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New Model for the Cancer Drugs Fund (England)

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Recent developments in access to CML drug treatments in England

Background:

Some of you may be aware of the NHS England public consultation on a new set of proposals for the way the Cancer Drugs Fund (CDF) should operate after 1st April this year.

Many of you will also know that dasatinib, bosutinib and ponatinib are only accessible by CML patients in England if their doctor applies to the CDF for funding to pay for the treatment.

A patient is also required to meet certain clinical criteria in order for an application to be successful.

However some patient's clinical profiles fall outside the criteria despite the CML drugs involved being licensed for treatments that they would benefit from.

A further complicating factor is that the operating procedures of the current CDF allows for the re-evaluation of the drugs that are included in the scheme.

This works using a points based scoring system to evaluate each drug and has an in:out dividing line score that is used to move drugs out of the list of CDF drugs. The dividing line score is not fixed but can be moved up or down which produces situations where a drug can move out of the CDF very soon after qualifying for entry even though there has been no change in the clinical evidence that supported its entry into the fund.

The most recent re-evaluation resulted in bosutinib no longer being available for CML patients showing resistance to nilotinib and dasatinib – although those showing intolerance are able to access this particular TKI.

The plans for drugs, including those for CML, already in the CDF:

Included in the consultation proposals is a transition process to enable drugs to be moved from the current CDF to the ‘new model’ CDF that will operate in a rather different way than the existing model. Details of the transition process for these drugs were released earlier in the year.

The first stage in the process has been to separate the drugs into groups. Drugs that have previously been appraised by NICE (the public body responsible for evaluating the clinical and cost effectiveness of drugs before they are routinely used in the NHS in England) are in Group 1. Both dasatinib and bosutinib are included in this group.

Drugs that NICE have decided they will not appraise, generally because the number of patients qualifying for treatment is very small, are included in Group 3. Ponatinib is included in this latter group.

The second stage in the process is movement of each drug through a step by step evaluation schedule to determine if they will be recommended for routine use in the NHS in England.

The schedule is the same for all transition drugs no matter what group they are in. The difference between the groups concerns the timetable for their entry into the process.

The published timetable for the evaluation of Group 1 drugs indicates they will emerge from the evaluation process with a decision on their future use as treatments for CML in autumn 2016.

Currently a timeline for Group 3 drugs has not been published. It is certain that no decisions will be published this year and it has been informally acknowledged that the original end of March 2017 timeline for process completion for all transition groups will be breached. A best guess would be a decision about ponatinib would be made in late 2017.

Evaluation recommendations for drugs included in the transition and for all new anti cancer drugs:

A new feature of the NICE evaluation process is that all new anti cancer drugs will, from April 1st 2016, be referred to NICE for evaluation (generally described as an appraisal). There will be three possible recommendations available to NICE Committees when they appraise cancer drugs. This includes a new recommendation which is formally described as ‘recommended for use within the CDF’.

In essence the three decisions are Yes, No and (a new designation) a ‘provisional Yes’.

‘Yes’ means a drug (e.g. imatinib or nilotinib) is to be routinely available in the NHS in England for CML treatment. The practical effect is that doctors can then write a prescription and the manufacturer the drug will be paid at the price agreed by NHS England.

‘No’ means that the drug will not be made available, unless a special case for its use is made using an Individual Funding Request (IFR), submitted by a patient’s doctor as an application to the CDF assessment panel.

However obtaining approval using the IFR method is very difficult indeed because of the very restrictive criteria governing this process.

For example, in 2014/15 only 0.5% of IFR applications for cancer drugs were successful.

The new ‘provisional Yes’ decision will trigger a two stage process where the company negotiates a price for their drug that NHS England is ‘willing to pay’ during the generally 24 month period when the ‘provisional Yes’ recommendation is operative, and secondly discusses with NICE and NHS England exactly what kind of evidence would be necessary to convert a 'provisional Yes' into a ‘permanent Yes’ (or perhaps a No).

It remains unclear exactly who will manage this evidence gathering process. Although there are some details available, it's fair to say there is much that remains to be published about how evidence gathering will be undertaken, exactly what will count as evidence, the number of patients involved and how it can be ensured that the process is progressing as agreed.

