A review of the process and criteria used for consideration of treatments under the CDF framework. This work describes the extent of evidence collection while in the Fund.

1. NICE technology appraisal documents for all drugs included in the CDF (10.05.2018.) were
identified. Corresponding recommendations of the Scottish Medicines Consortium (SMC)
were also considered.
For each identified entry in the CDF, data was collected on:
• route to CDF entry (company submission, ERG identification or Committee identification),
• drug indication,
• reasons for inclusion in the CDF,
• ICERs preferred by the company, ERG and Committee,
• details of data collection whilst on the CDF,
• whether the drug was considered under the end-of-life criteria,
• SMC recommendation for use of the drug in the same indication within NHS Scotland.
Where possible, available data was summarised using descriptive statistics and plots.
Introduction
Objectives
Methods
Information was available on 17 entries in the CDF referring to 12 drugs. Drugs
were most frequently included in the CDF following an application in the company
submission to NICE (n=10). The drugs were indicated for a range of cancers, most
commonly non-small-cell lung cancer (n=4), urothelial cancer (n=3), lymphocytic
leukaemia (n=2) and multiple myeloma (n=2). The identified indications were
frequently for locally advanced or metastatic disease (n=10) and second or
further lines of treatment (n=12). NICE criteria for end-of-life care were met
in 12 cases.
The median preferred ICERs were: £43,227 (range £15,772 to £58,315) based on
company submissions, £54,386 (range £31,691 to > £100,000) for the Committee
conclusions, and £60,000 (range £44,504 to £ 106,457) based on ERG reports.
The median difference between ICERs provided by both the company and the
Committee (available for n=14 cases) was £11,602 (range -£1,686 to £51,053).
In all cases where the ERG provided a preferred ICER, it was greater than the
company’s (median £17,536, range £2,400 to £62,299). Where available, ICER
information for the company, Committee and ERG is shown in Figure 1.
Insufficient data on certain outcomes was the reason for inclusion of 16 entries in the CDF.
Other frequently cited reasons included uncertainties around the duration of treatment in
real world practice (n=6), short-term follow-up in studies (n=3) and insufficient data on
efficacy in subgroups identified by molecular markers (n=3).
NHS data collection methods were used in all cases and ongoing clinical trials were the next
most common data collection method (n=15).
Data was to be collected on the CDF for five outcome categories, as shown in Figure 3. These
categories included a range of outcomes, for example pre- and post-progression overall
survival.
In corresponding recommendations, the SMC recommended 13 of the 17 CDF entries.
Celia Sabry-Grant, Kinga Malottki, Alex Diamantopoulos
A REVIEW OF RECOMMENDATIONS
FOR INCLUSION AND DATA COLLECTION
WITHIN THE CDF FRAMEWORK
Figure 1: Company, Committee and ERG preferred ICERs (£/QALY) for all CDF entries.
Figure 2: Duration for which drugs are included in the CDF
Figure 3: Outcome data collected on drugs whilst on the CDF
Since 2016, the NHS Cancer Drugs Fund (CDF) has been closely aligned with the
National Institute for Health and Care Excellence (NICE) and is providing access
to new drugs for a limited period only (expected to be up to two years). During this
time, data is collected in order to address the uncertainty that precluded NICE
from issuing a recommendation on adopting the drug for routine use. To our
knowledge, no study has explored the type of drugs included in the CDF and the
extent of data collection under these new rules.
To explore the process and criteria used for consideration of treatments under the
CDF framework, and to describe the extent of evidence collection while in the Fund.
Results
It appears that often drugs on the CDF are being made available for patients with
advanced disease, who have failed prior treatments and are considered to require
end-of-life care. The uncertainty in the evidence seems to largely revolve around over-
all survival and is likely reflected by large discrepancies between ICERs preferred by the
company and the Committee and ERG.
The planned duration of data collection was frequently longer than two years and often
involved continuation of an ongoing trial. This suggests the CDF is enabling patients
to receive treatments that may otherwise not have been available during this time.
Interestingly, the majority of these drugs have received a positive recommendation
for routine use in Scotland.
The current analysis is to our knowledge the first attempt to characterise drugs included
in the CDF under the new rules. However, our results are exploratory, limited by the small
dataset currently available and the reliance on publicly available documents, which often
do not contain all important details.
Discussion
LL,LymphocyticLeukaemia;WM,Waldenstrom’sMacroglobulinaemia;NSCLC,non-small-celllungcancer;IXA,Ixazomib;LEN,lenalidomide;
DEX,dexamethasone;EGFR,epidermalgrowthfactorreceptor;ICER,incrementalcost-effectivenessratio;QALY,qualityadjustedlifeyear
Some of the ICERs were not available in the documents we identified.
OS, overall survival; PFS, progression free survival; QoL, quality of life