rare disease to endure long term intensive care, with all of the burdens this entails, when fellow doctors in the EU Member State just next door have successfully developed a cost-effective medical treatment to safely get the baby off of the ventilator machine, and home to her parents, where the baby belongs. Doctors should not play God; they should facilitate patient access to care, not obstruct patient access to care. A patient’s rights should take priority over a physician’s ego.

L’Assurance Maladie’s unfounded rejection is particularly inappropriate now, with Brexit. The cohesion of the EU has become a central issue; each Member State should solemnly respect its obligations vis-à-vis EU law. “Europe is crossing an important milestone,” as you have said. Now is not the time for a leading Member State like France to act as if EU law should apply to other Member States, but not to her. Intentional obstruction, market interference, and non-cooperation is not at all how the EU community is supposed to operate.

Furthermore you have expressed your wish to “totally integrate” the French and German markets, and “restore a strong trust between France and Germany.” L’Assurance Maladie’s senseless refusal to authorise French babies to receive this breakthrough treatment in Germany casts doubt upon France’s commitment to this goal. If France is unwilling to cooperate with Germany to reduce the suffering of newborn French babies who are struggling to breathe, then France will have a very hard time cooperating with Germany on major international projects which are actually divisive and complex.

This case raises an issue of wider principle which matters to all French citizens, and which affects their daily lives: if a newborn French baby suffering from a rare disease, immobilized in an intensive care unit and connected to a ventilator machine, doesn’t have the right to obtain a highly specialised, medically proven and cost-effective treatment for her rare disease in another EU Member State – then who in France does have the right to obtain cross-border medical care?

Mr. President, this document, with its extensive evidence, and letters of support, make it clear that L’Assurance Maladie’s rejection was unfounded. My partner and I urgently request your assistance obtaining Lysiane’s S2, which L’Assurance Maladie illegally and senselessly denied. We hope to hear from you very soon. Thank you.

Sincerely,

Philippe Pakter and █████ ███████ – the parents of Lysiane
2 rue Gérard Maire
69100 Villeurbanne
France

Letters of Support page 4
Summary of Dispute page 20
Background Information page 22
Medical Analysis page 33
Legal Analysis page 62
Conclusion page 73