Is the Cancer Drugs Fund beyond fixing?
Barbara McLaughlan discusses England's Cancer Drugs Fund (CDF) and suggests ways of securing a new, sustainable assessment and funding model for cancer medicines.
Background
Access challenges and cancer drugs have been inextricably linked for as long as many of us can remember. Controversy around postcode prescribing and variability of care across England led to the introduction of the National Institute for Health and Care Excellence (NICE) in 1999. However NICE did not solve the problem. In fact, the barriers around access to medicines were catapulted into the public consciousness like never before through those early patient group campaigns demanding NICE reconsider some of its decisions in cancer.
Cancer: a special case
Cancer has always seemed to be more affected by the NICE process than other disease areas. There are many reasons for this but it has long been felt by many stakeholders that the stringent health economic assessment by NICE does not reflect the specific challenges associated with determining the cost-effectiveness of cancer drugs, particularly in the metastatic setting.
In 2013, NHS England established a single national Cancer Drugs Fund (CDF), designed to provide funding for new cancer drugs based on a defined set of clinical criteria. This decision was not without its controversy and was perhaps a reaction to cancer outcomes, and levels of medicines access, falling behind other countries in Europe and beyond. Accusations of a political sticking plaster and criticism of preferential treatment of cancer were rife and continue to this day. But even as it divided opinion, the CDF has provided access to important cancer drugs for tens of thousands of patients in England since its inception.
However one concern around the CDF – its sustainability – has proved to be well founded. While the Fund was underspent in the first three years of its operation, it has experienced increasing budgetary pressures that were only partly mitigated by increased budgets. This is due to the increasing number of innovative cancer medicines launched and the fact that many of them were getting 'stuck' on the CDF rather than moving successfully through the NICE process and into routine commissioning.
Why is the CDF now unsustainable?
A key reason for this bottleneck is that the NICE appraisal system has not evolved to take into account the way medicines are being increasingly licensed in Europe. The system of conditional approval by the European Medicines Agency (EMA) is a route designed to allow faster access to promising medicines for patients with high unmet need. But this 'fast-tracked' approval means that drugs are launched in the UK with only immature, often phase II, data available to NICE for appraisal.
This more often than not leads to NICE rejection, given that the NICE appraisal system is predicated on certainty of data and usually focuses on overall survival as a key indicator of value.
So as drugs continued to be funded by the CDF, and new drugs were added to the fund, the system started to break down. As a result, over the past nine months, NHS England has conducted two rounds of delisting, removing a large number of drugs or imposing very substantial rebates through a rather simplistic costing element. In addition, no new drugs will be reviewed for inclusion in the CDF list for the foreseeable future, removing it as a potential route of access to new medicines in advance of NICE review.
We have argued for a long time that a long-term solution is required and must be implemented as a matter of urgency. These two rounds of delisting confirm how the uncertainty around funding is having a negative impact on manufacturers, clinicians and, not least, patients.
The way forward - the CDF as a 'managed care fund'?
The wider NHS England proposals for the CDF as a managed care fund overseen by NICE are broadly shared with many stakeholders. The new CDF needs to be sustainable and the only way to achieve that is to ensure that drugs have a reasonable chance of being approved by NICE. That way they will move into routine commissioning, create headroom for new drugs to enter the CDF and, where appropriate, benefit from a period of conditional approval without the current access barriers. After all, conditional approval acknowledges the issues created by immature data at launch and the need to either collect real-world data or allow data from ongoing trials to mature.
It is clear that real-world data can be important in affirming the most appropriate use of a medicine in clinical practice. However, as a side note, we must be cautious around its interpretation. Poorer outcomes in the real world can be an indication of a treatment being less effective when used in day-to-day clinical practice. But it can also be a reflection of the level of care provided to patients outside clinical trials. So real-world data must be looked at in the round – to both supplement and reinforce trial data but also to highlight where care can be improved, and indeed how pharma and the NHS can work together to help achieve this for patients.
Barriers beyond reimbursement
The current reimbursement system is not the only barrier to cancer drug access in the UK as there are many other factors that need careful consideration if we are to succeed in improving access to innovative medicines.
The Accelerated Access Review (AAR), and the stakeholders informing it, has identified a number of challenges that reflect our experience in clinical practice.
"Fast uptake of generic medicines is positive as long as the savings realised are then used to prescribe the most promising new medicines"
Prescribing practice is one. The UK is seen as a country where clinicians tend to be conservative in their prescribing practice and are also more likely than other countries to prescribe generic medicines as soon as they become available. Fast uptake of generic medicines is positive as long as the savings realised are then used to prescribe the most promising new medicines for patients, thereby saving the NHS money where appropriate but not at the expense of better outcomes for patients from the latest advances in medicine.
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Sir Hugh Taylor, chair of the Accelerated Access Review[/caption]
Sir Hugh Taylor, who is leading the AAR, rightly points out that there are a number of potential reasons that can drive this conservatism, such as perceived lack of evidence around patient benefit, lack of empowerment and pressure to save money.
Ultimately, the best way to address conservative prescribing is for manufacturers to ensure that they develop drugs that unequivocally demonstrate patient benefit and are cost effective. Where a drug has been approved for funding, no further barriers should be put in place to restrict its use in eligible patients and we do not support the notion that the NHS requires additional affordability criteria.
Another barrier is capacity and this has also come through clearly during the AAR engagement exercise. Adoption of innovative products needs to be easy and quick and the system needs to reflect this. NHS professionals are already stretched and if choosing innovative medicines means increased workload and less time to spend with patients then this becomes a significant barrier to uptake.
Going beyond cancer
We would support a wider 'Innovative Medicines Fund' in England where similar principles to those being proposed for the new CDF could be applied. The reason why discussions currently focus on cancer medicines is because this is where the system for appraisal and reimbursement is most evidently broken. Yet, issues around immaturity or paucity of data and the need to bridge the time between launch and a NICE decision apply equally to patients with life-threatening, non-cancer and severe long-term conditions, particularly when we consider rare diseases and medicines that are not a priority for NICE review due to limited budget impact. We still need to see the Scottish example of an Innovative Medicines Fund work in practice before being able to conclude whether this might be a good model.
In the interim, the focus on cancer is justified, as patients often do not have any treatment alternatives and most new treatments are initially developed in the metastatic setting where time is precious and maintaining quality of life is vital. Ultimately, the CDF should be seen as a useful pilot that has helped thousands of patients, brought some of the challenges in our reimbursement system to the forefront and could eventually be extended to other disease areas for the benefit of the entire NHS and the patient population in England.
About the author:
Barbara McLaughlan is Head of External Affairs at Novartis Oncology and a member of the Oncology Leadership Team within Novartis. Her role is focused on ensuring that Novartis has a strong voice and an active role alongside other stakeholders from industry, the NHS and government in improving patient access to medicines for cancer and rare diseases in the UK.
Before joining the company in 2011 Barbara was Eye Health Campaigns Manager for RNIB and before that in government affairs (both in Europe and the UK), and education and communications roles in Germany, Belgium and the UK.
She has an MA in Political Sciences, Russian and Polish and a postgraduate degree in European Affairs from the College of Europe, Bruges, Belgium.
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