Friedmann, B.

Nijhuis, T.

Schuchardt, M.

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 VA L U E  I N  H E A LT H  1 6  ( 2 0 1 3 )  A 3 2 3 – A 6 3 6  A429
and a combination of tegafur, gimeracil and oteracil. Across these oncology assess-
ments a total of 19 patient subgroups have been evaluated. Eleven subgroups (58%) 
showed an additional benefit according to the G-BA. Eight subgroups (42%) received 
the rating “no additional benefit” or “less benefit than comparator”. The compara-
tors chosen by G-BA within subgroups vary widely depending on the indication. 
Key factors for the positive outcome of these assessments were increased overall 
survival, reduction of symptoms or improved quality of life. Main reasons for the 
G-BA to attest no additional benefit include inappropriate indirect comparison and 
lack of adequate patient subgroup analysis. ConClusions: Analysis of HTA reports 
in oncology shows that while overall survival is a strong end point, also increased 
quality of life and reduced side-effects can be sufficient to achieve a beneficial 
outcome (crizotinib: considerable benefit). Importantly, the provided data must be 
applicable to the German regulations under AMNOG, showing clinical evidence 
against the specified comparator. The amount of the additional benefit plays an 
important role in the reimbursement amount negotiations following the definition 
of the additional benefit by the G-BA.
PCN211
HealtH teCHNology assessmeNt: Is It tHe RIgHt PIeCe foR tHe 
JoRdaNIaN HealtH CaRe Puzzle?
Al Rabayah A.A., Jaddoua S.
King Hussien Cancer Center, Amman, AL, Jordan
objeCtives: To study the pharmaceutical reimbursement/Coverage decision 
making processes in Jordan to highlight the importance of conducting formalized 
technology assessments Methods: To review publically available data regarding 
the reimbursement/Coverage decision making processes in Jordan through search-
ing related organization’s websites and publically available regulations. Results: 
Jordan is characterized with a fragmented health care system. Pharmaceutical 
registration and pricing are under the responsibility of the JFDA. Furthermore, it 
is responsible about medication selection for the Rational Drug List (the national 
formulary). The medication supply chain differs between the public and the private 
sectors in term of process and out puts. The medication selection process is not 
governed by criteria and not empowered by an independent review body to support 
decision making by the national appraisal committee (national P&T). The rational 
drug list is publically available but without details of the decision or the processes 
of decision making process. Listing of new medication is wide without indication 
specification or date of listing. The role of cost-effectiveness is limited and the ten-
der prices are not linked to any type of cost effectiveness analysis ConClusions: 
The National Agenda, the National health Policy and the National Drug Policy 
tackled the high health expenditure in Jordan as an essential priority. This chal-
lenged is due to the characteristics of the Jordanian health care system that is 
fragmented with a divided funding system between public and private sectors. A 
more formalized medication selection processes empowered with drug information 
services that provide evidence based data and analysis in the form of technology 
assessment would play a role in decreasing health care expenditures. All of these 
recommendations should move parallel with improving the level of transparency 
and patient engagement.
PCN214
ePIdemIology foR oNCologICal dRugs RegaRdINg tHe BeNefIt dossIeR 
PRePaRatIoN IN geRmaNy
Kürschner N., Schmitter S.
Pfizer Deutschland GmbH, Berlin, Germany
objeCtives: An early benefit assessment of drugs after launch has been imple-
mented since 2011 in Germany. The Institute for Quality and Efficiency in Health Care 
(IQWiG) assesses the benefit of the drug based on a dossier submitted by the phar-
maceutical manufacturer. Based on this assessment and the statements by industry, 
scientific community and patient organizations the Federal Joint Committee (G-BA) 
reviews and decides on the extent of the additional benefit. The dossier needs also 
to contain information about the number of patients treated with the new drug. The 
