Prioritisation and funding of expensive treatments

• Abstract
• Purpose of health policy or idea
• Characteristics of this policy
• Political and economic background
• Purpose and process analysis
• Expected outcome
• References

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Abstract

In recent years some very expensive drug therapies have emerged for treatment of rare enzyme deficiency diseases, i.e. Fabry disease, Gaucher disease and mucopolysaccharidosis. In February 2005 the Parliamentary Ombudsman stated that, according to law, enzyme replacement treatment must be given and prioritisation decisions can not be based on economical grounds.

Purpose of health policy or idea

In recent years some very expensive drug therapies have emerged for treatment of rare enzyme deficiency diseases. These diseases include for example Fabry disease, Gaucher disease and mucopolysaccharidosis I (MPS I, also known as Hurler, Hurler-Scheie, and Scheie syndromes). The duration of these new treatments is life-time and the costs are about 200.000-350.000 eur/patient/year in Finland. Research solely conducted by the pharmaceutical company shows that there is some benefit of the treatment to the Fabry patients, but benefits of long term treatment is largely unknown. Because of the high costs and uncertain evidence on benefits, some hospitals have been reluctant to offer the treatment. In Finland there are about 30 Fabry patients who live mainly in the area of one hospital district.

The Finnish Fabry Society made a complaint of the situation of Fabry patients to the Parliamentary Ombudsman. The task of the Parliamentary Ombudsman is to oversee the legality of actions taken by the authorities independently from the parliament. In February 2005 the Parliamentary Ombudsman put forward a statement that according to law, enzyme replacement treatment must be given to the patients. In addition the Ombudsman stated that according to her conception, prioritisation decisions can not be based on economical grounds.

Main points

Main objectives

The decision by the Parliamentary Ombudsman forced hospitals to give treatment for Fabry patients. The decision may also have broad implications, because it stated that cost of the treatment can not be a reason in decision-making concerning priority setting in health care.

Type of incentives

The task of the Parliamentary Ombudsman is to oversee the legality of actions taken by the authorities independently from the parliament. Incentive of Fabry patients is to get the treatment. 

Groups affected

Fabry patients and patients with other rare enzyme deficiency diseases, Authorities (hospital districts, municipalities) and politicians involved in health care administration.

Innovationsgrad	traditionell		innovativ
Kontroversität	unumstritten		kontrovers
Strukturelle Wirkung	marginal		fundamental
Medienpräsenz	sehr gering		sehr hoch
Übertragbarkeit	sehr systemabhängig		systemneutral

The decision of the Parliamentary Ombudsman is an important step in the discussion on how priority-setting decisions should be made in health care.

Political and economic background

 Research on and access to orphan drugs has received attention in EU during last few years. To get license orphan drugs does not need as good quality evidence on benefits as normal drugs. However, lack of good quality evidence is a problem for payers when assessing the effectiveness of the treatment.

Municipalities have had serious problems in Finland to keep the increase of health care expenses reasonable.

 In recent years, municipalities and municipality funded hospital districts have argued that the state should pay very expensive treatments instead of municipalities which are responsible for providing and funding public health care. There has been also public discussion in media specifically on Fabry patients' problems of receiving necessary care.

In this context the Parliamentary Ombudsman assessed if the actions taken by the hospital authorities concerning the medication for Fabry patients follow the constitution and other legislation.

Complies with

Constitution of Finland and other legislation

Purpose and process analysis

Current Process Stages

	Idee	Pilotprojekt	Strategiepapier	Gesetzgebung	Umsetzung	Evaluation	Veränderung/Richtungswechsel
Implemented in this survey?

Origins of health policy idea

Enzyme replacement therapy for the Fabry disease became available in 2001. In 2002 the pharmaceutical company who developed enzyme replacement therapy conducted very small scale clinical study in Finland during which company provided therapy for free for 13 patients. While the free enzyme treatment was not available anymore after the study ended in 2003/2004, the enzyme replacement therapy was stopped by the hospital district responsible for these Fabry patients' treatment due to the high costs (200.000 euros/patient/year) and uncertain evidence on benefits. Municipalities and hospital districts tried to pressure the Finnish government to fund very expensive treatments, with no success (in Finland municipalities are responsible for funding public health care).

As a response, the Finnish Fabry Society made a complaint to the Parliamentary Ombudsman. One argument was that enzyme replacement therapy is provided in many other European countries.

