Access to treatments

In this section of the website you will find information about how to access kidney cancer treatments, and, in particular, we explain the NHS policies governing access to cancer drugs. There are different and complex funding policies in place, depending upon whether you live in England, Scotland, Wales, or Northern Ireland. Here we explain how they work.

Please click on the + on the left hand side of each line below to access the information held in the corresponding section.

If your clinician recommends a treatment that is not available routinely via the NHS, you may still be able to access that treatment using another method.

If it is for a drug, your clinician can make an application for the treatment to be funded through the Cancer Drugs Fund (CDF). Please note this route is only available to patients resident in England.

If you live in Northern Ireland, your clinician will need to submit an Individual Funding Request (IFR) to your local Health and Social Care Trust. In Scotland, your clinician will need to submit an Individual Funding Request (IFR) to your Local Health Board. If you live in Wales, your clinician will need to submit an Individual Patient Funding Request (IPFR) to your Local Health Board.

Cancer patients living in England may benefit from the Cancer Drugs Fund (CDF), which was formally launched in April 2011. The CDF was established to enable cancer patients to access drugs that their doctors think will help them, but which are not routinely available within NHS England.

At the end of 2015, NHS England decided that the CDF in its current format was not working efficiently and needed to change. A new appraisal and CDF system was developed in partnership by NHS England, NICE, Public Health England and the Department of Health. Following a 12 week consultation period with key stakeholders, including patient groups and industry, that ended in February 2016, proposals for a new operating model were agreed.

The new system, including a new CDF, replaces the previous fund, which closed on 31 March 2016.

The new CDF operating model came into effect on 29 July 2016. However, some drugs are still being transitioned from the old CDF list to the new operating model. In the meantime, while these drugs are being appraised/reconsidered, they will continue to receive funding from the CDF. Any new licensed drugs will be appraised by NICE using the new operating model.

The main features of the new CDF include:

  • All cancer drugs/indications expecting to receive a marketing authorisation (license) will now be appraised by NICE. NICE will then make one of three recommendations:
    • Yes – the drug should be commissioned for routine use
    • No – the drug should not be commissioned for routine use
    • Maybe – the drug is given a conditional recommendation
  • An early funding option is available for those drugs given a conditional recommendation. This is called interim funding, and is for those drugs given either a draft recommendation for routine commissioning use, or a draft recommendation for use within the CDF
  • The CDF will become a ‘managed access fund’, with clear entry and exit criteria for drugs entering the fund
  • Managed Access Agreements between NHS England and pharmaceutical companies will be put in place. These agreements will set out the terms of entry into the CDF, and the means by which data will be collected to resolve any uncertainty relating to the clinical and cost-effectiveness of a drug
  • All eligible patients will receive CDF drugs, not just the number of patients needed to resolve uncertainty
  • Expenditure control mechanisms have been put in place to reduce the risk of overspend and ensure the fund never needs to close to new entrants
  • A new, joint NHS England/NICE CDF Investment Group will manage and oversee the CDF budget
  • There are similar opportunities for off-label drugs to gain access to CDF funds, if they show clinical promise.

 

Those drugs given a conditional recommendation will be made available to NHS patients and paid for by the CDF.

The drug will remain available within the CDF for up to 2 years while the manufacturer gathers more evidence to show that the medicine works and is fairly priced.  After 2 years, NICE will conduct a review, using a shortened process, to consider the drug for routine commissioning on the NHS. This will either result in the medicine moving out of the CDF and into routine funding, or, if the manufacturer has not been able to demonstrate its case, made available on an exception basis only.

Further details and full guidance on the operation of the CDF can be found here.

What does the new system mean for patients?

  • Faster access to the most promising new treatments
  • Clearer, faster decision-making, meaning less uncertainty about a drug’s availability
  • Similar opportunities for off-label drugs/indications, often used to treat rarer cancers, to obtain CDF funding
  • Access to CDF-funded drugs for all eligible patients
  • Any drugs that were in the CDF as of 31 March 2016 will continue to receive funding while awaiting reconsideration or appraisal by NICE
  • All patients currently receiving treatment via an existing CDF drug will continue to do so, regardless of the outcome of any NICE appraisal or reconsideration, until their clinician decides otherwise.

