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11 December 2019
The Swedish system for medicinal products – including its reimbursement system – is generally product based. Prescriptions as such are product based (ie, by brand name or generic product name) and the indication for which the product is intended cannot be filled in anywhere by the prescriber. Off-label prescriptions are therefore not generally possible in the 'official' prescription system.
However, when it comes to accessing unlicensed medicinal products, the system for licences on a named-patient basis works differently: the prescriber must add a rationale or motivation for the licence and provide supporting documentation. This rationale or motivation will normally include the indication. Prescription for the off-label use (of an unlicensed product) is possible under normal off-label prescription requirements in Sweden (ie, it should be based on science and proven experience). The Medical Products Agency (MPA) then assesses the treatment need of the individual patient and whether it can be fulfilled by a medicinal product approved in Sweden.
In a number of cases, the holder of a marketing authorisation has been found not to have standing before the courts in matters relating to licences on a named-patient basis. This is especially unsatisfactory for orphan drugs, as this article explains.
The medicinal products in question contain the active substance Idebenone, which is off-patent and has been allegedly used for a long time against various indications, such as certain mitochondrial diseases. The original product has been deregistered in Sweden, but remains available in other countries (eg, Italy).
In 2007 a company was granted orphan drug designation for a product containing Idebenone for treating Leber's hereditary optic neuropathy (the Idebenone orphan drug). A marketing authorisation for the product was granted to the company in 2015.
In the beginning of 2018, a prescriber together with a pharmacy (in accordance with regulations) filed a prescription of the unlicensed original product on a named-patient basis. The application was granted by the MPA. The decision was challenged by the marketing authorisation holder of the Idebenone orphan drug, which was the only approved product containing the substance on the Swedish market.
The administrative court found that the marketing authorisation holder lacked standing before the court, referring to its previous case law. The marketing authorisation holder appealed this decision to the administrative court of appeal.
In its appeal, the marketing authorisation holder argued that the named-patient licence for the original product had been incorrectly granted and that the general rule was that a licence on a named-patient basis could be granted only if the need for a medicinal product could not be fulfilled by a product approved in Sweden.
The marketing authorisation holder referred to Swedish case law on:
The marketing authorisation holder further stated that:
It was claimed that in all matters concerning a named-patient licence for Idebenone, the MPA should initially investigate whether the approved Idebenone orphan drug was suited for the treatment that the application concerned and explicitly state why the Idebenone orphan drug could not be used instead of the original product.
The MPA stated that the case law referred to by the marketing authorisation holder concerned a specifically regulated situation and could not be used as grounds for other situations.
The administrative court of appeal denied the appeal. According to the court, the circumstances in the case differed from the case law referred to by the marketing authorisation holder. Although the essential aim for all production, distribution and use of medicinal products in the European Union (ie, to safeguard public health) was still valid, the primary aim for a licence on a named-patient basis was to safeguard the treatment need of a specific patient and offer that specific patient good quality healthcare.
The court further reasoned that, when assessing an application for a licence on a named-patient basis, the MPA cannot assess whether a medicinal product fulfils the requirements to be approved as a fundamental element. Thus, the situation in the referenced case law was not relevant and there were no safeguarding reasons to grant the marketing authorisation holder standing before the court.
The court found no reason to question that the marketing authorisation holder's financial position would have be affected should the Idebenone orphan drug have been prescribed to the patient instead of a named-patient basis licence having been granted. However, the court held that this factor was due only to the companies being competitors and their respective interest in sales, finding no support in regulation or case law that such interest should be considered in a licence decision by the MPA or that this issue in itself could form the basis for standing before the court.
In conclusion, the court found that the marketing authorisation holder neither represented an interest protected by the legal system that should be considered within due process nor could be considered to have a legal interest worthy of consideration to have the legality of the decision questioned. Therefore, the administrative court had been correct in denying the marketing authorisation holder standing before the court.
In the case in question it would appear that the approved 'on-label' indication is quite rare, while there has been a wide off-label prescription for many indications which appear to be more prevalent. The treatment cost when using the Idebenone orphan drug is naturally high given the marketing authorisation procedure for that product, higher in fact than using the original product. Off-label prescription of the Idebenone orphan drug in the 'official' prescription system would therefore be quite costly.
The MPA guidelines for licences on a named-patient basis explicitly state that financial reasons cannot justify a licence on a named-patient basis (eg, the licensed product is cheaper than the approved alternative or the patient gets a product which is reimbursed when the approved alternative is not).
However, given the differences between the abovementioned systems, any off-label prescription for a licence on a named-patient basis will not breach regulations – while a product may contain the same active substance, it remains possible that no medicinal product for that indication approved in Sweden can fulfil the need of an individual patient.
For a marketing authorisation holder to which the MPA has not prescribed a licensed product based on the indication of its approved product, transparency is available by a freedom of information request of the rationale or motivation for the licence (any patient information will be redacted).
Nevertheless, it is questionable why cases where the holder of an orphan drug with a small patient population in Sweden should not get standing before a court to get a judgment on the substantive issues of a named-patient basis licence, as generally not many granted licences would be required to fulfil a large part of or even the full treatment need for Sweden. Consider, for example, a case where it is questioned whether the prescription for a named-patient licence is off or on label of an approved product.
For further information on this topic please contact Jonas Löfgren at Westerberg & Partners Advokatbyrå Ab by telephone (+46 8 5784 03 00) or email (firstname.lastname@example.org). The Westerberg & Partners Advokatbyrå Ab website can be accessed at www.westerberg.com.
The materials contained on this website are for general information purposes only and are subject to the disclaimer.
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