GREV WEDELS PLASS, OSLO (Dagens Medisin): Onasemnogene abeparvovec (Zolgensma) is introduced to treat spinal muscle atrophy.
- After a long, almost three-year process, we have come to an agreement. It is an assessment of benefit to patients based on available documentation, effectiveness and cost proportionality. As Inger Cathrine Bryne, chairwoman of the Decision Forum for Dagens Medisin, says.
"This is a comprehensive decision with many criteria," says Bryne, who is also CEO of Helse Vest.
The most expensive medicine in the world
On Monday, the Decision Forum received a case of what was previously referred to as "the most expensive medicine in the world" on its table.
A forum, made up of regional health authority (RHF) governors, decides whether methods should be used in the public health service.
The assessment was of a historical nature. Because for the first time, the Decision Forum has opened up to a performance-based deal, not just a flat-rate discount.
Onasemnogene abeparvovec (Zolgensma) is used against the genetic disease of spinal muscular atrophy (SMA). Dagens Medisin announced earlier this week that the drug recently received the green light in three European countries as a result of international cooperation.
Limited transparency
Bryne says the forum cannot come out with all the details of the new deal.
- There is a limit to what we can say about an agreement, but it is a question of performance goals. The pharmaceutical company wants the details of this not to be disclosed. But it's about motor performance, the need for respiratory support and, of course, death is a very serious disease.
Bryne also cannot reveal how much the contract will cost during the year as a result of an agreement with the company.
- But we found the agreement to be within acceptable limits.
Complicated structure
- It is a complicated contract structure, a contract based on the results. Therefore, good cooperation with the national professional group for SMA has long been extremely important. In addition to Procurement by Oslo University Hospital and Hospital Pharmacies - on all possible professional and legal matters, which are part of the basis of this agreement.
Treatment will be carried out at Oslo HF University Hospital (OUS) and the treating physician will record the relevant treatment data in the SMA registry so that the effect can be tracked over time in close collaboration with the national SMA professional group.
The most expensive medicine in the world was introduced in Norway. Newborns will receive the drug
A diagnosis made before the onset of symptoms, that is, before symptoms, is one of the inclusion criteria (see list of criteria at the bottom of the case).
Thanks to screening of newborns for SMA, which was introduced on September 1. It is possible to detect the disease in infants before symptoms appear.
- Babies diagnosed at neonatal screening with two or three copies of this gene - will be relevant for this treatment. This will likely be the most common application, says Terje Rootwelt, CEO of Health South-East at Dagens Medisin.
From the head of the clinic to a member of the Decision Forum
Rootwelt, until recently head of the Department Children and Youth at the University Hospital in Oslo. He previously described the drug as "obviously a very exciting treatment option" for Dagens Medisin.
He was involved in making today's decision.
- It is so that impartiality ratings are related to whether or not you are getting personal gain. Or the benefits of participating in the decision-making process. It is quite obvious that both the Contracting Forum and the Decision Forum have doctors with them when making decisions and that this is a great advantage for the quality of the decisions made, says Bryne of Rootwelt's participation in the forum.
The doctor himself is pleased with today's decision.
- This process has been going on for several years with the extensive involvement of the professional community. It is a long process that has led us to make this decision, which I find satisfactory.
The use of the drug for SMA was introduced, when?
The drug is due to be introduced as soon as possible and we want to start using the new treatment, but this is not yet possible, says leader Bryne.
- There is something that is not quite finished. This applies to privacy, GDPR and is related to the transmission of information between OUS and the company. This is something the company should introduce. We hope it will take a very short time.
In the minutes of the meeting we read that “before the contracts can come into force and apply the method. Novartis Gene Therapies must develop a satisfactory solution for the encryption of personal data transmitted when placing an order and other communication between Sykehusapotekene / Oslo University Hospital and Novartis Gene Therapies. "
a drug for SMA in Norway. The following criteria are used as a basis for initiating treatment with Zolgensma:
- Infants <6 months of age diagnosed with 5q SMA type 1 with a mutation in the SMN1 gene. The patient should have no obvious symptoms at birth (type 0) and should not need ventilator support
- Infants <6 months. with before symptomatic bi-allelic mutation in the SMN1 gene and 2 or 3 copies of the SMN2 gene detected
- Children 6-18 months. and those weighing <13,5 kg, in whom treatment with Spinraza resulted in adverse reactions or complications, switching to Zolgensma may be considered in special cases, if they are not clearly impaired and can breathe independently
- Patients <6 months of age. who started treatment with Spinraza before Zolgensma was available can be upgraded to Zolgensma
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Source: Dagens Medisin.