What does the new system mean for taxpayers?

  • A fixed CDF budget of £340m
  • Expenditure control mechanism to reduce the risk of overspend, meaning that the fund never has to close to potential new entrants
  • The CDF operating as a new, managed access fund designed to resolve any uncertainty around a drug’s clinical and cost effectiveness as swiftly as possible
  • A joint NHS England/NICE CDF Investment Group to oversee budget management
  • Closer working with the pharmaceutical industry to encourage the responsible pricing of cancer drugs, driving stronger value for money in drug expenditure, so that more money can be spent in other areas of cancer care and elsewhere in the NHS

What does the new system mean for industry?

  • A new fast-track system, including an accelerated NICE appraisal process
  • Earlier funding, from the point of marketing authorisation, for the most promising drugs through new interim funding arrangements
  • A managed access approach to rapidly support and resolve any areas of uncertainty for drugs showing clinical promise
  • Each drug/indication looked at on an individual basis with bespoke data collection and commercial access arrangements – no ‘one size fits all’ approach.

Individual Funding Requests (IFRs)

Individual funding requests (IFRs) relating to cancer drugs will no longer be considered via the CDF process.

All IFRs relating to cancer drugs will be considered using NHS England’s single, national IFR system, which is currently undergoing a public consultation closing on 15th January 2017.

More information about the NHS England IFR system, and how to apply, can be found on the NHS England website. See also Julia’s opinion piece about the new NHS England IFR system.

 

KCSN will provide further details as they are made available. You can contact us at any stage for clarification on the process, as we appreciate it can be very confusing.

Only funding for cancer drugs is available via the CDF. If your clinician recommends a treatment that is not a drug, such as a specialised radiotherapy (proton beam therapy, selective internal radiotherapy or stereotactic ablative body radiotherapy) the process is slightly different. Your clinician will need to submit an Individual Funding Request (IFR) to your regional NHS Commissioning Board (CB). Further information and guidance for submitting requests via this route is available on the NHS England website.

We realise this is a distressing and complex process for any cancer patient who has been recommended a treatment that is not routinely available on the NHS. Above is a summary intended to outline the main funding options available. It does not detail every step of the process as this would be too lengthy, but hopefully it will provide you with a brief overview to help you make sense of what can be a bewildering situation.

There are other ways to access a treatment your clinician feels will benefit you, but is not routinely available via the NHS:

 

These are described in the following sections.

Your clinician may suggest a clinical trial as a means of getting you the best treatment available.

Clinical trials are medical research studies involving people. The purpose of clinical trials includes looking for better treatments or better ways to prevent, screen or diagnose a disease such as cancer. If you are asked to take part in a clinical trial, you need to be given enough information to help you make up your mind as to whether or not to participate. Taking part in a clinical trial is completely voluntary.

Our Clinical Trials Hub has a wealth of information about clinical trials, including a searchable clinical trials database to help you find a suitable clinical trial in your region. There are also patient stories detailing clinical trial experiences that may help you to decide whether this is a route you want to take.

The Royal Marsden Hospital also has some very good information about clinical trials on their website.

Alternatively you can search current UK clinical trials at the following links:

The Early Access to Medicines Scheme (EAMS) aims to make promising new medicines available to patients sooner. It was started in March 2015, and it is a process through which patients can access new drugs that are not yet licensed for use in the UK.

EAMS is run by the Medicines and Healthcare Products Regulatory Agency (MHRA), the government organisation that ensures the safety and effectiveness of all medicines and healthcare devices on the market in the UK. The MHRA approves product licenses (marketing authorisation) for all new drugs or new indications for existing drugs, allowing them to enter the UK market.

