Conflict of interest: the proposals reviewed here are similar to those advocated in a 2014 BMJ editorial “Reforming the Cancer Drugs Fund” to which I was a co-author. I have had no involvement with the development of the proposed policy.

The Cancer Drugs Fund continues to pose problems not least a rising spend, up from its £200m in 2011 to £340m in 2015/16. Set up as an interim measure until 2013, it has had to be renewed each year with a current extension until 2016. Given the state of NHS finances, a review has long been due. A consultation document published on 19 November outlines a proposed way forward. 
In July 2015, NHS England proposed that the fund should become a “managed access” fund that pays for promising new drugs for a set period before NICE decides whether the drugs should be routinely available on the NHS. The present consultation is part of preparing new arrangements from April 2016.

The consultation document acknowledges the context: “the NHS in England is currently allocating an increasing share of the cancer budget to treatments of uncertain value, and the impact of this is being felt in other cancer services and in other parts of the NHS.”

The wider context might also include concerns over “onco-exceptionalism” and the continuing challenge of bypassing NICE.

A recent National Audit Office review found that “All parties agree that the fund is not sustainable in its current form.”

The document is coy on what happened to value based pricing: “The CDF was originally intended to be a bridge to a new approach to the adoption of new drugs into the NHS, using “value based pricing.” As circumstances changed and alternative methodologies were explored, this approach was not, in the end, adopted.” For a different view of what happened with value based pricing see here and here.

The new consultation proposes to retain a Cancer Drugs Fund (CDF) but as a transitional fund “for selected cancer drugs with apparent clinical promise but uncertain value to move into and out of”….”while data is collected to inform a decision on whether to adopt the drugs for routine commissioning.”

The proposal is that “the CDF should become a ‘managed access’ fund for new cancer drugs, with clear entry and exit criteria. It would be used to enable access to those drugs which appear promising, but where NICE indicates that there is insufficient evidence to support a recommendation for routine commissioning. These drugs would be given a conditional recommendation by NICE and their use enabled by the CDF for a pre-determined period whilst further evidence is collected. At the end of this period the drug would go through a short NICE appraisal, using this additional evidence. It would attract either a NICE positive recommendation, at which point it would move out of the CDF into routine commissioning, or a NICE negative recommendation, at which point it would move out of the CDF and become available only on the basis of individual patient funding requests.”

Key to this is the data to be collected. The consultation states that the purpose of data collection is “to facilitate patient access with tightly focused research.
And
“Drugs could only remain in the fund for two years. During this period, data would be collected to resolve the uncertainty identified by NICE. “

More detail on data collection are provided in an appendix, which states:

“The data collection arrangements will be developed, during the consultation period, by the company, NHS England, and NICE with input from clinicians and patients, and on advice from NHS England’s Chemotherapy Clinical Reference Group and NICE’s Observational Data Unit (ODU). It will be completed before the final guidance is published. Funding for data collection and analysis will be provided by the company holding the marketing authorisation for the product.”

And “Progress with data collection will be reviewed regularly. An annual report, provided by the company or the organisation collecting the data, will be submitted to NICE to check whether the data collection is on track, and to establish whether any additional action is needed. This will be coordinated through the NICE Observational Data Unit.”

The decisions on what data to collect will be made by a joint committee of NHS England and NICE working with the company in a managed access scheme.

“To advise on when more data is necessary to reach a definitive view on their clinical and cost effectiveness. NHS England will therefore ask NICE to identify those drugs which are appropriate for time-limited funding under the CDF. The recommendations of the NICE Appraisal Committees for drugs they consider suitable for entry into the Cancer Drugs Fund will be received by a joint committee of NHS England and NICE (the Cancer Drugs Fund Investment Group), which will be responsible and accountable for confirming that an acceptable commercial access arrangement (the financial arrangements which determine the cost of the drug to the NHS, agreed between the company and NHS England) and data collection arrangements, which together form the managed access agreement, are in place before accepting the drugs into the Fund.”

From April 2016, it is proposed that all new cancer drugs and significant new licensed cancer indications will be referred to NICE for appraisal leading either to recommendations for or against routine use or for entry to the Cancer Drugs Fund.

