Guidance

Off-label vaccines: a guide for parents - text version

Updated 13 March 2023

Applies to England

Like all medicines, vaccines have to have a license or authorisation before they can be given to members of the public. Sometimes, however, healthcare provider may tell you that the vaccine that your child is being offered is ‘off-label’. This leaflet explains what this term means and why it’s important that you understand why the vaccine is still recommended.

Licensed vaccines

A vaccine with a license means that it has been approved for use in certain patients to protect against certain diseases. So a vaccine can be licensed for use in babies, but not older children, or in adults but not children.

There are many possible scenarios in which a vaccine could be used but the trials done before licensing can’t study every possibility.

This means that vaccines are invariably given licences that restrict their use in certain ways – it would not be ethical to delay granting a license whilst every possible scenario was studied because in the meanwhile many people would be denied the benefits of immediate protection. When a vaccine is used in a way other than that described in its license, this is called ‘off label’.

How vaccines get their authorisation

All vaccines used in the UK are authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) or the European Medicines Agency (EMA). Vaccines will only be submitted to the MHRA after they have been trialled by the manufacturers on their target audience (which can be children or adults) and fully tested to see that they are:

• acceptably safe – they do not cause other illnesses or make existing illnesses worse and that any side effects produced are generally tolerable like pain at the injection site or headaches, nausea and rashes

• effective – they offer good protection against the disease they are designed to protect against

• manufactured to a high standard of quality

This exhaustive testing process from the first batch of a vaccine being made in a laboratory to its use in the general population can take more than 10 years.

Only when this information has been reviewed and accepted by the MHRA or EMA, will the vaccine be given a license and be produced and promoted by the manufacturers for general use. Amongst other things, the license specifies who can receive the vaccine, how many doses are required, what side effects may occur and how the vaccine should be handled and stored.

The vaccine is still safe and effective. So these ‘off-label’ vaccines aren’t unlicensed they’re licensed for different patients, conditions or schedules.

Reasons your child may be offered an ‘off-label’ vaccine

All vaccines currently used in the UK are licensed, so being described as off-label doesn’t mean a vaccine is unlicensed. It just means that it’s being used in people for whom the license wasn’t originally given in teenagers instead of adults, for example, or in a slightly different way, such as at a different schedule. They are just as safe and effective.

Sometimes, after the MHRA has licensed a vaccine, circumstances change such as an outbreak of a disease in a different age group, or new information comes to light for example from independent studies on the length of protection from the vaccine. Decisions may need to be made to use the vaccine outside the strict terms of the license, so that as many people at risk can be vaccinated as quickly as possible.

These decisions are normally taken by the Joint Committee on Vaccination and Immunisation (JCVI) which is made up of many independent experts on all aspects of vaccination in the UK. Usually, they involve recommending that a vaccine that is licensed for one purpose or in one group of patients can be used off-label for another purpose or in another group of patients. Once these decisions are made, GPs and other suitably qualified professionals can offer the vaccine ‘off-label’.

Examples of successful off-label vaccine use

Whooping cough vaccine for pregnant women

One example is the use of a whooping cough vaccine in pregnant women. In the years up to 2012, the UK experienced an increase in the number of whooping cough cases many in babies too young to be vaccinated themselves. An urgent decision on how best to prevent deaths and serious illness in these babies was required.

In 2012, JCVI agreed that the best way to protect these very young babies was by vaccinating pregnant women with pertussis (whooping cough) vaccine. This would ensure that babies were born with high levels of antibody from their mothers.

There were 2 vaccines suitable for boosting whooping cough protection in adults but neither had been tested on pregnant women because pregnant women are excluded from most clinical trials. However, data on the extensive use of vaccines with similar components was available and suggested that the vaccine would be safe and effective. One of the vaccines was therefore offered ‘off-label’ to pregnant women and around 70% of mothers now receive the vaccine.

This vaccine programme quickly resulted in a significant fall in the number of whooping cough cases and deaths in babies and detailed analysis has shown that the vaccine is safe for the mother and the pregnancy. Based on the success of this vaccination programme, and particularly the important data on safety and effectiveness in pregnant women generated in the UK, regulators should now be able to determine if use in pregnancy will be ‘within label’ in the future.

Meningococcal B (MenB) vaccine for infants

In 2015, the MenB vaccine was introduced into the UK routine childhood schedule to protect babies against meningococcal B disease which is a major cause of meningitis and blood poisoning. The vaccine is licensed as a 3 dose schedule in babies aged 2 months, followed by a booster dose at around one year of age.

However, after expert consideration of all the available evidence, the JCVI recommended that 2 doses (at 2 months and at 4 months) with a booster (at 12 months) would protect babies was just as well as the licensed schedule. So one of the infant doses was dropped even though the license stated that 4 doses should be given. The schedule recommended by the JCVI is working well as around 250 cases of MenB disease were prevented in the first 3 years of the programme.

Human Papillomavirus (HPV) vaccine for teenagers

A further example is the HPV vaccine which is given to 12 to 13 year-olds at school to protect them against cancers caused by infection with the Human Papillomavirus.

Based on studies in young adult women, the vaccine was originally given in 3 doses within a 6 month period. Subsequent studies, however, suggested that, in younger individuals, 2 doses, given 6 months apart, are as effective as the 3 dose course in young adults.

Based on the knowledge that younger individuals respond very well to vaccines and to make the programme work better with the school terms, JCVI recommended that the second dose of the HPV vaccine can be given between 6 and 24 months after the first, even though giving the second dose more than 6 months after the first dose is considered to be ‘off-label’.

‘Off-label’ can have several meanings. In these examples, and in all other cases, the decision to use vaccines ‘off-label’ has been taken in the best interests of those recommended the vaccine and the wider public.

Concerns about ‘off-label’ vaccine use

‘Off-label’ may be the only way that the vaccine is available on the NHS and refusal may delay your child becoming protected.

JCVI and your healthcare professional will only recommend that your child has an off-label vaccine for a very good reason so you should bear this in mind when making your decision.

More information

The Medicines and Healthcare products Regulatory Agency (MHRA) has comprehensive information on this topic.