If a drug has not been licensed, or recommended for use on the NHS, there may be some uncertainties about how safe and effective the drug is, and what side effects it might cause. For EAMS drugs, clinical trials might still be ongoing to gather more information about side effects and how well the drug works, or the drug may be waiting to be approved by the MHRA for a product license.

Clinical research and the licensing process can take many years. In addition, all medicines need to be recommended for use on the NHS before a doctor can prescribe them. The National Institute for Health and Care Excellence (NICE) carries out this process in England, the Scottish Medicines Consortium (SMC) in Scotland, and the All Wales Medicine Strategy Group (AWMSG) in Wales. These organisations decide whether the drug works better than available treatments and represents value for money for the NHS, and can take many months to complete.

During the time that a new, promising drug is undergoing approval for a product license or recommendation for use on the NHS, it is not available to patients, unless it can be accessed through the Cancer Drugs Fund (in England only). EAMS provides patients access to these drugs while the licensing/recommendation processes are ongoing.

EAMS can also be used to give doctors information to support a decision to prescribe a drug off-label i.e. for a condition, age group or dose that it is not licensed for.

EAMS can only be used for drugs that may benefit people with a life threatening or seriously debilitating condition when there is no other treatment available to them.

How are decisions made about which drugs can enter EAMS?

The new drug needs to have been through phase 1 and 2 clinical trials, and been proven to be safe and effective before it can enter EAMS. It may also have been through phase 3 trials too, in some cases.

1. Promising innovative medicine (PIM) designation

The manufacturer then applies to the MHRA for what is known as a promising innovative medicine (PIM) designation. To be awarded a PIM for a new drug, the manufacturer needs to submit the results from all the research carried out so far on the drug to the MHRA. A PIM is an early signal that the drug may be a possible choice for EAMS, and might be able to help people who have no other treatment options.

2. EAMS scientific opinion

If the MHRA decides that doctors can prescribe the drug, the next stage is a scientific opinion. The manufacturer must have a PIM before they can apply for an EAMS scientific opinion. The scientific opinion describes the risks and benefits of the drug based on data gathered from the patients who will benefit from the medicine. The opinion supports the prescriber and patient to make a decision on whether to use the medicine before its licence is approved. A positive scientific opinion lasts for a year. The company can then apply to renew it.

3. Public assessment report (PAR)

Following a positive EAMS scientific opinion, the MHRA produces a public assessment report (PAR). This has information about

  • How the product is used and how it works
  • Summary of key clinical studies/trials (research)
  • Which conditions it can be used to treat, such as a particular type and stage of cancer
  • Who can give the medicine
  • The reason for the positive EAMS decision by the MHRA
  • Any uncertainties about the use of the medicine for this condition
  • Possible benefits and side effects
  • Information about any on-going studies/clinical trials
  • How the MHRA will monitor and manage any risks

The PAR is used by the doctor and the patient to decide if the treatment will benefit the patient.

EAMS safety measures

Because the drugs in EAMS are all new drugs, doctors are still investigating how effective the drugs are and what side effects they cause. There may be some side effects that are not yet known about and haven’t been seen in the clinical trials conducted so far.

Doctors need to monitor side effects and how well the drug works while a drug is in EAMS. The manufacturer needs to provide regular updates to the MHRA as part of their agreement to supply the medicine. These updates are given every 3 months, at least.

1. Reporting side effects

To enable the manufacturer to provide these updates, you need to keep your doctor informed of any side effects and/or test results. If you have any serious side effects, you need to let your doctor, nurse or pharmacist know as soon as they happen. Your doctor needs to report all serious side effects to the manufacturer within 24 hours of them happening. All other side effects and test results need to be reported within 5 days.

2. Patient alert card

The patient alert card holds information about the drug you are taking and its known side effects. It also has the contact details for your doctor and specialist nurse. This should be carried with you at all times.

Which drugs are available on EAMS

EAMS started in early 2015 and the MHRA has only looked at a few drugs. The UK government website contains details of which drugs are available on the EAMS.