….“Recommended for routine use would require the incremental cost effectiveness ratio to fall within the standard NICE range (£20,000 to £30,000 per QALY gained), taking into account the application of the End of Life criteria where appropriate.

… Recommended for use within the Cancer Drugs Fund would require the drug to display plausible potential for satisfying the criteria for routine use, taking into account the application of the End of Life criteria where appropriate. Entry into the CDF would be subject to the company agreeing to fund the collection of a pre-determined data set, during a period normally lasting no longer than 24 months, and a commercial access arrangement which is affordable within the available CDF budget.”

Linked to these proposals are two others to do with amending NICE’s End of life criteria:
• removing the restriction of the cumulative patient population; and
• emphasizing the discretion that exists for NICE Appraisal Committees to interpret the uncertainty criteria when considering a drug for inclusion in the Cancer Drugs Fund.

NICE’s usual methods for technology appraisal will apply: “If the NICE Appraisal Committee cannot recommend a drug for routine use, it will consider whether the drug is eligible for recommendation for use within the Cancer Drugs Fund. The Appraisal Committee will apply its usual technology appraisal methods and process…. To inform this decision, the Committee will take into account the following factors:

• Whether the incremental cost effectiveness ratio considered has the potential to lie within the thresholds specified in the NICE technology appraisal methods; and
• The extent and nature of the uncertainty in the clinical effectiveness of the drug; and
• The likelihood that the timeframe for data collection (including research already underway) will be able to inform a subsequent NICE appraisal, normally within 24 months.

Drugs whose potential range of cost per QALY does not include £30,000, taking into account any QALY weight applied in line with the End of Life criteria where appropriate, will not be accepted into the CDF.

The budget for the new Cancer Drugs Fund will be controlled: “To ensure the financial sustainability of the CDF, investment control mechanisms will be put in place to enable it to operate within a fixed budget. These measures will ensure that companies are encouraged to develop the most competitive commercial access arrangements and that there is an incentive for companies to generate and publish the required data as quickly as possible.”

“A CDF Investment Group (a joint committee of NHS England and NICE) will be established, consisting of staff from NHS England and NICE. The Group will be responsible and accountable for ensuring that the CDF is managed within its budgetary limits. It will receive and make decisions on recommendations from the NICE Appraisal Committees for drugs to enter the Fund, determine the managed access agreement in each case and monitor the use of the CDF.”

To summarise:
• NICE will appraise all new cancer drugs
• Using amended End of Life criteria abolishing the limit of 7,000 patients and emphasing scope for judgment
• Those cancer drugs refused by NICE but with a plausible case for achieving NICE’s £30k per QALY threshold will be eligible for the new CDF,
• In which case they can be funded for up to 2 years while data is collected aimed at resolving the uncertainties identified by NICE
• The cost of data collection will be borne by the companies and the cost of the drugs by the Cancer Drugs Fund,
• Data will be monitored closely involving a new NICE Data Observational unit,
• After a maximum of two years a second NICE appraisal will take place leading to the drug either being rejected or accepted.

What to make of this?

First, it will only help for those cancer drugs refused by NICE which have potential to achieve the threshold. Some cancer drugs are so highly priced that they have no scope to meet the NICE cost/QALY threshold, even if they qualified for the revised End of Life criteria. But these proposed new arrangements might increase the pressure on companies to consider price reductions within confidential patient access schemes, which continue unchanged.

Second, the proposed arrangements put NICE back centre-stage. It will do a first and then, after two years monitoring and data collection, a second appraisal. The latter will be binding. NICE will no longer be by-passed.

Third, a major question arises about the kind of data that can be collected in the two years allowed. This will be too short for most randomized clinical trials unless novel arrangements are made. While observational data on cancer outcomes could certainly be improved, the challenge will be to do with designing appropriate studies.

James Raftery is a health economist with several decades’ experience of the NHS. He is professor of health technology assessment at Southampton University. A keen “NICE watcher,” he has provided economic input to technical assessment reports for NICE, but has never been a member of any of its committees. The opinions expressed here are his personal views.