Restricting the number of drug options that can be used to treat a disease results in unsatisfactory outcomes for patients, prescribers and payers alike.

Abstract

Developing new drugs is extremely expensive often costing upwards of a billion dollars to bring a drug to market¹. This is driven by the studies required to demonstrate the safety of the drug and bears little relation to the cost of manufacture. Demonstration of efficacy generally requires much smaller numbers. The level of safety required is, quite rightly, stringent, and is to give confidence that unexpected side effects are unlikely to be found when the drug is released for use in the general population. If a lower level of safety was accepted, then drugs could be considerably cheaper, but at a risk. However, the study patients are selected, usually for having active disease and few other conditions to complicate response. It is estimated that only about 30% of patients seen in clinic are suitable for any particular study² so we can’t know for sure what will happen when it is available to the other 70%. This is a weakness of the evidence base³.

Drugs are licensed on a risk benefit analysis by the drug regulators eg the MHRA. More recently, driven by the expense of new drugs, in the UK, before they can be used they have been required to show cost effectiveness through a National Institute for Clinical Excellence (NICE) assessment where the cost of the improvement in quality of life needs to hit an acceptable threshold⁴. There is often press and political interest in these approvals. The aim of NICE is to ensure value for money and equality of access to drugs, abolishing postcode prescribing where different geographical areas have different access to drugs. However, the Clinical Commissioning Groups (CCGs) who pay for the drugs, may have their own interpretation of guidelines, and NICE approvals, which apply in their area. An example of this was a restriction on the number of higher cost drugs (HCDs Biologics and JAKi) that could be tried for RA by a substantial number of CCGs in England^5,6.

At a session of the BSR annual meeting in 2019 discussing what Rheumatologists do when the patient in front of them doesn’t fit the guideline for a treatment that they think will be effective, it became apparent that a substantial number of rheumatology services were restricted by their CCGs⁷. Those of us from unrestricted areas were surprised and could not understand the logic for this restriction. The main reason discussed was that these drugs had not been shown to be cost-effective at higher choice points. That was, of course, true but only because the studies had not been done, another weakness of the evidence base³. Lack of evidence is not evidence of lack of effect.