Money for something: the importance of cost, benefit and evidence regarding expensive drugs on the NHS

Science and Technology

Following yet another winter crisis in the NHS, it’s hard not to wonder about the distribution of resources in our healthcare system. One component of this is the provision of expensive drugs for various disorders. It is particularly interesting that there are disparities in their usage that sometimes may not match individual benefit; provision in the first place may not even be backed up by sufficient evidence from clinical trials. There are inconsistencies in how much the system is willing to shell out for patients and this exemplifies some of the core difficulties in medical ethics.

‘There are inconsistencies in how much the system is willing to shell out for patients.’

Expensive drug treatments made headlines in 2017, with a childhood case of phenylketonuria (PKU) co-occurring with autism providing one example. PKU in its classical form is a genetic disorder involving loss of function of the enzyme that breaks down the amino acid phenylalanine (Phe), resulting in toxic accumulation of this molecule leading to mental disability and other functional brain problems. Conventionally, newborn screening to make the diagnosis, followed by maintenance of a low Phe diet through restriction of foods such as fish and poultry, can prevent the onset of symptoms. However, in recent years a drug called Kuvan, which acts to increase the function of the enzyme that degrades Phe, has been suggested for use to lower blood Phe concentration.

NHS England refused a request for this drug for the aforementioned patient, despite the existing precedent from a different case. Potential underlying reasons include the expense of the drug, costing between £50,000 and £100,000 a year for an adult patient. With the well-established dietary treatment being significantly cheaper, one can sympathise with the idea of only using this drug in exceptional cases. In addition to this, Kuvan only works in some cases of PKU, and finally a low Phe diet still has to be maintained whilst using it.

On the other hand, the parents have stated that the child doesn’t understand his metabolic condition and can even become violent when not allowed to eat particular foods. Thus, there could be much individual benefit if the drug improved his Phe tolerance so that he could eat a richer variety of foods. A High Court challenge by the parents has since forced NHS England to reconsider its decision.

This case, where there is evidence of cost/benefit analysis, seems to contrast with some approval decisions concerning cancer drugs. A study published in the British Medical Journal in 2017 concerning new cancer drugs approved between 2009 and 2013 by the European Medicines Agency, revealed that for just over half the drug uses studied (as one new cancer drug may have multiple uses), there was a lack of evidence for extension of  life or improvement in the quality of life. This was even the case after a follow-up period awaiting new evidence to support some of these treatments. A contributing factor to this problem is the fact that some studies focus too much on surrogate outcomes – a buzz term in Dr. Ben Goldacre’s books – such as progression-free survival. This means the time period for which a patient’s cancer simply does not worsen following the start of drug treatment, rather than more useful measures such as overall survival rate.

The Cancer Drugs Fund (CDF) is a money pot rolled out in 2011 to enable cancer drugs that are not yet approved by the National Institute for Health and Care Excellence (NICE) to be available to NHS patients in England. To exacerbate matters, the finances of the CDF do not make pleasant reading, with a spend of, for example, £416m occurring in 2014-2015, which was 48% over-budget. In summary, there was essentially a system for expensive drugs to be acquired that lacked sufficient evidence for improving basic outcomes. Thankfully, the unsustainability of this was realised, the CDF was reformed in 2016 and it now involves NICE in the review of newly approved cancer drugs.

Certain philosophical questions concerning how we view especially costly treatments emerge from the two aforementioned examples. Even if there were strong evidence for an increase in life expectancy of a month for a certain cancer drug, how would patients feel about using it? How about two months, or six months? How much faith should be placed on experimental treatments and evidence for efficacy that we don’t yet have, especially if effective alternatives are lacking? Where could a hypothetical utilitarian overlord draw the line for what is cost-effective as well as clinically beneficial, as well as satisfying patient expectations? The answer, of course, is that there isn’t some blanket measure that can be applied to such questions.

However, I believe the take-home message should be that even though these decisions are difficult, there must be transparent, functional systems in place that can at least approve treatments that show efficacy in real-world terms. However, these aren’t informed decisions if we don’t have the information.

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