objective is to investigate the sources considering the calculation of patient numbers 
for oncological drugs. Methods: A review of oncological drugs which passed through 
the benefit assessment was conducted to evaluate which data sources and methods 
were used to calculate the potential patient number. The results were compared with 
IQWiG’s assessment and the final decision by G-BA, to detect possible methodologi-
cal difficulties. Results: The data sources regarding German epidemiological data 
were mainly collected through publicly available sources such as national and local 
cancer registries. Difficulties occurred with small cancer entities or when specific 
data regarding patient subpopulations (e.g. through age, tumor stages, ECOG perfor-
mance status or previous therapies) was needed. The pharmaceutical manufacturer’s 
calculations were often challenged by IQWiG and G-BA without suggesting a precise 
alternative or more suited data source. ConClusions: The data collection and data 
availability within the benefit dossier process for oncological drugs is in most cases 
challenging and the efforts needed should not be underestimated. Authorities, indus-
try and medical community should work on a common solution for a more valid and 
reliable calculation of the potential patient number in oncology.
PCN215
eCoNomICal losses due to dIsaBlemeNtPaReNts CaRRINg foR CHIldReN 
wItH oNCoHematologICal dIseases
Ganieva D.1, Zhukovskaya E.1, Spichak I.2
1Federal Scientific Centre of Children Hematology, Oncology, Immunologyl, Moskow, Russia, 
2Medical Academy, Chelyabinsk, Russia
objeCtives: Socio-economic phenomena, caused by disease of children are reflected 
primarily in the formation of non-medical costs. Methodology for calculation of non-
medical costs includes a number of parameters, including the cost of lost output 
by persons caring for children during the treatment period. Methods: The study 
involved patients from Oncohematological Chelyabinsk Center for Children and 
Adolescents behalf of prof. V. Gerein been treated in the period from 2008 to 2013. 
10, respectively). Similarly, patients admitted to hospital with oesophageal cancer 
experienced a high 90-day mortality rate, ranging from 22% to 21.9% in 2007-08 and 
2009-10, respectively. However, between 2006 and 2010, no therapies were submitted 
for NICE appraisal for oesophageal cancer, suggesting that there may have been a 
lack of research interest and potentially explaining why there was no substantial 
decrease in mortality from 2007, as compared to indications where therapies had 
been approved, such as lung, colon and breast cancer. ConClusions: The recom-
mendation of therapies and their uptake in the UK may at least partially explain the 
trends noted in this study, although other factors such as delay in therapy uptake 
and off-label use may also need to be taken into account.
PCN208
do NICe evIdeNCe RevIew gRouPs (eRg) foCus oN dIffeReNt asPeCts of 
maNufaCtuReR suBmIssIoNs IN oNCology?
Heemstra L., Sweeney N., Van Engen A.
Quintiles, Hoofddorp, The Netherlands
objeCtives: Evidence Review Groups (ERGs) provide a critical appraisal of the manu-
facturer submission in the NICE single technology appraisal (STA) process. As the aca-
demic centres may differ in experience and methodology, the objective of this study was 
to evaluate whether the focus areas and key criticisms differ between ERGs. Methods: 
The NICE website was searched for all NICE oncology STAs, published between June 
2010 and June 2013. The ERG reports were retrieved, and the main critiques were cat-
egorised for the five centres that performed the most evaluations. The focus areas of the 
ERGs were further studied. Results: A total of 27 STAs were identified with evaluations 
performed by 9 different ERGs. The most evaluations were performed by Liverpool (9), 
followed by Sheffield (4), and PenTAG, West Midlands and York (3 evaluations each). All 
ERGs would report uncertainties related to the extrapolation and gain in overall survival 
(OS), maturity of data, trial comparator, and the quality of life (QoL) data. In addition all 
critiques covered submission quality and disease specific challenges, yet variation was 
found in focus area between ERGs. For example a specific focus area of Liverpool was 
the OS modelling method. Proposed changes to survival modelling included separating 
