There are strict requirements for reimbursement of healthcare treatment abroad. These include compliance with The State of the Science and Practice. In late January, the Arnhem-Leeuwarden Court of Appeal ruled that the treatment procedure of the rare disease of an 11-year-old boy must be reimbursed by his health insurer. This patient suffers from Acute flaccid myelitis (AFM): an acute flaccid paralysis caused by inflammation of the spinal cord. Martin Buijsen, Professor of Health Law at Erasmus School of Law, discussed the the ruling by the court with EditieNL. "This is fairly unique because judges and health insurers have quite strict requirements for treatment abroad."
The State of the Science and Practice
The parents of the patient had been fighting for years to get treatment for their son. The insurer rejected their application for reimbursement several times, claiming the so-called Activity-Based Restorative Therapy treatment (ABRT treatment) did not meet The State of the Science and Practice. The parents hoped that court proceedings could provide a solution for reimbursement. This treatment is possible at the Kennedy Krieger Institute in Baltimore in the US. Through crowdfunding, the patient underwent this treatment in 2019, which positively affected ventilaton withdrawal and skill progression.
The court found that it is sufficiently plausible that the ABRT treatment meets The State of the Science and Practice. This requirement looks at scientific literature and evidence of effectiveness inpractice. The available substantiation is scarce because this is an extremely rare disease. In fact, there are only 15 AFM patients registered in the Netherlands. The court, therefore, finds that the insurer did not give it sufficient weight in assessing the case against The State of the Science and Practice.
"For a treatment to be collectively funded, it must legally comply with The State of the Science and Practice. This means that the Health Insurance Act requires proven effectiveness of a treatment", Buijsen explains. According to Buijsen, the best evidence of effectiveness can be provided by RCTs: randomised controlled (clinical) trials, research in which subjects are randomly divided into an intervention group and a control group. "The problem with this method is that it requires participation of a large number of subjects. In patients with rare conditions, such studies are hard to organise, and such evidence is hard to come by."
Less hard evidence may suffice
The court states that science and practice are united in the benchmark. This means that when scientific sources fall short, practice comes into play. The determining factor is to what extent practitioners regard such help as professionally correct practice. Buijsen: "The court has indicated in this ruling that less hard evidence will suffice. If no hard evidence can be provided because the condition is rare and RCTs are consequently difficult or impossible to organise, the degree of acceptance of the treatment in practice becomes more important. In that case, even the opinions of a few experts may suffice, as in the present case."
Treatments for rare diseases
Buijsen explains that conditions within the EU are rare if fewer than 5 in 10,000 people have the condition. Rare diseases are chronic, often disabling and sometimes fatal conditions. "However, the problem is that the number of rare conditions is large. Estimates range from 5,000 to 8,000. The chance of getting a rare condition is 1:17. That chance is not that insignificant at all, and according to estimates, 6 to 8 per cent of the Dutch population suffers from a rare condition: around 1 million people”, Buijsen satates. The situation is no different in other European countries and this ruling, according to Buijsen, should therefore not be seen as a judgment by a judge in an extravagant case, a case of extreme and rare suffering. "It concerns a big social problem.”
Ethical considerations
Buijsen explains the ethical considerations involved in rejecting reimbursement for treatments that could potentially be life-enhancing for patients with rare diseases. "It is obviously immoral to reimburse a treatment that is not effective.” If a treatment is effective, the question of efficiency becomes relevant, Buijsen says. "The resources to collectively fund treatments are not infinite. If the same result can be achieved in another way with the use of fewer resources, that more cost-effective treatment should be in the package", Buijsen argues. The problem with treating patients with rare conditions is that existing therapies are usually only marginally effective and very expensive, and more cost-effective alternatives are lacking. "This means that a relatively small group of patients can greatly demand available resources. And we have a hard time coping with this dilemma in the Netherlands."
- Professor