To get access to a drug that is in the EAMS, please talk to your doctor to see if the drug is suitable for your situation. If your doctor agrees that you might benefit from the EAMS drug, he/she will make an application to the Department of Health (DH) in your local area.

If you are not able to access an EAMS drug, it can be very disappointing. Please talk to your doctor about how you are feeling and why you are not able to have the drug. You could also talk to your doctor about taking part in a clinical trial for a new kidney cancer drug: please see our KCSN Clinical Trials Database for lists of kidney cancer clinical trials, and our Clinical Trials Hub for information about taking part in a clinical trial, including patient stories, videos and clinical trial regulations.

Please contact us for more information about EAMS.

Compassionate use/expanded access programmes allow the use of unauthorised/unlicensed medicines for patients for which there are no licensed drugs available, and who are unable to enter a clinical trial. They are intended to allow patients to access new treatment options that are still under development. Your doctor will be able to advise you more specifically if this is an option available to you. Access to medicines via compassionate use programmes usually ends once the medicine receives a product licence.

A pharmaceutical company may choose to run an expanded access programme to allow early access to their medicine, for example, for patients who have been treated with the medicine during a clinical trial and wish to continue treatment. In an expanded access programme, patients are usually followed up in the same way as patients in a clinical trial.

In some cases doctors may approach a manufacturer directly to request the supply of a new medicine that does not have a UK product licence, to be used for a patient under their direct responsibility. This is often called supply on a “named patient basis”. Named patient supplies are more informal arrangements between the pharmaceutical company and the prescriber.

If you are unable to access the treatment your doctor wishes to prescribe by any of the means detailed above you may wish to consider ‘co-payment’ or ‘topping up’.

Co-payment involves paying for a treatment and any costs related to giving that treatment whilst still continuing with your NHS treatment you would otherwise receive. If this is something you wish to consider you should discuss this option in detail with your clinician.

Please do contact us for further assistance if you have been recommended a treatment that you are told is not routinely available via the NHS.

A NICE technology appraisal recommends how new and existing treatments can be used within the NHS. The technology being appraised can be:

  • A new medicine
  • A medical device, such as a hearing aid or an inhaler
  • Diagnostic techniques, such as tests used to identify diseases
  • Surgical procedures, such as repairing hernias
  • Health promotion activities, such as ways of helping people with diabetes manage their condition.

 

NICE base their recommendations on a review of clinical and economic evidence.

  • Clinical evidence shows how well the medicine or treatment works
  • Economic evidence shows how well the medicine or treatment works in relation to how much it costs the NHS, in other words, does it represent value for money?

 

A health technology appraisal (HTA) can be for single or multiple technologies:

  • Single technology appraisals (STA) cover a single technology for a single indication
  • Multiple technology appraisals (MTA) normally cover more than one technology, or one technology for more than one indication.

 

The following glossary compiled by Health Technology Assessment International (HTAi), an international society for the promotion of health technology assessment (HTA), defines some of the common terms that you may come across when reading about HTA: HTAi Consumer and Patient Glossary

NICE technology appraisal process

This is an overview of the NICE technology appraisal process. For more information, please visit the NICE technology appraisal website.

