Expensive medicines: ensuring objective appraisal and equitable access

Editorials 4 In response to requests for the funding of new drugs, reimbursement agencies are re-evaluating some of the methods used in assessing these products. Many trials submitted for the regulatory review of new drugs do not provide adequate data for subsidy decisions. We argue that all involved in bringing medicines to market need to be explicit about the additional information required, decide how these data should be collected and assessed and the methods that should be used to set a fair price for a new drug. In Australia, a formal appraisal of the cost–effectiveness and budget impact of a new medicine precedes any subsidy decision at national level. 1 If a new product is subsidized, the government pays an agreed price to the manufacturer, sometimes with requirements for financial contracts to manage expenditure. 2 Increasingly, patients are asking for early access to new drugs, particularly for treating cancer. In consequence, strategies to subsidize drugs for use under conditions of coverage with evidence development or managed entry are being proposed. A new drug might be approved even if there is no evidence to show that it satisfies the standards typically applied in health technology assessments. However, this approval is often contingent upon additional requirements for subsequent randomized trials or the collection of data on the drug's effectiveness and safety in practice. There is no consensus on the best methods for identifying drugs appropriate for managed entry schemes, for collecting post-approval data or for the use of such data to modify decisions about coverage. In Australia, as in many other countries, several questions need to be answered. Can stakeholders produce a workable framework for managed entry schemes? What can be done to reduce variation in the inputs used for cost–effectiveness models? How can drug or disease registries contribute useful information to inform reimbursement deci-sions? How should registry data be evalu-ated? What can be done to make registry data more representative of the population and what types of post-progression data should be included in trials of targeted cancer therapies? Regulatory agencies, insurers and clinicians also need to be able to determine if a new drug represents good value for money and what to do if an effective drug appears too highly priced for the benefit that it offers. The prices of several recently-introduced drugs – for example aflibercept, ivacaftor and sofosbuvir – have been questioned. The way in which drug prices change …