Drug development and approval

The treatment of lymphoma is changing all the time. New drugs are constantly being developed and approved for use on the NHS in order to improve outcomes and the overall wellbeing of people affected by lymphoma.

Developing a new drug can take up to 15 years and cost more than £1 billion. For every new drug that makes it on to the treatment plan of patients, many more failed during the rigorous process. Here we outline what that process is.

Step 1: Pre-discovery and drug discovery

To start with, scientists have to understand how lymphoma develops, and what makes a lymphoma cell different to a normal, healthy cell. They can then identify possible targets for a new drug to act on. They could, for example, identify proteins which usually stop your immune system from recognising and destroying the cell. They use this information to create, or search for drugs which act on this protein and stop it from working.

Step 2: Pre-clinical tests Before a drug can be given to people it needs to be thoroughly tested in laboratories. These tests help to see how the drug works, what the potential side effects are, and what dose is the safest to start testing in people. If there are any serious problems then the drug will not be developed any further.

Step 3: Clinical trials

Clinical trials are when the drug is tested on people, and they are organised in phases. Phase 1 or 2 are early phase trials and only involve a small number of people. Their main aim is to see what side effects the drug has, and what is the best dose to use. Phase 3 are later stage trials involving more people and try to establish whether the treatment is effective or not. If a drug is not successful at any phase, or deemed unsafe it will not go on to the next phase. This process usually takes years but can be quicker depending on the treatment or condition. Some drugs may be approved after phase 2 trials for example, if the condition is rare or there is a large unmet need.

Step 4: Licensing approval

If a drug has been successful in clinical trials, the pharmaceutical company that produced it applies for it to be licensed for use. Your doctor can usually only give you treatments that have been officially licensed. In order to obtain the license the company needs to provide evidence of the new drug’s safety, and the benefits it has for people with lymphoma. In most of the UK this application is to the Medicines and Healthcare Products Regulatory Agency (MHRA). In Northern Ireland it is to the European Medicines Agency (EMA) who license drugs throughout the European Union.

What happens next?

When a new drug receives its license it does not mean that it can be prescribed on the NHS. For this to happen a decision has to be made as to whether the treatment is both clinically effective and cost effective. In other words that it works well and is good value for money. These decisions are made by independent organisations which are different depending on which part of the UK you live in.

• The National Institute for Health and Care Excellence (NICE) makes decisions in England. Wales and Northern Ireland usually also follow their guidance.
• The Scottish Medicines Consortium (SMC) makes decisions for patients who live in Scotland.
• The All Wales Medicine Strategy Group (AWMSG) sometimes makes decisions for Wales before NICE makes their decision.

The process of deciding whether a new drug can be funded for use is called a Health Technology Assessment (HTA). This process usually takes months and follows a largely similar structure in all regions of the UK. Initially evidence is gathered from multiple sources including the drug company, independent economists, health care professionals and patient groups like Lymphoma Action. All of this evidence is presented to a committee of independent panel members who use it to determine the clinical and cost effectiveness of the new treatment. They then make a decision whether to approve it for use or not. If a treatment is successfully approved the NHS has three months to make it available to patients.

When Lymphoma Action are submitting evidence we will approach our community for help. This may be by answering a few questions about your experience of lymphoma and your treatment, or as a patient expert who would attend the meeting either in-person or virtually. We do this because putting across the patient voice is so important for the decision making process.

What happens if a treatment is not approved for use on the NHS? There are a few ways you might be able to have treatment even if it is not approved for funding on the NHS.

• The Cancer Drugs Fund - Sometimes there are promising results from clinical trials for new cancer drugs, but not enough evidence to recommend them. In these cases, NICE will make it available for a short time – usually 2 years – while more evidence is collected. This is done via the Cancer Drugs Fund.
• The Early Access to Medicines Scheme - For some treatments, the pharmaceutical company which developed the drug sometimes offers an Early Access Scheme. This allows patients to access a drug before it’s licensed or commissioned and is provided without cost by the pharmaceutical company. This aims to give people with serious or life-threatening conditions early access to promising new drugs.
• Individual funding request - Occasionally, in very unusual circumstances, if your medical team thinks you need a treatment that is licensed but not available on the NHS, they can request for you to have access to it. These are infrequently approved because it has be to for a very rare and exceptional indication.

With thanks to Professor Andy Davies, Senior Lecturer in Oncology, University of Southampton and Dr Wendy Osborne Consultant Haematologist in Newcastle, for reviewing this information.

There has recently been a number of new treatments successfully approved for use in many different types of lymphoma. Lymphoma Action is proud to have played a part in these and will report on some of them, and more detail of our role, in the next issue and on our website.