CLL Support Association responds to NHS consultation on the proposals for the new Cancer Drugs Fund operating model

Image: 

‘The aim of the new CDF is to help patients receive new treatments with genuine promise, while real world evidence is collected for up to two years on how well they work in practice. This will then help determine whether the treatment should be accepted for routine use in the NHS in the future.

The original CDF was established in 2011 to fund cancer drugs in England that are not currently approved by NICE.

Overview

Since its inception in 2010, the Cancer Drugs Fund (CDF) has provided access to treatment for patients whose individual circumstances suggest that they will benefit from drugs that have not been adopted for routine use in the NHS. This includes drugs which have not been recommended by the National Institute of Health and Care Excellence (NICE), are for rare cancer licensed drug indications not selected for NICE appraisal, or are planned to be used off-label.

https://www.engage.england.nhs.uk/consultation/cdf-consultation

The budget for the CDF was initially set at £200m; however, this has been increased twice, most recently to £340m for 2015/16.  However, and as recognised by the Cancer Taskforce in their recent report, while the CDF has helped to unlock access to new treatments for a large number of patients, its implementation under the current model does not enable access to innovative drugs in a smart or sustainable way.   England is currently allocating an increasing share of the cancer budget to treatments that are less cost-effective, towards the end of life, and the impacts of this are being felt further down the cancer pathway.  The arrangements for the current CDF are due to end in March 2016.  In light of this, and the escalating overspend under current arrangements, the NHS England Board requested that proposals be developed for a new CDF operating model, to be introduced from April 2016, following a public consultation.’

The NHS proposal for consultation outlines a new system, planned to be fully integrated into the NICE appraisal process, where the CDF will become a transitional fund – with clearer criteria for entry and exit.  

The purpose of the review is to determine the future of the Cancer Drugs Fund with an aim to ensure patient access to new life-saving drugs. Under the proposed model, NICE will award all new cancer drugs with a “yes” (in which they are commissioned by NICE), a “maybe” (where they will be CDF funded for a time limited period) or a definite “no” (where they will not be funded) decision within 90 days of market authorisation.

Under the proposals put forward by NICE the CDF will provide interim funding for new treatments that demonstrate “genuine promise” (but where the data is uncertain) whilst evidence is collected on how well the drug works in real world situations. Each treatment will be funded for no more than two years whilst the “real world evidence” is being collated and it will then be determined whether the treatment should be accepted for routine use in the NHS in the future. If NICE decide it should be commissioned, it will be funded. If they decide not to recommend the drug then it is proposed that the pharmaceutical company will continue to fund the drug for patients who have already been receiving it via the CDF.  The proposal suggests a method of ensuring patient access to treatments that may be more financially sustainable than the previous one.

Simon Stevens, Chief Executive of NHS England, said: “Over the next five years we're likely to see many new cancer drugs coming on to the worldwide market - some of which will be major therapeutic breakthroughs, and some of which will turn out to offer little extra patient benefit but at enormous cost. The new Cancer Drugs Fund offers a route for sorting out the wheat from the chaff, so that patients in England get faster access to the genuinely most promising new treatments. For those drug companies willing to price their products affordably while sharing transparent information about 'real world' patient benefit, the new CDF will offer a new fast-track route to NHS funding.”[Source: https://www.england.nhs.uk/2015/11/19/cdf-consultations/]

The CDF has enabled thousands of patients to access innovative treatments, but in its current form the CDF has become financially unsustainable, leading to these proposals for its reform. CLLSA are working with other blood cancer and rarer cancer groups during this process of change to ensure patients with CLL will gain access to needed effective treatments. Our consultation comments to the questions below outline our position and issues regarding the proposals for reorganisation to the new CDF operating model:

 

Cancer Drug Fund Consultation:  CLL Support Association

Consultees are to answer with Agree, Disagree or Unsure to questions 1-8, 11 & 14    

1 Do you agree with the proposal that the CDF should become a ‘managed access’ fund for new cancer drugs, with clear entry and exit criteria?

