As part of the development process, any new medicine, treatment, medical device, diagnostic technique, vaccine procedure, or system developed to solve a health problem goes through an evaluation called a health technology assessment (HTA).
An HTA is a process that assesses whether a new treatment is clinically and cost effective. Clinical evidence shows how well the medicine or treatment works. Cost effectiveness shows how well the medicine or treatment works in relation to how much it costs the NHS – whether it represents value for money. It is performed by an independent multidisciplinary group which ensures that patients are receiving the best treatments, and the NHS and government are getting value for money.
Before any drug can be recommended, it first needs to be approved and licensed by the European Medicines Agency (EMA) for use in Europe (this currently includes the United Kingdom). This means it has been rigorously assessed to prove that it is safe and effective.
An HTA is then used to decide whether or not it should be funded on the NHS. To speed up the process, and make drugs available for patients more rapidly, the EMA and HTA assessment are often carried out at the same time.
At the request of the government’s Department of Health and Social Care, HTAs are carried out by the National Institute for Health and Care Excellence (NICE).
NICE is independent from the NHS and government. For a health technology assessment, NICE reviews the clinical and cost effectiveness of new treatments in order to make recommendations to the NHS in England; this is often referred to as ‘NICE guidance’. Wales and Northern Ireland usually follow NICE guidance as well. Scotland follows a different process, run by the Scottish Medicines Consortium (SMC).
The NHS is legally obliged to fund and resource medicines and treatments recommended by NICE's technology appraisals programme. When NICE recommends a treatment 'as an option', the NHS must make sure it is available within 3 months (unless otherwise specified) of its date of publication. This means that, if a patient has a disease or condition and the doctor responsible for their care thinks that the drug or technology is the right treatment, it should be available for use, in line with NICE's recommendations.
There are a number of steps involved in a HTA, but broadly they fit into the following key areas:
- gathering the evidence
- reviewing the evidence
- final recommendations
Scoping: At the scoping stage, NICE develops a detailed framework that specifies what the HTA is aiming to answer. The scoping document defines: the drug or technology that’s being assessed; the disease it’s being assessed for; where in the treatment pathway it’s expected to fit; and what other treatments it should be compared to. NICE sends the draft scoping document to stakeholders, including medical experts and patient representative groups like Lymphoma Action, for comments. The drug company that produces the technology being assessed also has the opportunity to comment.
Gathering evidence: NICE contacts stakeholders to gather evidence from a wide, multidisciplinary group. Patient carer organisations, NHS organisations and professional organisations are all involved. As a patient carer organisation, our role is to provide information on how the disease being assessed impacts the day-to-day lives of patients and carers, what it’s like to have the treatments that are currently available, and the impact that the new treatment might have, based on clinical data and patient experiences.
Reviewing the evidence: NICE holds a committee meeting to review the evidence from the consultation. Stakeholders, including patient experts and patient representative groups such as Lymphoma Action – are invited to attend and contribute to these meetings. Members of the public are allowed to observe the meeting. If necessary, draft recommendations are issued for stakeholder and wider public consultation. This is called an appraisal consultation document (ACD).
Final recommendations: Either following stakeholder/wider public consultation on the draft recommendations, or when a consultation is not needed, NICE produces a final appraisal document (FAD) containing its final recommendation. Stakeholders have an opportunity to appeal the decision in the FAD before NICE publishes its final decision.
In England, HTAs involve NICE, clinical stakeholders (such as medical professionals), patient stakeholders - such as Lymphoma Action – the drug company that makes the new technology and representatives of independent health economic groups called the evidence review group (ERG). NICE appoints the ERG to do an independent economic assessment on their behalf.
Lymphoma Action are invited to represent the patient voice at all HTAs that are related to blood cancer. However, as specialists in lymphoma, we focus only on lymphoma-specific HTAs. We are made aware of these when we receive a scoping document.
NICE writes to stakeholders with a set of questions to respond to and a timetable. These assessments can span a long period of time – it can take up to 18 months (although NICE aims to produce final recommendations within 3 months of a new drug receiving its licence).
We sometimes contact our Medical Advisory Panel (MAP), which is a group of health professionals who specialise in the care of people with lymphoma. This is to assess whether there are any reasons for us not to support an HTA, for example if there is not yet enough clinical trial data to support the new technology. The MAP can also advise on where the new technology would fit into current treatment pathways, if approved.
In order to respond to the questions posed by NICE, we aim to involve people who have been treated with the specific drug being assessed, usually as part of a clinical trial. However, this is not always possible. In this case, we involve people with the specific lymphoma type. We usually invite people to come forward on social media. If we are unable to get enough responses, we might ask the MAP to help us find suitable people. We always try to get as wide an input as possible. We send a questionnaire to people who have volunteered to be involved, and then collate all the responses. We use these to prepare a patient organisation response that we submit to NICE.
We are then invited to take part in the committee meeting. If possible, we nominate a patient representative to attend. This can be a person with lymphoma or the carer of a person with lymphoma. Only patient organisation stakeholders such as Lymphoma Action can put people forward; individuals are not able to put themselves forward to NICE.
An HTA is a very formal process that can feel quite overwhelming. All participants need to sign a confidentiality agreement and receive some very detailed paperwork. We give our representatives clear instructions on what to expect, but NICE also has an excellent patient involvement team who put people at their ease and talk them clearly through the process. They are also very good at keeping everyone updated at every stage of the process, which can stretch out over time.
Typically, a patient representative shares their experience of treatment for lymphoma at the meeting in order to put the assessment in context; it adds a human element and puts the patient in the mind of the group who are otherwise looking at very technical and economic information. The people who have represented us to date have given very positive feedback about the whole experience of an HTA. However, it must be emphasised that the committee is not swayed by emotions, but are making a practical, evidence- and economics-based decision.
We are kept involved throughout, and stakeholders are usually told within a week of the meeting whether NICE is recommending the treatment, or whether further investigations are needed. It is fairly common for a drug not to be recommended at the appraisal consultation document stage, with many going on to need further evidence before the final appraisal document is prepared. We are invited to submit further evidence at that stage. We can appeal a decision, but in reality there has not been the need to so far, as most drugs relating to the treatment of lymphoma have eventually been recommended by NICE.
As soon as the final NICE recommendation has been published, we report it on the news section of our website. We also report on the decisions of the Scottish Medicines Consortium.
If NICE recommends a treatment through its technology appraisals programme, the NHS is legally obliged to fund it and must make sure it is available within 3 months (unless otherwise specified) of its date of publication.
The NHS Constitution states that patients have the right to drugs and treatments that have been recommended by NICE for use in the NHS, if their doctor believes they are clinically appropriate.
At Lymphoma Action we believe it is important to be involved in health technology assessments and we are proud to ensure the patient voice is heard.