At the end of the ‘evidence gathering process’ there will be another NICE appraisal called a Shortened Technology Appraisal (STA) that will examine the new evidence, compare it with the evidence used to make the initial 'provisional yes' recommendation and finally come to a decision on whether or not to grant routine access to the drug.

At this point there will only be a Yes or No recommendation available for Committees to use in reaching their decisions.

The company involved can also of course revise the drug’s price over the duration of the process to meet the more exacting requirements introduced into the process to ensure drugs remain within the limits NHS England is willing to pay for a drug’s use via the NHS.

The willingness to pay limits are called ‘thresholds’ and these can vary upwards in the case of a class of drugs called 'End of Life' drugs that must meet certain criteria. Such criteria becomes operative when a drug enters the NICE appraisal system.

The company will be expected to supply their drug at zero cost to CML patients with the same profile as the group of patients required for evidence gathering. They will also be expected to continue to supply the drug at zero cost to these patients, as well as the patients within the evidence gathering group, even if their drug is eventually awarded a ‘No’ recommendation.

Finally, to give a full overview of the system, there is another appraisal system that NICE operates called the Highly Specialised Technology appraisal system (HST) but to date there is an informal bar on anti cancer drugs being permitted entry into this system.

CML patients in England currently being treated with CDF drugs (dasatinib, bosutinib & ponatinib):

A very clear commitment has been given by NHS England that these patients will continue to be treated after April 1st as they are currently, unless they and/or their doctors decide otherwise.

UK CML patients outside England:

The proposals described above and the current CDF machinery does not apply to CML patients being treated in Wales, Scotland or Northern Ireland.

CML patients with clinical profiles that fall outside of current CDF criteria, or who would have received treatment with a drug that was previously inside the CDF but has since been removed:

Examples (and these are not exhaustive) include for the former category, ponatinib licensed for CML treatments other than for that showing the the T315i mutation.

An example of the latter category would be bosutinib for the treatment of patients resistant to dasatinib or nilotinib.

NICE has indicated that their transition review process would include a full appraisal of all clinical profiles ponatinib has a license for and not just the T315i patient population, providing the technical details involved are jointly agreed between the company (Ariad) and NICE.

NICE has also agreed that, in principle, it is willing to review drugs that were once, but are no longer, in the CDF although this will not be a priority.

However, since bosutinib remains within the CDF for the treatment of patients who are intolerant of dasatinib and nilotinib, it will be reviewed in the manner described for ponatinib, despite it being removed from the CDF for patients resistant to dasatinib and nilotinib. The difference being that ponatinib has never been assessed by NICE, whereas bosutinib has.

It would seem, although details are lacking, that the review of bosutinib will be heavily constrained by the evidence that was originally submitted to NICE when first appraised, whereas Ariad will not be as constrained in their submission of evidence for ponatinib.

CML Support Group's position:

Our consultation response, see the attached pdf, argues that the proposals for the new model are simply a more sophisticated version of the current model. As such, there remains great uncertainty about the outcome of both the transition reviews and indeed the appraisal of any new targeted therapy for CML.

We suspect that the highly unusual feature of CML TKI drugs (that post treatment survival for most CML patients is likely to be decades long and that the patient populations for the newer TKI treatments are small and becoming smaller), will result in the outcome of the transition reviews being a provisional Yes (‘recommendation for use in the CDF’).

That means another two years of evidence gathering and a subsequent six month evaluation before a final decision is reached in late 2019 or early 2020.

Our view is that the new proposals do not address the core problem, which is that the way NICE appraises medicines is now, at 15 years since the appraisal process was initiated, an outdated model no longer fit for purpose, especially given the progress made in drug development over the last two decades.

Targeted therapies for CML have lead the way in posing this challenge since the appraisal of imatinib well over a decade ago.

The current government and the Coalition government preceding it, remain committed to the reform of NICE, but in reality virtually nothing has changed. Claims made about the possibilities offered in the consultation proposals by more comprehensive coverage and the additional recommendation remain highly tentative because the way evidence is assessed remains unchanged.

It is entirely possible that a recommendation in 2019 or 2020 may be a 'No' rather than a ‘Yes'.

We think this is bizarre if not bordering on the ridiculous.

David Ryner