CDF proposals force pharma to prepare for NICE at the same time as EMA submissions

Will the CDF proposals force pharma to prepare for NICE at the same time as EMA submission?


In February, NHS England announced that the UK Cancer Drug Fund (CDF) will get a new lease of life from
July 2016 as a ‘managed access’ fund with a fixed annual budget of £340 million. Currently, the CDF can
choose to pay for new cancer drugs even if NICE has rejected the drug for widespread use on the NHS.
Come July, NICE will gain decision power by saying yes, no, or maybe to cancer drugs entering the UK
market. Drugs in the yes category will be offered on the NHS while those given a maybe rating will be
considered to enter the CDF. Those drugs rated with a no will be denied access to the NHS in England.

So what’s new?

One of the most important aspects of the revised CDF is that new cancer drugs or significant new licence
indications will now undergo an evaluation by NICE and receive draft guidance before receiving regulatory approval in the UK. This process is aimed at accelerating patient access for cancer drugs that are rated cost-effective by NICE. Sir Andrew Dillon, NICE chief executive said of the changes: “Patients in this country will now have access to clinically and cost-effective, innovative new cancer drugs faster than ever before.

In a first of its kind approach, NICE will issue draft recommendations on the use of cancer medicines before they receive their licence, with funding from NHS England available if approved. No other country in Europe does this.”

Drugs with a promising clinical profile, but for which the evidence is not strong enough for routine use, will be given a conditional recommendation by NICE. These drugs will be available to NHS England patients via the CDF. Conditional recommendation is granted for up to two years during which time the manufacturer must gather more evidence on the cost-effectiveness of the drug. NICE will then use the new evidence to determine whether to move the drug out of the CDF into routine NHS funding, or if insufficient evidence
was provided to make the drug only available on an exception, individual patient, basis.
Drugs currently funded by the CDF will stay on the list but will undergo a new appraisal by NICE in the near future. Any patients currently receiving those drugs will be entitled to complete their treatment.

Not everybody is happy

As in the past, pharma companies will need to show cost-effectiveness for their novel cancer treatments
either at time of market entry or two years down the line. Alternatively, pharma companies might engage
in a patient access scheme with the Department of Health in order to obtain patient access for their drug.
Pharma companies are not happy with the new plans and many think this is a repacking of the past. Dr
Paul Catchpole of the Association of the British Pharmaceutical Industry, said pharma companies were
‘disappointed’ by the plans. He believes that “this carries the very real risk of significantly setting back
patient access to cancer medicines, now and for the foreseeable future” and that “turning the clock back
five years just doesn’t make sense”. It is feared that “we will largely get the same answers as before – the
majority of medicines will be turned down”.

The Cancer Research UK is more positive and likes the faster initial recommendation and the introduction
of a ‘maybe’ recommendation with subsequent data collection. However, they feel uncertainty about how
the new CDF will fit with the established Early Access to Medicines Scheme (EAMS). The latter allows
unfunded earlier access to promising products before marketing authorisation, which has previously
included new cancer drugs such as Keytruda.

What does this mean for pharma?

The change to the funding of cancer drugs in the UK will have two major impacts on pharma companies.
Firstly, the CDF will only be used for generating new ‘real world’ evidence to support the cost effectiveness of the product. This means that pharma companies need to provide a real world evidence plan at product launch, to ensure that the necessary data can be collected within two years. Implementation of a real world evidence program is a resource intensive initiative and developing the right real world evidence plan for launch (when commercial teams are focused on a global launch) will be challenging. The second major consideration is the early NICE evaluation for novel cancer treatments prior to regulatory approval. This means that the NICE dossier should be ready ~ 35 weeks prior to marketing approval. Consequently, the pharma company must start preparing its NICE submission and health economic model at the time of making the regulatory submission to the EMA. This is likely to put significant additional pressure on already stretch commercial teams.

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