  1. Provisional appraisal topics chosen
    The Department of Health (DH) produces a list of treatments or technologies that they would like NICE to appraise.
  2. Consultees and commentators identified
    Consultees are organisations or groups that are consulted about the technology under appraisal, such as the manufacturer of the treatment/technology, patient groups, organisations representing health professionals, the Department of Health, the Welsh Government, NHS England, and clinical commissioning groups (CCGs). Consultees can make a submission and participate in the appraisal consultation. All non-company consultees can nominate clinical experts and/or patient experts to present their personal views to the Appraisal Committee. Commentator organisations include the manufacturers of comparator technologies, Healthcare Improvement Scotland, any relevant National Collaborating Centres, research groups working in the area, and others. Commentators can participate in the appraisal consultation, but are not asked to make a formal submission to the appraisal. Non-company commentator organisations can nominate clinical experts and patient experts to present their personal views to the Appraisal Committee.
  3. Scope prepared
    Once the consultees and commentators have been identified, NICE works with the Department of Health to develop a scope. which defines the disease, the patients and the technologies covered by the appraisal, and the questions it aims to answer. Consultees and commentators are asked to comment on the draft scope.
  4. Appraisal topics referred
    The Department of Health then refers the draft scope as a technology appraisal topic to NICE.
  5. Evidence submitted
    The manufacturer is invited to submit a report containing all the relevant information (evidence), both published and unpublished, for an appraisal. All consultees are also invited to submit a statement on the potential clinical and cost effectiveness of the technology under consideration. In the case of an MTA, NICE invites consultees and commentators to provide a submission.
  6. Evidence Review Group (ERG) or assessment report prepared
    After the submission has been made by the manufacturer, NICE commissions an independent academic centre to form an Evidence Review Group (ERG) to review the evidence submission and prepare a report. In the case of an MTA, the ERG reviews published evidence on the technologies and prepares an assessment report. Consultees and commentators are invited to comment on the report.
  7. Evaluation report prepared
    NICE then prepares an evaluation report, including all of the evidence that will be looked at by the Appraisal Committee. This evidence includes:
    • The ERG or assessment report, and any comments received on it
    • Written submissions from consultees
    • Personal statements from patient experts and clinical experts.
  8. Appraisal committee
    NICE appoints an independent advisory committee to consider the evaluation report and hear evidence from the clinical experts, patient experts, and NHS commissioning experts. The appraisal committee meeting is held in public.
  9. Appraisal consultation document (ACD) produced
    Following the appraisal committee meeting, the committee produces an appraisal consultation document (ACD) in which it makes provisional recommendations about the use of the technology or treatment under consideration. Consultees and commentators have four weeks to comment on the ACD, which is also made available on the NICE website for health professionals and members of the public to read and provide comment. An ACD will be produced regardless of the nature of the recommendations from the appraisal committee.
  10. Final appraisal determination (FAD) produced
    The appraisal committee considers the comments received on the ACD, then produces a Final Appraisal Determination (FAD) in which NICE makes its final recommendations on how the technology should be used in NHS England. Consultees can appeal against the final recommendations in the FAD.
  11. Guidance issued
    If there are no appeals, or an appeal is not upheld, the final recommendations are issued as NICE guidance.

 

Kidney cancer technology appraisal guidance

The following technology appraisal guidance has been issued by NICE for kidney cancer treatment:

 

The following technology appraisals are currently in development:

 

Some kidney cancer treatments have not been approved by NICE and are not routinely available in NHS England, however. These are:

 

Further information about NICE guidance, including links to the relevant guidance documents, can be found on the NICE Kidney Cancer Guidance page on our website.

 

Update on NICE nivolumab single technology appraisal (STA)

October 2016

Nivolumab (Opdivo) has been recommended by the National Institute for Health and Care Excellence (NICE) for use by the NHS in England and Wales for people with advanced renal cell carcinoma (RCC) that has been previously treated.

This is fantastic news, and heralds a new era of treatment for kidney cancerpatients. The recommendation of nivolumab increases the arsenal of drugs available to oncologists, enabling them to select the most effective drug for individual patients when first-line treatment with targeted therapies fails. This decision also gives hope to many people with advanced kidney cancer, who have come to the end of the line with respect to treatment options routinely funded by the NHS.

Nivolumab is the first immunotherapy to be recommended for kidney cancer, and is a new and innovative class of drug (a PD-1 checkpoint inhibitor) with proven long-term benefits in about one third of cases. Nivolumab has been proven to be a clinically effective and well-tolerated drug, and was designated a breakthrough therapy by the US Food and Drug Administration (FDA) for the treatment of advanced or metastatic RCC. As a breakthrough therapy, nivolumab has been fast tracked for approval in a number of countries, and was previously approved for use under the Medicines and Healthcare products Regulatory Agency (MHRA) Early Access to Medicines Scheme (EAMS) in the UK. Nivolumab is already being used by the NHS for the treatment of advanced melanoma patients.

