Access to cancer drugs: planning for every outcome

The new assessment procedures for oncology medicines in the UK launch this week. Angela McFarlane examines the process and implications for pharma and patients.

After a series of delays, the new NICE and NHS England (NHSE) arrangements for patient access to cancer medicines come into force on 29 July, bringing with them sweeping and far-reaching change for UK market access.

All new cancer medicines will now be assessed by NICE, without ministerial topic selection and, if there is uncertainty about clinical effectiveness, will be recommended for conditional access via the NHSE Cancer Drugs Fund (CDF) – which has been capped at £340 million – as an interim measure on the road to reimbursement and before market authorisation.

The promised pay-off is that the new scheme is intended to grant patients in England access to life-saving medicines faster than ever before, while encouraging manufacturers to launch innovative medicines in the UK.

There are other benefits to NICE reviewing all oncology drugs prior to marketing authorisation, including clear entry and exit criteria, rapid appraisal, and interim funding from the point of approval. Additionally, industry has learned to understand NICE’s expectations over the past 17 years’ experience and, although there have been tough learnings for both sides, patient access has improved to the extent that, since 2010, the UK has had the third-highest uptake in market share of speciality products in Europe, according to our research.

Yet, there remain significant concerns within the pharmaceutical industry about a number of elements of the new arrangements. These include the timelines, the risk behind conditional funding and lack of visibility around the real-world evidence (RWE) criteria that will need to be delivered in the event of CDF funding.

However, with a new (and hopefully transparent) NICE process underpinning access to the revised CDF that harnesses the UK’s position as a global leader in RWE, in theory at least, we can hope for shorter timelines in terms of patient access to oncology medicines.

Essentially, there are three possible outcomes for NICE drugs appraisal: a drug can be approved, rejected, or granted ‘conditional approval’ and given temporary funding through the CDF while more evidence to address the uncertainties established is collected.

It's this middle ground of ‘conditional approval’ that is giving manufacturers pause for thought. If a drug is handed a ‘maybe’, manufacturers must fulfil an obligation to collect RWE, additional Health Economic Outcomes Research (HEOR), for a two-year period in a bid to address the areas of uncertainty, as NICE sees it, ahead of securing full approval.

Quite how this will work in practice is the cause of considerable consternation within the pharmaceutical industry and patient advocacy groups alike. This is the first time a national HTA organisation has made RWE a mandatory element in the approvals process – albeit only under certain circumstances – and, while many pharmaceutical companies are engaging in RWE exploration on some level, there is confusion and inconsistency surrounding the ideal models for RWE study and best practice.

(What does this new process look like? Click HERE for a snapshot)

Guidelines, clarifications and RWE

While many questions remain over the new legislation, NHSE has taken steps to inform the industry of its responsibilities with the publication of an extensive Standard Operating Procedures (SOP) document, unveiled on 8 July.

While designed to offer a broad overview of the motivations underpinning the new system – and advice for pharmaceutical companies seeking oncology market access – the SOP fails to address the many questions surrounding the technical aspects of the submissions process and the possible RWE/HEOR requirements and quality standards. On top of this, NICE and NHSE do not yet have a clear set of guidelines on the approach that should be taken when analysing and interpreting RWE.

Implications for pharma

This is unfortunate given that the revised CDF represents a significant departure from the legacy system and, as such, there are a number of implications for pharma. One major impact will be that pharma companies will be required to submit their evidence dossier to NICE well ahead of market authorisation, coupled with a commitment and well-thought-through strategy to provide substantial RWE should NICE return with a ‘maybe’ decision.

• Existing CDF drugs: If the drug is currently funded via the legacy CDF system, then it will be reappraised by NICE under the new guidelines. The process of reappraisal began in April of this year, with Pfizer’s Bosulif (bosutinib) the first to have been given a green light for approval.
• CDF is no longer a long-term reimbursement option: The new CDF, under the jurisdiction of NICE, cannot be considered a long-term alternative source of funding. Instead, by bringing the CDF into the NICE approvals process, it has become an interim measure – the end game being complete approval through the usual NHS reimbursement channels.
• RWE is no longer optional: With the unrelenting pace of change heading towards outcomes-based pricing, RWE is billed as the centrepiece of the future approvals process. This will require pharmaceutical companies to develop and implement an HEOR strategy in line with the new NICE review process – and to be able to rapidly adapt and respond with a RWE tactical plan if conditional approval is granted.

Plan for every outcome

A robust RWE strategy should now be a staple for oncology market access in the UK. Aim for a positive appraisal from NICE, but have the strategy and tactics in place for a CDF ‘maybe’, before submitting the dossier.

The risk in not making these preparations is that a conditional approval outcome, without a strategy in place and ready to go, could set market access back by a number of years, and influence reimbursement decisions in other key markets.

In short, every pharmaceutical company should prepare a ‘Plan A’ and ‘Plan B’ from the outset. Companies must do all they can to gain a positive decision through the now accelerated initial NICE approval pre-marketing authorisation, but be able to swiftly back this up with their own RWE strategies in response to NICE’s demands.

So, while the new CDF guidelines and RWE requirements may seem to present more challenges than opportunities, provided NICE, NHSE, the wider NHS, patient organisations and pharmaceutical stakeholders all focus to make the new system work, it should result in accelerated market access for oncology medicines.

Ultimately, the acid test of the new arrangements will be accelerated access across England to new cancer medicines that extend both the quantity and quality of patients’ lives. That has to be worth getting the approach right.

About the author:

Angela McFarlane is Market Development Director, QuintilesIMS Market Access.

Learn more in next week’s live panel discussion:

These concerns, and the strategies and tactics required to successfully steer through this new oncology landscape, are the focus of an upcoming pharmaphorum webinar and NHSE/NICE panel discussion on Tuesday 2 August.

Angela McFarlane, Market Development Director, QuintilesIMS, will be joined by Nina Pinwill, Associate Director at NICE and Malcolm Qualie, Pharmacy Lead, Specialised Services at NHS England, to discuss the reinvigorated Cancer Drugs Fund.

The session will be hosted by pharmaphorum media, and will provide exclusive insights into how manufacturers should navigate the new system, how to engage with NICE and NHSE and how to design a successful real-world evidence (RWE) study.