In her decision the Parliamentary Ombudsman pointed out, that the Finnish legislation does not allow that some patients are left without treatment they need because of costs of the treatment. The decisions should be solely based on need of the patient, nature of disease and effectiveness of the treatment.

Initiators of idea/main actors

• Regierung

• Parlament

• Leistungserbringer

• Kostenträger

• Patienten, Verbraucher

• Bürgergesellschaft

• Privatwirtschaft, privater Sektor

• Medien

• Meinungsführer

• Andere

Stakeholder positions

The complaint was made by the Finnish Fabry Society. Pharmaceutical companies producing enzyme replacement drugs and some clinicians supported the idea.

 The authorities of hospital districts wanted that the government would fund the enzyme replacement treatment and other very expensive treatments. Some of them doubted that cost effectiveness of treatment was too poor.

After the decisions some experts opposed the idea of the Parliamentary Ombudsman that costs can not be considered when setting priorities in health care.

Actors and positions

Regierung
Government	sehr unterstützend		stark dagegen
Parlament
Parliament	sehr unterstützend		stark dagegen
Leistungserbringer
Providers	sehr unterstützend		stark dagegen
Kostenträger
Payers	sehr unterstützend		stark dagegen
Patienten, Verbraucher
Finnish Fabry Society	sehr unterstützend		stark dagegen
Bürgergesellschaft
Civil Society	sehr unterstützend		stark dagegen
Privatwirtschaft, privater Sektor
Pharmaceutical industry	sehr unterstützend		stark dagegen
Medien
Media	sehr unterstützend		stark dagegen
Meinungsführer
Some clinicans treating these patients	sehr unterstützend		stark dagegen
Andere
Parliamentarian Ombudsman	sehr unterstützend		stark dagegen

Actors and influence

Regierung
Government	sehr groß		kein
Parlament
Parliament	sehr groß		kein
Leistungserbringer
Providers	sehr groß		kein
Kostenträger
Payers	sehr groß		kein
Patienten, Verbraucher
Finnish Fabry Society	sehr groß		kein
Bürgergesellschaft
Civil Society	sehr groß		kein
Privatwirtschaft, privater Sektor
Pharmaceutical industry	sehr groß		kein
Medien
Media	sehr groß		kein
Meinungsführer
Some clinicans treating these patients	sehr groß		kein
Andere
Parliamentarian Ombudsman	sehr groß		kein

Positions and Influences at a glance

Adoption and implementation

Due to the Parliamentary Ombudsman's statement, the hospital districts are starting to provide expensive enzyme replacement therapies to the patients who need them. The hospital districts have already decided on guidelines on how patients should be diagnosed and treated.

 Although scientific evidence on benefits of enzyme replacement therapy is not very convincing, the Parliamentary Ombudsman stated that enzyme replacement treatment is necessary treatment for Fabry patients. The decision may have an influence on the assessment of scientific evidence on other treatments also.

The further impact of the statement regarding costs as a reason of priority setting is difficult to predict. In the future, the implications for legislation and policy making have to be reconsidered because an increasing number of very expensive treatments targeted to small patient groups can be expected to be introduced.

Monitoring and evaluation

There is not any explicit monitoring or evaluation mechanism for this process.

Expected outcome

 In practice, costs are one component when priority setting decisions are made in public health care in Finland (for example cost-benefit analyses). This is in conflict with the view of the Parliamentary Ombudsman that costs should not be considered when making prioritisation decisions. While very expensive treatments targeted to a small patient group are expected to be increasingly introduced in the future, a thorough assessment should be conducted on the conflict between legislation and prioritisation policies.

Impact of this policy

Qualität	kaum Einfluss		starker Einfluss
Gerechtigkeit	System weniger gerecht		System gerechter
Kosteneffizienz	sehr gering		sehr hoch

The decision had significant positive impact to the patients having rare enzyme deficiency diseases. The decision made more difficult to include cost-benefit measures in health care decision-making.

References

Sources of Information

Decision 921/4/04 of the Parliamentary Ombudsman of Finland (only in Finnish)

http://www.oikeusasiamies.fi/Resource.phx/eoa/english/index.htx

Author/s and/or contributors to this survey

Lauri Vuorenkoski & Sirpa-Liisa Hovi

Empfohlene Zitierweise für diesen Online-Artikel:

Lauri Vuorenkoski & Sirpa-Liisa Hovi. "Prioritisation and funding of expensive treatments". Health Policy Monitor, April 2005. Available at http://www.hpm.org/survey/fi/a5/4