As a breakthrough therapy previously available through EAMS, NHS England organisations have been instructed to implement this recommendation within 30 days of its publication, rather than the usual 90 days for implementation (NHS Wales organisations are given 3 months). However, nivolumab will be available almost immediately, since it will receive interim funding from the Cancer Drugs Fund until the guidelines are implemented.

KCSN would like to thank all those patients who contributed to to the nivolumab appraisal, our original statement, and our response to the Appraisal Consultation Document (ACD), especially the patient experts who attended the appraisal meeting to represent the views and opinions of the wider kidney cancer community, Alison Fielding and Jon Birchall – without your dedication and perseverance on behalf of the kidney cancer community, the decision might not have been so positive – a BIG thank you!

We would also like to thank all the clinicians who fought so hard for access to this innovative new treatment on behalf of their patients. Special thanks goes out to the oncologists who submitted expert clinical opinions at the NICE appraisal meeting, and who highlighted the value of nivolumab in improving survival for people with advanced kidney cancer – we are STRONGER TOGETHER!

Read the NICE press release here and media coverage here.

 

Second committee meeting: 7th September 2016

KCSN attended the second committee meeting for the NICE nivolumab single technology appraisal (STA) in London today. This meeting was arranged to discuss the responses received from the publication of the nivolumab for renal cell carcinoma Appraisal Consultation Document (ACD). KCSN submitted a response to the ACD on behalf of kidney cancer patients in the UK.
As always, the meeting was very difficult to gauge what the decision will be regarding nivolumab. The manufacturer, Bristol-Myers Squibb (BMS), have submitted further evidence regarding long-term survival with nivolumab from their phase I/II programme (CheckMate 003 and 010 clinical trials) and 2-year survival data from CheckMate 025, and these data have been included in the health economic models for the calculation of the incremental cost effectiveness ratio (ICER). They also presented further evidence to prove that nivolumab is equally effective in the second- and third-line setting. The committee seemed happy to accept these data and agreed with most of BMS’s economic modelling. However, there was some discussion regarding the overall survival model scenarios (no long-term survival ‘tail’ versus survival tail of up to 5 years), which the committee were not prepared to accept and which may impact the ICER (e.g. the use of a different model suggested by the committee, which may increase the ICER by £30,000!).
There was also some discussion about the cost of missed doses/delayed doses to the NHS and how this impacts the ICER calculations. BMS emphasised that the dose will not be wasted if the patient doesn’t turn up for their appointment (it is only prepared ready for infusion once the patient has been through some pre-dose assessments), and if the dose is delayed more than 7 days, then it is considered a missed dose and would not be made up later in the cycle. Therefore, the cost of missed/delayed doses would be negligible.
The committee accepted the clinical experts’ and BMS’s views regarding the ongoing benefit of nivolumab, even after disease progression and stopping treatment. This modelling was included in all analyses.
However, they did not give anything away as far as the ICER was concerned and the discussions surrounding the patient access scheme (PAS) were commercial in confidence, so I don’t know what was agreed! We get the feeling that the meeting was a formality; they have already made up their minds and we hope that nivolumab will be funded by the new Cancer Drugs Fund (CDF) while further survival data is collected from CheckMate 025 and the Early Access to Medicines Scheme (EAMS).

 

 

We’ll keep you posted with more news when we hear it! The Final Appraisal Document (FAD) is expected to be published next month.

