Paper of the Week: 20th December 2019

This week’s blog is brought to you by: Dr Tim Wilson

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Full reference and title from the journal:

Funding orphan medicinal products beyond price: sustaining an ecosystem , Author- De Sola-Morales, Oriol, The European Journal of Health Economics (2019) 20:1283–1286

Web link to paper:

Authors conclusion:

“it is not the individual price that matters, but whether the overall budget impact is perceived as acceptable and that manufacturers have an accept- able profit.”

3V bottom line:

Ideally, a decision whether a treatment or intervention should be used in a health system is largely be driven by value and values. In the case of effective but expensive treatments for rare conditions, societal values are the most important driving force. We need to apply the same thought process to all interventions.

3V – Implications for value:

In this editorial, de Oriol-Morales summarises three big issues regarding rare but highly effective treatments, so called orphan drugs. These are pharmaceutical products which will be used for a small number of people, but because of the high development and marketing costs, the cost per individual is very high (in economic terms, the QALY or DALY is much higher than interventions for commoner conditions). If we were to take a strictly utilitarian approach, then we would not fund these treatments; but society is not like that.

First, the author reminds us that these medicines are often “life-saving”, or at least have a major impact, mostly on children. Second, the author points out, we look for some intergenerational fairness. Adults are willing to give up resources for the benefits of the next generation.

Thus, as a society we are willing to fund orphan medicines, despite their price. As Dostoevsky put it in The Brothers Karamazov

“Imagine that you are creating a fabric of human destiny with the object of making men happy in the end, giving them peace and rest at last. Imagine that you are doing this but that it is essential and inevitable to torture to death only one tiny creature…in order to found that edifice on its unavenged tears. Would you consent to be the architect on those conditions? Tell me. Tell the truth.”

Thus, we see how “values” can drive our decisions to about where to invest resources; values drive value. Alas, we do not see the same in many, or any other circumstances in healthcare.

This is why cost-effectiveness is insufficient as means of increasing value in healthcare. Once a procedure is proven effective, and it has been deemed to be cost-effective, the amount it is used is irrelevant. But demand for these highly cost-effective interventions has grown enormously:

  • Testing by ~350% between 2000-2016
  • PCI Stenting by ~350% between 2000-2013
  • Hip replacements by ~200% between 2005-2017
  • Statins by ~600% between 2000-2013
  • Novel oral anticoagulants by ~800% 2014-2019

Each of these is individually cost-effective. Where the evidence exists, in all these procedures there are high levels of inequity-people living in the most deprived communities are less likely to have access to them.

Sadly, we never ask as a society ‘how much” of our finite resources do we want to invest in these procedures, what will we have to not do as a consequence and do we want to ensure we are fair in how we provide them?



Relevance to NHS Constitution Principles:

Principle 1: The NHS provides a comprehensive Service, available to all.

Principle 2: Access to NHS services is based on clinical need, not an individual’s ability to pay.