Cost-effectiveness: the need to know
- John Marley
- Aust Prescr 1996;19:58-9
- 1 July 1996
- DOI: 10.18773/austprescr.1996.056
Last year a British health authority refused to pay for further treatment for Jaymee Bowen, a girl suffering from a rare form of childhood leukaemia. Jaymee had already relapsed after chemotherapy and a bone marrow transplant. The health authority considered the cost-effectiveness of further treatment and, taking account of the low chance of success, plus the extra suffering involved, refused to pay the estimated $150 000 the treatment would cost. To Jaymee's father, any chance of survival was worth trying. The publicity he achieved resulted in an anonymous benefactor coming forward to pay privately for an experimental treatment. Jaymee survived.
Australia has led the world in requiring pharmaceutical companies to submit economic analyses when applying for public subsidy for the supply of new drugs.1 However, the data submitted to the government are usually 'commercial in confidence'. They are not in the public domain where they would be available to prescribers and patients. This secrecy raises many serious questions.
The community ultimately pays for all subsidised drugs, yet the community is denied access to the information it needs to judge whether its money is being spent wisely. Whether the decision to list or not list a drug on the Pharmaceutical Benefits Scheme (PBS) is right and in accordance with society's values and priorities cannot currently be judged.
Commonly, when prescribers reach for their prescription pads, they choose an individual drug from a number of classes or from within the same class. They assume that the drug chosen is both efficacious and safe. If they wish to check their assumptions, even though the licensing application is confidential, they will be able to find peer-reviewed efficacy and safety data in the literature. They presently have little hope of checking the cost-effectiveness of the treatment they are prescribing. Prescribers are required to have sufficient information to enable patients to make informed choices, which may include paying a greater co-payment or full price for a non-PBS listed drug. Health economic data cannot usually be obtained from either the government or the manufacturer and, in particular, comparative data for drugs in the same class are not available. In the current climate of secrecy, how are prescribers able to counsel patients fully? If Jaymee Bowen had been in Australia, would her doctors have had access to sufficient data to be able to check the decision of the authority or to advise her father? Interferon beta-1b is approved for use in the relapse-remitting type of multiple sclerosis. It is very expensive: approximately $20 000 per patient per year. It has to be injected on alternate days, not everyone responds, it has no proven effect on disability and frequently has adverse effects. If a patient with multiple sclerosis asks whether paying for interferon would be good value for their money, how is a doctor to answer? If interferon remains unlisted on the PBS, how can the correctness of that decision be scrutinised?
Consumers make purchases on the basis of the perceived value of a commodity to themselves and their families. One family, for example, will buy the cheapest toilet paper while another will make an individual choice to pay more for the softest, or the most expensive. However, for pharmaceuticals, the patient -purchaser is usually completely uninformed because the doctor -provider and pharmacist-vendor are denied access to many of the pertinent facts and part of the bill may be paid by a third party, the tax payer. The lowest price may not be the best all round value: cheaper toilet paper may have fewer sheets per roll. If the newer antidepressants are better tolerated, they may reduce costs to society overall compared with the older tricyclics, despite an increased cost of medicines per day. Consumers should be in a position to choose for themselves, on the basis of freely available cost-effectiveness data, whether they will elect to pay for a marginal benefit not provided to society as a whole. To Jaymee Bowen's father, parent of a dying child, any marginal cost, however large, was worth paying.
Economic analyses should be published like any other research data. If they are not, why not? The obvious question is, what is there to hide? Freely available information would make it easier to defend decisions about subsidising or not subsidising the cost of drugs. Both licensing and economic data submitted to government are currently protected by legislation, which could be changed. Economic data are most important when a drug has received marketing approval and its subsidy has become a public concern. Perhaps there is a need to have an economic information document outlining the cost-effectiveness of a product in the same way that there is a data sheet on the indications, dosage, etc.
The Jaymee Bowen story occupied much space in the news media and international medical journals last year. In defence of its position, the health authority pointed out that in the same week it had approved a $600 000 treatment for a boy with haemophilia. Had more data, rather than just the emotional facts, been available from the start, a more balanced discussion might have been possible.
On emotional grounds, the public rejects the idea that any treatment, however expensive and unproven, should be denied. Jaymee said in a message to the health authority, 'Now look at me, I'm fine. You could have paid for it. You had the chance and you blew it'. Any new development will always be demanded by the desperate, especially when promise is offered but not the detail with which to evaluate it. The question for doctors is at what point to endorse its use. The difficulty for society as a whole is to decide when to pay for it. The debate cannot be adequately informed until cost-effectiveness data are freely available.
Professor, Department of General Practice, University of Adelaide, Royal Adelaide Hospital, Adelaide