 

First committee meeting: 8th June 2016

prescription_drugsA small group of us from KCSN went to the NICE nivolumab single technology appraisal (STA) meeting on Wednesday 8th June 2016. KCSN recommended two members of our patient support community as patient experts to contribute to the meeting, while the rest of us were public attendees and unable to voice our opinions (unfortunately!). We were the only kidney cancer patient group/charity to make a formal submission or to nominate expert patients to give evidence to the appraisal committee.
The chair opened the meeting by reminding everyone of the aims of an STA as being to evaluate a new drug to see if it is more effective than treatment currently available, and to determine whether the new drug offers value for money to the NHS.
The clinical experts were Dr James Larkin, Consultant Medical Oncologist from the Royal Marsden Hospital, London, and Dr Paul Nathan, Consultant Medical Oncologist from Mount Vernon Hospital, Middlesex and chair of the NCRI Renal and Bladder Clinical Studies Group. Both clinical experts did an excellent job of describing how they envisaged nivolumab to be used in clinical practice and commenting on the clinical data presented during the meeting.
The patient experts were Alison Fielding and Jon Birchall, both stage 4 kidney cancer patients. Jon is taking nivolumab via EAMS and Alison has been on pazopanib for 15 months. Both patient experts were great advocates for second line treatment with nivolumab, and presented their arguments professionally and emotively.
The scope of the single technology appraisal (STA) was nivolumab for patients previously treated for advanced or metastatic renal cell carcinoma. Comparator treatments were axitinib, everolimus (available via the CDF), and best supportive care. Outcomes were overall survival, progression-free survival, response rate, adverse effects and health-related quality of life.
The main discussion points were as follows:
  • The bulk of the evidence for nivolumab came from the CheckMate-025 trial of nivolumab versus everolimus in the second-line, and a network meta-analysis that compared nivolumab with axitinib. There were differences in the trial populations, and there were more poor performing patients in the meta-analysis than in CheckMate-025. The committee questioned whether the trial populations were comparable, since the CheckMate-025 patients had an overall better prognosis than the meta-analysis patients, thereby potentially underestimating the effectiveness of axitinib. Which trial population is more generalisable to NHS patients? The clinical experts both agreed that in clinical practice only a small proportion of poor performing patients would qualify for second/third- line treatment, and therefore the CheckMate-025 population was closer to reality.
  • The network meta-analysis was carried out because there are no randomised clinical trials to compare nivolumab and axitinib or axitinib and everolimus. The clinical experts stated that in clinical practice axitinib and everolimus were similar, although clinical trials show that patients do better on second line TKIs.
  • In the CheckMate-025 trial, nivolumab reduced the risk of death compared to everolimus; however, the survival curves for nivolumab and everolimus appeared to converge at around 28 months, meaning there was no difference between everolimus and nivolumab. The clinical experts disagreed with this, and made the committee aware of new phaseI/II data presented at ASCO last week showing that the survival curves for nivolumab plateaued (levelled off) and around 1/3 of patients survived for at least 5 years. These data had been presented to the NICE committee, but not included in the health economic modelling.
  • The patient experts were asked about an intravenous injection versus daily tablets, and the impact on quality of life. Both agreed that because nivolumab was well tolerated and appeared to provide benefit to patients, the impact of an intravenous injection on quality of life was acceptable.
  • The NICE committee questioned some of the methodology used by the company for the cost effectiveness analyses, and whether the assumptions used in the analyses reflected NHS patients, and whether the data represented second or third line use of nivolumab. The clinical experts suggested that if they had the choice between second and third line, they would give nivolumab sooner to enable a greater duration of action.
  • The cost effectiveness calculations were done using the list price of nivolumab and did not include any discounts/patient access schemes negotiated with the company (this was conducted in private after the public meeting). Because of the NICE committee’s disagreement with the cost effectiveness methodology used by the company, they calculated a different incremental cost effectiveness ratio (ICER). The company’s ICER was £42,417 and the committee’s ICER was £74,132. Both estimates are higher than the NICE threshold of £30,000.
Unfortunately, we were not party to the confidential discussions between NICE and the company about discounts and patient access schemes, and we don’t know the outcome of these discussions yet. Although the ICER presented by the company was high, the positives that favour nivolumab are:
  • It is the first checkpoint inhibitor immunotherapy to gain marketing authorisation for advanced RCC – it is an innovative, new drug and we do not yet know the long-term benefits of this class of drug in RCC patients.
  • It is better tolerated than the drugs we currently have for RCC – quality of life is improved, and for some patients this has been life-changing and people have been able to live normal lives again.
  • There is some evidence, although small, that the drug can prolong the lives of RCC patients for at least 5 years – the company need time to collect more evidence to prove this effect.
We will keep you informed of their decision, which is expected next month.
Huge thanks to Alison and Jon for doing such a great job representing kidney cancer patients at the meeting, and to the clinicians for ensuring the committee were aware of the latest data for nivolumab and for putting forward a strong case for NICE approval.

