The publication of new arrangements for the Cancer Drugs Fund by NHS England on 8 July marked the end of over a year of intense debate about how best to get potentially life changing cancer medicines to NHS patients. It has not been an easy journey and there has been much frustration and consternation on the way with politicians, charities, clinicians, NHS and pharmaceutical industry executives trying to understand and make sense of what is a highly complex and challenging problem. So now is a good time to pause and reflect on what we have actually achieved and what might happen under the new arrangements, which will come into effect from 29 July.
So now is a good time to pause and reflect on what we have actually achieved and what might happen under the new arrangements, which will come into effect from 29 July.
The good news is that a number of fundamental issues have indeed been addressed in the new arrangements. The original CDF had a design flaw, which was that once a medicine entered into the fund there was no way for it to leave, inevitably causing expenditure to grow, exponentially, and that is precisely what happened. During about its third year of operation, the fund switched from an underspent to a massively overspent situation. The new fund now has clear entry and exit criteria allowing medicines to move in and out over time. We have also created a brand new mechanism for getting some promising cancer medicines to NHS patients more rapidly. This includes those medicines to be routinely commissioned and those to be be conditionally approved. The latter category built on the premise that whilst the evidence base is mostly in place for medicines at the time of licensing, for some, further data is required to support full reimbursement and confirm the place of the medicine in routine clinical practice. This is good news for patients who should be pleased that these medicines will now be paid for under the CDF during the period whilst more data is collected.
The other piece of good news – for cancer medicines at least – is that we have managed to 'join up' the system, and fully re-connect NICE and NHS England processes in a coherent way. Using the previous CDF to pay for medicines rejected by NICE created a major disconnect and perverse incentives, with companies often caught up in the middle.
Finally, we must not lose sight of the significant benefit in the new CDF arrangements for collecting 'real world data' from the NHS and for the first time being able to potentially utilise this in the pricing and reimbursement environment, alongside more traditional types of evidence. In the longer term this may be prove to be one of the most important components of the new arrangements, but for the moment it is the least developed and relies on an infrastructure and an analytic framework which is yet to be fully worked up and proven. Nevertheless, it is most welcome that Public Health England now joins forces with NICE and NHSE to support the cancer drugs fund going forwards. We need to ensure that the commitments made in the HTA environment around data collection are, in practice, able to be followed through in a timely manner, particularly so once the clock starts ticking on data collection arrangements. Only by working in close partnership from the outset with companies wanting to take part in the new CDF will this be achievable.
So there is some progress, and everyone has tried to work with best intent. But have we really been addressing all of the right problems, and have we now created some new problems that lie around the corner?
We have a solution for getting medicines requiring more evidence to NHS patients, but what about those medicines that do have the requisite evidence at launch? These medicines will have to go through a largely unchanged NICE appraisal process that necessitated the setting up of the CDF in the first place. There is no change to the decision making framework used by NICE and what's more NICE will now be appraising all new cancer medicines including those for rarer cancers. History tells us that these medicines, targeted at smaller patient populations generally do less well under a 'one size fits all' appraisal process and even the flexibilities for evaluating rarer cancer medicines that were provided by NHSE under the previous CDF have been lost.
It is also of concern that we have not yet managed to find a way of better linking horizon scanning information about new medicines to the budget setting process. Moving to a fixed ring-fenced CDF budget, which does not reflect the dynamic nature of the pharmaceutical pipeline, creates a new problem. Industry is being asked to bear 100% of the entire risk of any overspend on the entire CDF budget which, individually, companies have no control over potentially leaving them exposed to open ended levels of financial risk. Moreover, taking a slice of the new CDF to provide 'interim funding' for all cancer medicines that garner positive provisional NICE guidance up until the expiry of the 90-day mandatory implementation period, again 100% underwritten by companies creates another new and significant pressure. The problem is that historically managing the CDF budget has been very difficult and with the new fund being used to support eight separate categories of expenditure it is not going to get any easier. Only time will tell if this will be acceptable to companies.
There remains an enormous amount of work to do to make the new arrangements work, not least the complex task of scheduling and delivering a huge NICE work programme consisting of existing CDF medicines, those that have been 'in limbo' since the fund was closed to new applications, and new medicines launched or nearing launch. Whilst this is now underway, I was reminded only last week about a new promising treatment for refractory thyroid cancer, which may not complete its NICE appraisal until 2018, some three years after being licenced in the UK, and for a relatively small number of NHS patients with a high unmet medical need. Sadly, there are other medicines caught 'in limbo' in a similar position.
So, a number of positives: a new managed entry fund; conditional approvals for some cancer medicines, with real world evidence collection; earlier access for some patients. But some new challenges: an unproven data collection infrastructure; an untried arrangement which sees companies being asked to bear 100% of the entire risk of any further over expenditure; and a huge backlog of NICE appraisals to do because it has taken us so long to sort the new arrangements out.
So do we have a better deal for cancer medicines?
My glass remains half full rather than half empty, but only just.
The new CDF is to become a 'managed access' fund and will in future be used to pay for eight categories of medicines by providing:
Interim funding for cancer medicines which have been provisionally approved for eventual routine use on the NHS from the NHSE specialised commissioning budget, from the point of issuing draft NICE guidance, through the period up to the issuing of final NICE guidance, and then to the end of the mandatory 90-day implementation period.
Interim funding for cancer medicines which have been provisionally given conditional approved for use on the CDF from the point of issuing draft guidance to the point of finalization of NICE guidance
Funding for these same CDF medicines throughout the period of gathering additional evidence via managed entry arrangements up until the completion of NICE re-review, this will usually be around 2 years but will be determined on a case by case basis.
Funding for 'off-label' cancer medicines approved through NHSE specialised commissioning processes.
Funding for existing CDF medicines in the transition process being re-reviewed by NICE (a process which will take around 18 months to complete).
Funding for the continuing costs of cancer medicines that were removed in previous rounds of so-called 'delisting' where existing patients are still being treated with these medicines.
Individual funding requests for cancer medicines up until August 2016.
Administration costs of NHSE, NICE and Public Health England, 2% of the fund annually.
The management of the CDF budget is the responsibility of NHS England and a new NHSE-led CDF Investment Group will be set up consisting of representatives from NICE and NHS England to perform that function.
But, industry will be required to commit to paying back 100% of any overspend of the allocated CDF budget, fixed at £340m, on a retrospective basis on all but the last three of these categories.