Unsure

Please provide comments to support your response:

The CLL Support Association is not able to give unqualified support to this consultation paper because significant details are not provided at this stage from the documents provided. In addition to increasing the speed and clinical and cost effectiveness of the introduction of new drugs to CDF, there are two vital factors that must be recognised:

Firstly, patient and patient organisation involvement must be at the heart of any new proposal. It is essential to include patient perspectives, needs and priorities throughout the process, allowing knowledgeable input on areas of unmet need, inequality of access and disproportionate mortality statistics. A stronger patient voice will help address the current built in bias against rare and less common cancers and cancers of unmet need. For a chronic disease such as CLL, the new drugs in the pipeline offer several important advantages in addition to the survival advantage, and do not currently have a high weighting in the current appraisal process eg Quality of life, toxicity and side effects are all vital parts of the survival advantage in chronic disease. The step change offered by these new drugs is therefore not recognised in the current system.

Secondly, there needs to be a recognition that it will be harder to provide robust data in the development and testing phases of rare and less common cancers due to the small patient populations and the even smaller groups available with the introduction of targeted drugs. For example, in the current system, overall survival is scored zero and PFS is halved for phase ii trial data even if the drug has 'breakthrough' status.

 

2 Do you agree with the proposal that all new cancer drugs and significant new licensed cancer indications will be referred to NICE for appraisal?

Unsure

Please provide comments to support your response::

We remain unconvinced, due to lack of evidence, that NICE will have the quality of capacity needed to conduct all this work in the timescales proposed. In addition, the shorter timescales will be demanding for all participants and it is evident from our own experience that the current system already struggles to produce timely information for committees and invited participants to consider in full in a timely manner. A way forward would be to consider a prioritisation and fast track system for 'breakthrough' drugs which will be used by patients with cancers of greatest need.

 

3 Do you agree with the proposal that the NICE Technology Appraisal Process, appropriately modified, will be used to evaluate all new licensed cancer drugs and significant licence extensions for existing drugs?

Disagree

Please provide comments to support your response::

CLLSA cannot agree with this proposal unless there are demonstrated commitment to involving patients at ALL stages of the process. If the major stakeholder is not at the table when decisions are made then the system will not have moved forward and the current inequalities that have arisen because of lack of understanding will never be addressed.

The CLLSA has contributed to the PACE approach introduced by the Scottish Medicines Consortium, and this has started to see more drugs for rare cancers and cancers of unmet need to be approved for use in Scotland. (This is detailed in Cancer52's recent report).

In the short term, we would like to see a review of the weighting system so that cancers with poor outcomes can be weighted when compared with forms of cancer where outcomes are relatively successful. For chronic cancers we would recommend extra weighting for the impact of toxicity, side effects and quality of life issues as these are extremely significant alongside the potential improvement in survival advantage.

Guidance for TA committees for rarer and less common cancers might need to be stronger in requiring a managed access fund arrangement to last for longer than 24 months if that is what is required to gather evidence to make a positive decision.

Long term there needs to be a reassessment of the QALY system.

 

4 Do you agree with the proposal that a new category of NICE recommendations for cancer drugs is introduced, meaning that the outcome of the NICE Technology Appraisal Committee’s evaluation would be a set of recommendations falling into one of the following three categories:

Agree

Please provide comments to support your response::

The use of 'real world' data is welcomed. Involvement of clinicians and patients in defining the subset for each cancer is essential. For rarer and less common cancers the period of 24 months may be too short to gather sufficient evidence to make a positive decision.

 

5 Do you agree with the proposal that “patient population of 7000 or less within the accumulated population of patients described in the

marketing authorisation” be removed from the criteria for the higher cost effectiveness threshold to apply?

Agree

Please provide comments to support your response::

We agree with this proposal, however the implication of this is that more cancer drugs will be approved by NICE for the more common cancers and therefore additional reform is needed for rarer and less common cancers to benefit in a similar way in the future.

 

6 Do you agree with the proposal for draft NICE cancer drug guidance to be published before a drug receives its marketing authorisation?

Agree

Please provide comments to support your response::

We agree in principle but require clarity on how this will be achieved in practice, as the timeframe for completing this for every cancer medicine will be extremely challenging. We also require clarification that the valuable scoping process will be maintained in the new system.

As noted earlier, it is vital that patients are involved from the very start of the process and throughout the entire cycle.

 

7 Do you agree with the process changes that NICE will need to put in place in order for guidance to be issued within 90 days of marketing authorisation, for cancer drugs going through the normal European Medicines Agency licensing process?