Commissioning through Evaluation (CtE) is a programme run by NHS England, which enables a limited number of patients to access treatments that are not funded by the NHS. Although not funded, these services/treatments have all been identified by clinicians and patient representatives as showing significant ‘promise’ as potential treatment options for the future. During a CtE programme, new clinical data (evidence) and patient experiences (real world data) are collected within a formal evaluation programme.

CtE programmes are particularly relevant to specialised services, and services and treatments for smaller numbers of patients; often there is less evidence available in these areas to support the development of a full commissioning policy.

There are two main phases to the programme:

  • Phase 1 – NICE helps to agree the total number of patients needed to support the data analysis. These patients are recruited at a few selected centres across England. The CtE programme will be stopped as soon as enough patients have been recruited to complete the analysis of the data. In other words, if enough patients are recruited to support the analysis, the scheme may be stopped sooner than expected. Also, the number of patients may be increased, if, for example, not enough patients are recruited in a particular group.
  • Phase 2 – this is the analysis phase, which varies in length depending on the types of evaluations that are done and the length of follow-up that is required. For example, to assess whether treatment benefits are maintained for 12 or 24 months, or to assess overall survival for a new cancer treatment.

 

NICE advises NHS England on whether a follow-up period is needed, the number of patients required for each assessment during the follow-up period, and the length of the follow-up. This ensures that the data produced by the CtE programme meet requirements to inform future commissioning policy decisions.

During the analysis of the data, all patients being treated in a CtE programme will receive appropriate follow-up care; however, no new patients will be funded by NHS England for the service/treatment being investigated and the service/treatment will not be routinely available within the NHS. NHS England’s published policy for the service/treatment concerned will be followed.

Once the analysis is complete and the CtE report is available, or if new data from clinical trials comes to light, the published policy for the service/treatment concerned will be reviewed and it will be decided whether or not to make the service/treatment available within NHS England.

The following CtE programmes are currently available. For more information about these programmes, including the centres that are participating and recruiting patients, please visit the NHS Commissioning through Evaluation page:

  1. Selective Dorsal Rhizotomy (SDR): This is a procedure that aims to increase mobility in children with cerebral palsy, by selectively cutting nerves in the spine, to improve spasticity (muscle stiffness).
  2. Selective Internal Radiation Therapy (SIRT): This is a form of radiotherapy using radioactive beads to treat cancerous tumours in the liver.
  3. Percutaneous Mitral Valve Leaflet Repair (Mitraclip): This procedure is used to treat mitral regurgitation in patients with heart failure.
  4. Patent Foramen Ovale (PFO) Closure: This procedure is used to prevent recurrent stroke.
  5. Left Atrial Appendage Occlusion (LAAO): This procedure is used in the prevention of stroke.
  6. Stereotactic Ablative Radiotherapy (SABR): SABR is a more precise delivery of radiotherapy, applying high doses of radiation whilst causing less damage to surrounding healthy tissues.

 

Selective Internal Radiation Therapy (SIRT) and Stereotactic Ablative Radiotherapy (SABR) might be of interest to people with kidney cancer.

You should discuss with your clinician if Commissioning through Evaluation (CtE) is an option that is relevant to your situation.

The following table lists the status of individual kidney cancer drugs in England, Wales, Scotland, and Northern Ireland, and whether they are available within the NHS in each individual country:

Download a pdf of the table here: Access to KC Treatments in the UK 2017-09-20

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