Ask the Nurse: Treatment options

In this month’s blog, we answer some of the questions you ask us about access to treatment.

On the Myeloma Infoline and Ask the Nurse services we regularly get asked questions about treatment options. Patients, carers and family members often want to learn about the treatment options available on the NHS, why some treatments are only available at specific times and if there are other options they should be considering.

In this month’s blog, we answer some of the questions you ask us about access to treatment.

How do new drugs and treatments become available for use through the NHS?

There are three main steps a new drug or treatment needs to go through before it is made available through the NHS.

Clinical trials

Clinical trials are research investigations that test new ways to treat and care for patients. They tell us how safe and effective a new treatment is for patients.


A drug or new combination treatment needs a licence (also known as marketing authorisation) before it can be sold in the UK.

A licence is obtained through a regulatory agency. Drugs for use in England, Scotland and Wales are licensed by the Medicines and Healthcare products Regulatory Agency (MHRA). This follows the departure of the UK from the European Union (Brexit). Previously, licenses could be obtained from either the MHRA or the European Medicines Agency (EMA). The MHRA are able to consider decisions made by the EMA. Drugs can still be authorised for use in Northern Ireland by the EMA or MHRA.

Before granting a licence, the agency will review clinical data, manufacturing details and product information to ensure the new treatment is safe, effective and manufactured to a high standard.

Health Technology Assessment

For a treatment to be made available through the NHS, it needs to be appraised by a health approval body. This is called a Health Technology Assessment. The health approval bodies assess the safety, clinical-effectiveness, and cost-effectiveness of the new treatment. This ensures that the cost of the new treatment is proportionate to the patient benefit.

Who decides whether a treatment is made available through the NHS?

Decisions about access to treatment are made by health approval bodies. Each country in the United Kingdom has its own health approval body.

In England, the National Institute for Health and Care Excellence (NICE) decides which treatments are available on the NHS.

In Scotland, the Scottish Medicines Consortium (SMC) decides which treatments are available on the NHS. Its decisions are separate from decisions made by NICE.

In Wales, the All Wales Medicines Strategy Group (AWMSG) decides which treatments are available on the NHS. In general, they choose to follow NICE decisions.

In Northern Ireland, the Department of Health makes decisions about health and social care. They usually choose to follow NICE decisions.

How do I find out which anti-myeloma treatments are available to me?

Your healthcare team can tell you which treatments are available and whether they are suitable for you. They will also be able to discuss the benefits and risks associated with each option so you can make an informed decision about your treatment.

You can also get in touch with us through the Myeloma Infoline and Ask the Nurse email service.

Can I access treatments that are not currently available through the NHS?

There are several ways that patients can access treatments not approved for use through the NHS.

Private healthcare

Private healthcare gives myeloma patients access to treatments that are licensed for sale in the UK but not approved for use on the NHS (i.e., they have been licensed by a regulatory agency but have not been through the health technology assessment process). Private healthcare can be self-funded or funded through health insurance but is not available at every hospital.

Private treatments are not necessarily better than the treatments available through the NHS, therefore, it is important you speak to your haematologist about the benefits and risks of all the options available to ensure you get the right treatment.

Clinical trials

Patients may be able to access treatments by joining a clinical trial. Trials are not run at every hospital so patients might have to travel to a different hospital to take part in a trial. A trial will only be an option for you if you meet the specific entry requirements for the trial (e.g. age, previous treatments, kidney function).

If this is something you would like to know about you should speak to your healthcare team, they will know if any trials are recruiting nearby and whether they would be suitable for you. You can learn more about clinical trials in our blog post about joining clinical trials.

Early access schemes

Early access schemes (EAMS) are only set up following a request from the drug manufacturer. They give patients early access to life-extending drugs whilst the license application is reviewed. For example, in 2019 isatuximab (Sarclisa®) was available through EAMS until its licence was approved by the EMA. However, they are not common and there are currently no early access schemes for anti-myeloma drugs.

Expanded access schemes (compassionate use schemes)

Expanded access schemes are set up by drug manufacturers to give patients access outside clinical trials to new drugs that are still in development. They are only an option for patients who have no other satisfactory treatment options and who can’t join a clinical trial.

There are currently two expanded access schemes available to myeloma patients in the UK:

If you think this may be an option for you, speak to your haematologist. Requests for expanded access can only be made by your haematologist.

Individual Funding Requests

In special circumstances, a haematologist can make an individual funding request (IFR) application to request access to a treatment not approved for use through the NHS.

Your haematologist can apply for an IFR if your clinical situation is different from other myeloma patients, or you have a higher chance of benefiting from the treatment than other myeloma patients.

These applications take time and are not always successful.

When considering your treatment and options, it’s important you speak to your healthcare team who will be able to recommend the most suitable treatment for your individual circumstances.

If you have any questions about treatments or treatment availability you can get in touch with us through the Myeloma Infoline on 0800 980 3332 (UK) or 1800 937 773 (Ireland) or by using the Ask the Nurse email service.

Best Wishes
The Myeloma UK Information Specialist Team

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