Unsure

Please provide comments to support your response:

We require further clarification on the initial private NICE TA Committee Meeting and whether this will involve patients and clinicians, and patient group input. It is not clear what information will be available or the timeframe allowed for patient/clinician input. This is central to the future effectiveness of the CDF.

 

8 Do you agree with the proposal that all drugs that receive a draft NICE recommendation for routine use, or for conditional use within the CDF, receive interim funding from the point of marketing authorisation until the final appraisal decision, normally within 90 days of marketing authorisation?

Agree

Please provide comments to support your response:

We have already noted the small patient population available for evidence collection. We therefore recommend that interim funding for such a drug be made universal for all clinically appropriate patients during this interim period, rather than potentially restricting access to just those involved in the gathering of clinical data.

 

9 What are your views on the alternative scenario set out at paragraph 38, to provide interim funding for drugs from the point of marketing authorisation if a NICE draft recommendation has not yet been produced, given that this would imply lower funding for other drugs in the CDF that have actually been assessed by NICE as worthwhile for CDF funding?

What are your views on the alternative scenario set out at paragraph 38, to provide interim funding for drugs from the point of marketing authorisation if a NICE draft recommendation has not yet been produced, given that this would imply lower funding for other drugs in the CDF that have actually been assessed by NICE as worthwhile for CDF funding:

It is unclear where this would happen, why it would happen and the frequency of these events. More information would be required to form a judgement.

 

10 Do you have any comments on when and how it might be appropriate for the CDF in due course to take account of off-label drugs, and how this might be addressed? Do you have any comments on when and how it might be appropriate for the CDF in due course to take account of off-label drugs, and how this might be addressed:

Not at this stage.

 

11 Do you agree with the proposal to fix the CDF annual budget allocation and apply investment control mechanisms within the fixed budget as set out in this consultation document?

Unsure

Please provide comments to support your response::

There is insufficient detail and working examples provided to form a judgement. Historically the CDF has been either significantly underspent or more recently overspent. There is no information on how the new budget will be set and so no comment can be made.

 

12 Do you consider that the investment control arrangements suggested are appropriate for achieving transparency, equity of access, fair treatment for manufacturers and operational effectiveness, while also containing the budget?

Are there any alternative mechanisms which you consider would be more effective in achieving those aims?

Do you consider that the investment control arrangements suggested are appropriate for achieving transparency, equity of access, fair treatment formanufacturers and operational effectiveness, while also containing the budget?

Are there any alternative mechanisms which you consider would be more effective in achieving those aims:

NO.

We do not believe the investment control arrangements suggested are appropriate for achieving transparency, equity of access, fair treatment of manufacturers and operational effectiveness, whilst also containing the budget.

As already noted, it is essential that patient/patient group/cancer charity involvement forms a key part of the price-setting and budget management.

 

13 Are there any other issues that you regard as important considerations in designing the future arrangements for the CDF? Are there any other issues that you regard as important considerations in designing the future arrangements for the CDF:

1. How will NICE/CDF put patients at the heart of its processes and information gathering?

2. Given the obvious issue of capacity, increased volume and speed of appraisals, should consideration be given to fast track system for 'breakthrough' therapies?

3. While we appreciate that high market prices can create a stumbling block and we support proactive, productive measures to control the public expenditure, we are concerned that this proposal focusses primarily on the pharmaceutical companies and the reduced timescale and therefore misses the opportunity to address the major improvements and advances to patient care that could be achieved.

There is an additional concern that intractable and inflexible discussions could discourage pharma from marketing in the UK.

4. Given the issues of inequity of access to treatment, generally poor outcomes and disproportionate mortality statistics for certain categories of cancer, we would welcome new separate entry points, or means of prioritisation, into NICE/CDF. These categories would include chronic cancers, treatment for cancers of unmet need, orphan-drugs/treatments for rare/less common cancers and end of life treatments.

 

14 Do you agree that, on balance, the new CDF arrangements are preferable to existing arrangements, given the current pressures the CDF is facing?

Unsure

Please provide comments to support your response:

A key feature of the existing CDF was its complete lack of transparency, particularly for patients, and the unwillingness to communicate or engage with patients and public. The inflexibility shown in the recent reassessment of drugs for removal from CDF was not encouraging. A longer term view of patient needs is a key requirement, together with an informed view of the drug pipeline, and it is greatly disappointing that this proposal falls short in this vital area.