"Using an innovative drug can decide over the life and death of a child"
Interview with Pierluigi Brazzola, MD, specialist in paediatric and adolescent medicine, paediatric oncologist and Head of the Paediatrics/Haematology Department at the Istituto Ospedaliere della Svizzera Italiana, Bellinzona
Dr. Brazzola, around 90 per cent of all medications used for children with cancer are not on what is called the speciality list. For the therapy costs to be covered, an application has to be submitted to the insurer for them to decide on whether costs will be covered or not. What does that mean in practice?
Cancer in children and adolescents is different from in adults. The disease is not only rarer, but it is also more aggressive. Therefore, we have to start treating our patients immediately as soon as the diagnosis has been made. Even though we know that we have to apply for the costs to be covered, we often do not have the time to wait for an answer. Some of the drugs we use to treat leukaemia fall into this category. It is important to know that leukaemia is the most common cancer in childhood and adolescence. The time required for these administrative procedures is thus considerable. In addition, not all insurers react in the same way and within the same time frame.
Most children are treated with standard therapies that have proven effective for decades. So why are some cost approvals not successful?
The insurers and their medical officers sometimes assess the applications according to fixed criteria that cannot always be met. In some cases, it is also difficult to prove why we want to prescribe a particular medicine in a child-friendly dosage form. But because an infant or young child, unlike an adult, cannot swallow tablets or cannot swallow them very well, we opt for a perhaps somewhat more expensive medicine in syrup form instead of tablets. Children have different needs from adults and it is important to take these into account. Certain decisions by insurers should therefore not be made solely on the basis of cost, effectiveness and sustainability. Especially in the case of vulnerable patients such as children, the focus should also be on practicality.
What about additional therapies that help prevent life-threatening infections or improve the quality of life of young patients?
There can even be problems with cost coverage in the case of additional therapies. What are referred to as supportive therapies comprise medication against nausea, pain and infections and are very important for our patients during treatment. It might be the case, for example, that the health insurance company or invalidity insurance (IV) won’t cover the costs if a medication is in a certain dosage form, such as syrup, that is not available in Switzerland and thus has to be imported from abroad. The prerequisite for approval would be that we could prove greater effectiveness or lower costs. It seems that in such cases common sense is not enough to realise that it is easier for a two-year-old child to take syrup than a tablet.
Innovative cancer therapies can be promising alternatives if the initial treatment does not work or if the cancer comes back. What is the situation with these newer and more expensive medications?
Until just a few years ago it was very difficult to find an alternative for different types of childhood tumours when they recurred. Luckily, today, research in paediatrics is making rapid progress in this field, too. In some situations using an innovative drug can decide over the life and death of a child. Or whether our patients experience fewer side effects and long-term consequences than with a standard therapy. When we are treating children we must not forget that they have a lot of years left in front of them. The question of the quality of life is therefore another factor in the choice of therapy. A lower risk of developing health problems in the future can thus also be decisive.
What happens in these cases if the medical officer of a health insurance company has turned down an application for costs to be covered? Who then pays for the life-saving therapy?
When an application is turned down, we usually contact the health insurance company again to ask why. We want to know the exact reasons to be able to react accordingly. We try to find arguments and evidence that will convince the medical officer of the benefits. But medical officers are not usually paediatric oncologists. So they often don’t have the necessary expertise. If they won’t reconsider, we contact the manufacturer of the particular medication and foundations to find alternative solutions. That all takes up a lot of time but we try everything to avoid the parents having to cover the costs of the therapy themselves.
To what extent do objections and unnecessary bureaucracy take their toll on doctors and parents?
For the doctors, we have to spend an incredible amount of time and energy on these applications. But for the parents concerned, the current situation is a nightmare. Naturally, they don’t understand why therapies that could save their child’s life are evaluated from a purely economic point of view. We paediatric oncologists always try to cure the illness at the first attempt. Because if a patient suffers a relapse, there are not always effective therapies available. Therefore, we have to use every small improvement in the chances of recovery. Parents know that. The idea that their child may not have access to a therapy because the medical insurance or the invalidity insurance (IV) does not cover the costs is extremely stressful. Especially if they do not have sufficient financial resources to pay for the therapy costs themselves in an emergency.
In your view, what could be a possible solution scenario in the assessment of contentious cases?
We paediatric oncologists are obliged to strictly adhere to the guidelines of international, standardised treatment protocols. These have been tried and tested for decades and make it possible to maintain the highest standards of treatment in Switzerland as well. No therapy is the initiative of a single doctor. That should really be proof enough that our suggestions have already been checked in terms of their effectiveness. We also discuss complex diseases on a case-by-case basis in an international group of experts where, after careful consideration, a consensus is then reached on how to proceed.
In contentious cases, when the insurer refuses to cover the costs on the basis of a recommendation of the medical officer, there should be an independent expert committee. This should comprise specialists from paediatric oncology and should be given the power to issue a statement that is binding for both parties. Unfortunately this kind of committee doesn’t actually exist.
In the case of newer medications, the Federal Council now wants proof that these will bring an added value of 35 per cent in comparison to standard therapies. Clinical trials are to be used as a basis for evidence. What do you think of this?
There is virtually no medication that would be able to meet this demand. The progress made in paediatric cancer medicine over the last 40 years has sometimes reached this threshold. But only in a slow and continuous way. No one therapy has had such success so quickly and on its own. As already mentioned, paediatric oncology is not only about better chances of curing someone, but also about reducing potential long-term side effects. Today, three out of every four patients are at risk of having long-term complications. So one of our major challenges is to cure and at the same time minimise long-term consequences. This aspect is something that is virtually missing in the treatment of adults. The proposed changes have hardly any genuine benefits. In fact, there is a real danger that access to necessary therapies will become even more difficult for our patients.
From your point of view, what would be the idea solution to ensure that children and adolescents suffering from cancer also have access to the best possible therapies in future?
As childhood cancer is a rare disease, the case numbers in Switzerland are low. The approximately 350 children and adolescents who are diagnosed with cancer every year here have to be treated throughout Switzerland according to strictly prescribed international trial protocols. These were first approved by Swissmedic and verified by Swiss Ethics Commissions. Their great therapeutic benefit has thus already been officially recognised. It would therefore be desirable for all medications that the standard therapies prescribe to be automatically reimbursed by the insurers. Better coverage of costs for additional therapies would also be important. As already mentioned, an independent committee of paediatric oncology experts could make decisions binding for all parties in contentious cases. A corresponding pilot project in adults has already been carried out and has produced very encouraging results. That certainly has to happen more and more.