the survival curves for pre- and post-progression, and removing any survival advan-
tage post-progression where this was considered inappropriate. Comments from other 
agencies on OS were mainly limited to the choice of parametric survival function. Other 
areas that differed between ERGs were the systematic review methods (more often 
reported by Sheffield) and comments on the QoL data (York). ConClusions: Although 
all ERGs focus on uncertainty around the evidence and quality of the manufacturer 
submissions, the focus areas differed between the groups. The key difference seems 
to relate to research focus of the academic centre.
PCN209
RevIew of NICe teCHNology aPPRaIsals IN oNCology: How does 
ClINICal evIdeNCe CHaNge oveR tIme?
Dequen P., Cooper N.J., Abrams K.R.
University of Leicester, Leicester, UK
Drug licensing and reimbursement authorities worldwide are considering new 
ways to stimulate market access for innovative medicines such as accelerated 
approval and conditional coverage. Early release of pharmaceuticals calls for more 
responsive decision-making alongside continuous evidence generation through-
out clinical development. We explore whether changing trends in clinical evidence 
considerations for health technology assessment (HTA) by the National Institute 
for Health and Care Excellence (NICE) may help inform future evidence require-
ments for rapid and early HTA. objeCtives: We investigate how the submission 
and acceptability of clinical evidence for single, multiple and repeated assessments 
of cancer drugs by NICE have changed in the past decade. Methods: We reviewed 
technology appraisals published online since February 2002 by NICE for pharma-
ceuticals in oncology. Information regarding the clinical evidence included and the 
methods used to analyse relative treatment effects across relevant comparators 
was extracted. Manufacturer submissions, assessment reports, and final appraisal 
determinations were considered for longitudinal comparison. Results: Out of a 
total of 254 appraisals identified since 2002, 85 assessed cancer drugs and 76 of 
these were included for review based on available documentation. Only 11 prod-
ucts had been re-assessed to date with initial guidance superseded by a multiple 
technology appraisal or clinical guideline. We found a greater reliance on phase 
II and observational data in recent appraisals, particularly for novel therapies in 
areas of high unmet need. Limited data was also accompanied by an increase use 
of surrogate outcomes and extrapolation of observed short-term clinical benefits. 
Recent submissions were also marked by the uptake of network meta-analysis 
methodologies. ConClusions: NICE has previously recommended cancer drugs 
based on immature clinical data allowing for considerable uncertainty in ‘real-
world’ effectiveness estimates. However, these examples remain the exception to 
the rule; moreover our review highlighted a need for methodological development 
to deal with early clinical evidence.
PCN210
g-Ba assessmeNts of oNCology tRIals: Is INCReased oveRall suRvIval 
a “must Have”?
Schuchardt M.1, Nijhuis T.1, Friedmann B.2
1Quintiles, Hoofddorp, The Netherlands, 2Quintiles, Mannheim, Germany
objeCtives: Objective of this research was to provide an overview of Health 
Technology Assessments (HTAs) in oncology after introduction of AMNOG in 
Germany. Methods: Quintiles’ HTA database (HTA Watch) has been used to ana-
lyse HTAs in Germany. The timeframe chosen for analysis was 1st of January 2011 
to 24th of June 2013. All reports have been analysed in detail to reveal key factors for 
success or failure, which are presented in the following. Results: Since introduc-
tion of AMNOG in 2011, thirty percent (13 out of 43) of all completed assessments by 
the G-BA (Federal Joint Committee) evaluated cancer drugs. The products assessed 
were abiraterone acetate, axitinib, brentuximab vedotin, cabazitaxel, crizotinib, 
decitabine, eribulin, ipilimumab, pixantrone, ruxolitinib, vandetanib, vemurafenib 


PCN210 G-Ba Assessments of Oncology Trials: Is Increased Overall Survival a “Must Have”? 

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PCN210 G-Ba Assessments of Oncology Trials: Is Increased Overall Survival a “Must Have”?

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