Later this year, the UK is to become the first country to introduce a nationwide scheme to vaccinate babies against Meningitis B. In 1999 it was the first to introduce Meningitis C vaccination. What makes the Men B interesting is the way in which it was decided to incorporate this as free vaccine when last year it was deemed too expensive. It provides a case study of how "single payer" systems decide whether to include a treatment in the list of those available under their scheme.
Meningitis B is a horrible disease which primarily affects the very young. Even if caught early enough to avoid death, which still happens in about 1 in 10 of cases, around one in four will be left with some sort of effects. The nature of the disease means that gangrene can set in and amputation of the limbs can be necessary. Other side effects can include deafness and mental impairment. You might, therefore, think it would be an obvious candidate for universal child immunization. The problem of course is all the other calls on limited funds that have to be weighed against the benefits of new drugs and vaccines. To take an extreme example, there are a couple of candidates for an effective vaccine against Ebola, the potentially fatal effects of which we are familiar with. However it would be totally illogical to spend enormous amounts of money on inoculating the whole population of a western country against it. How then do you assess whether a new drug or vaccine is cost effective?
Perhaps the worst method of determining the range of treatments allowed is for legislators to draw up a prioritized list of them, get the projected costs and run down from the top until the money runs out. It is as far from the ideal of treating according to clinical need, within cost restraints, as possible. Legislators will pass value judgements on the patients in their prioritization. So to one they might (in effect) say "you can have your heart bypass" but to another "no gender re-assignment surgery for you" and yet another "we won't be able to afford your gastric band for at least three years". I think you might detect a nonsense in this method. A gastric band may very well mean the patient does not go on to develop the need for a heart bypass - or will at least have a productive life extension so the operation is paid back by their economic contribution.
The UK and other countries use a method of calculating the cost/benefit of a new treatment by reference to a calculation of its QALY, standing for"quality-adjusted life year".
A measure of the state of health of a person or group in which the benefits, in terms of length of life, are adjusted to reflect the quality of life. One QALY is equal to 1 year of life in perfect health.
QALYs are calculated by estimating the years of life remaining for a patient following a particular treatment or intervention and weighting each year with a quality of life score (on a zero to 1 scale). It is often measured in terms of the person's ability to perform the activities of daily life, freedom from pain and mental disturbance.
Although criticized, no other reasonably understandable method over a wide range of new products has to be devised. In very crude terms, it is unlikely that a new treatment will be approved for use within the NHS if the cost of a QALY exceeds £20-30,000. It's important to note that this does not preclude a patient receiving the treatment outside of the free NHS system, nor does it give an absolute right for a patient to demand it; that will depend on the judgement of their clinicians.
The question then becomes who should assess the QALY value for a particular treatment. For most, this is done in England by an arms-length government agency; the National Institute for Health and Care Excellence (abbreviated to NICE for historical reasons). The governments of the other countries in the UK have similar agencies as health is a devolved matter. They tend to use the data from NICE. To take a very recent example, they will issue a recommendation like:
Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said: “Based on the evidence considered, the independent Appraisal Committee concluded that rivaroxaban, in combination with aspirin plus clopidogrel or with aspirin alone, was more effective than aspirin plus clopidogrel or aspirin alone for preventing further cardiovascular deaths and heart attacks in people with acute coronary syndrome and raised cardiac biomarkers.
"The Committee therefore recommended rivaroxaban as a cost-effective use of NHS resources."
Don't worry, I glazed over the details but I hope you get the point that recommendations are not arbitrary but science based. For vaccines, England uses a similar institute, the Joint Committee on Vaccination and Immunisation (JCVI). The HepB vaccine had been assessed in 2013:
The JCVI has concluded that the MenB vaccine did not meet the economic criteria at any level. In other words, introducing the vaccine would not be a good use of limited NHS resources, which could be better spent elsewhere.
So what has made the difference in the intervening couple of years? New data about the vaccine's efficacy led the JCVI to reconsider its recommendations so that last year it issued a new position statement but one which contained an important condition:
The Joint Committee on Vaccination and Immunisation (JCVI) position statement includes a recommendation to offer the Bexsero MenB vaccine to children at 2, 4 and 12 months. This recommendation depends on securing a cost effective price for the Bexsero vaccine.
Here is where the second feature of "single payer" systems comes into play, the ability to negotiate with suppliers in the knowledge that as a virtual monopoly, they have a huge bargaining advantage. Another development was the change of manufacturer to one which had experience of selling into a market where such QALY considerations had to be stuck to. Bexsero was made by a Swiss company called
Novartis. A British multinational, GSK bought the vaccine rights from them and continued negotiations with the English NHS who were acting in concert with the other three NHS organizations. With the price reduced, it means that the whole of the UK will be offering the first vaccinations to 2 month old babies from later this year.
To some extent, this is an experiment as one of the factors that was difficult to assess a value for was the "herd immunity" the vaccine will provide once a cohort of babies has received the course.
Understandably, for parents with a child who died or lost limbs because of MenB, the value of the vaccine would be incalculable. Sadly in a system where competing priorities of different treatments for limited resources have to be taken into account, a equitable method of bringing on new treatments has to be in place. I hope this gives an idea of how such decisions can be reached fairly and without value judgements preventing, or indeed accelerating, the introduction of life saving measures.