Abrams, K

Beutner, E

Deruysscher, D

Grutters, J

Joore, MA

Lambin, P

Peters, H

Pijls-Johannesma, M

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PCN149 DECIDING UPON NEW AND EXPENSIVE TECHNOLOGIES IN HEALTH CARE: REAL OPTIONS ANALYSIS IN PROTON THERAPY 

A280 13th Euro Abstracts
cancer staging or surveillance in asymptomatic patients. Further studies are needed to 
characterize patients’ typology who deserve intensive staging and follow-up 
procedures.
PCN149
DECIDING UPON NEW AND EXPENSIVE TECHNOLOGIES IN HEALTH 
CARE: REAL OPTIONS ANALYSIS IN PROTON THERAPY
Grutters J1, Abrams K2, Deruysscher D3, Lambin P3, Pijls-Johannesma M3, Beutner E1, 
Peters H1, Joore MA4
1Maastricht University, Maastricht, Limburg, The Netherlands; 2University of Leicester, 
Leicester—UK; 3MAASTRO Clinic, Maastricht, Limburg, The Netherlands; 4Maastricht 
University Medical Center, Maastricht, Limburg, The Netherlands
OBJECTIVES: Radiotherapy with protons is a promising new treatment modality, for 
which adoption decisions are being made worldwide. However, the investment costs 
of proton therapy (PT) are high (roughly c90 million) and limited clinical evidence is 
available. Also, previous studies have indicated that PT may be cost-effective, but show 
considerable decision uncertainty. Consequently, it is unclear whether we should 
adopt PT now, or wait for more information. Adoption involves a risk of facing high 
sunk costs, while delay may impose opportunity losses because patients receive sub-
optimal treatment. Real options analysis (ROA), a technique originating from ﬁ nancial 
economics, assists in making this trade-off. METHODS: We examined whether to 
adopt PT, as compared to stereotactic body radiotherapy, in the treatment of stage I 
non-small cell lung cancer (NSCLC). Three options are available: adopt without 
further research (AN); adopt and undertake a trial (AT); or delay and undertake a 
trial (DT). The decision depends on the expected net gain of each option, which is 
calculated by subtracting its total costs from its expected beneﬁ ts. RESULTS: The 
expected net gain of at and DT were positive, indicating that we should not decide to 
adopt without further research (AN). Up to a sample size of 1000 patients, the 
expected net gain of at was higher than DT, indicating that the best option was to 
adopt and trial. The expected net gain of at was highest for a sample size of 450 
patients, which is thus considered the optimal sample size. CONCLUSIONS: Based 
on these results, we recommend to adopt PT in the treatment of stage I NSCLC, and 
to perform a trial with 450 patients. We have shown that ROA provides a transparent 
method of weighing the costs and beneﬁ ts of all available options, to assist in decision-
making upon new and expensive technologies.
PCN150
SYSTEMATIC LITERATURE REVIEW ON THE INTER AND INTRA 
LABORATORY VARIABILITY OF MOLECULAR TESTING OF RESPONSE 
TO TREATMENT OF CHRONIC MYELOID LEUKEMIA (CML) PATIENTS 
AND THE ASSOCIATED COSTS AND COST-EFFECTIVENESS
Ratcliffe M1, Hudson PE1, Ossa D2
1Phmr Consulting, London, UK; 2Novartis Pharma AG, Basel, Switzerland
OBJECTIVES: During disease monitoring of patients with CML, for patients with a 
complete response, residual leukemia can be assessed by real-time quantitative 
polymerase chain reaction (RQ-PCR). There are several “home-brew” and commer-
cially available BCR-ABL gene transcript detection methodologies in use, each 
requiring internal validation for the speciﬁ c laboratory and giving rise to laboratory-
speciﬁ c data. Harmonization of results according to an international scale is under-
way, but use is limited for several technical reasons. Information is required for 
decision makers on the accuracy and reproducibility of the tests and their costs 
and cost-effectiveness. The objective of this study was to assess the quantity and 
quality of such information. METHODS: English language systematic literature 
review on the intra- and inter-laboratory variability for BCR-ABL molecular monitor-
ing testing, inter-rater reliability across manual assays and the costs and cost-effec-
tiveness of molecular testing in CML. RESULTS: From 88 papers retrieved for detailed 
analysis, we found no studies which conducted a repeated test procedure on the same 
patient sample using the same technical approach in the same laboratory. There are 
a large number of studies which have compared alternative approaches using the same 
patient sample in molecular monitoring in the same laboratory. Several well-conducted 
studies have examined the variability of results from different laboratories in con-
trolled environments. We found no studies which compared inter-rater reliability or 
examined the costs or cost-effectiveness of molecular testing in CML. CONCLU-
SIONS: There is a reasonable body of evidence on certain aspects of analytical validity 
for CML molecular testing, but other aspects of analytical validity and the costs and 
economics of molecular diagnostics in CML appear to be an unresearched area. 
Testing variability has potentially serious implications for patient outcomes and more 
information for decision-makers to assess relative costs and cost-effectiveness is 
required.
PCN151
TARGETED CANCER THERAPIES: PRICING, ACCESS, AND UPTAKE
Aggarwal S1, Stevens CA2
1PAREXEL Consulting, Bethesda, MD, USA; 2PAREXEL Consulting, Waltham, MA, USA
OBJECTIVES: The oncology market has become one of the major focus areas for 
pharmaceutical and biotech ﬁ rms. As of March 2009, 15,752 of 39,747 Phase I, II, 
and III trials listed on clinicaltrials.gov were related to cancer (approximately 40%). 
This large interest in oncology stems from market success of cancer therapies launched 
in the past decade and the existence of high unmet need to treat different types of 
cancers. As the number of FDA approved cancer therapies increases, there is a need 
to understand treatment patterns of these cancer drugs. METHODS: To understand 
the trends in usage and sales of cancer therapies, we analyzed the US market (sales 
and prescription) 2005–2008 data for all FDA-approved cancer drugs. Drugs were 
categorized as targeted cancer therapies, chemotherapies, monoclonal antibodies, 
small molecules, branded, and generics. RESULTS: During the past 5 years, the usage 
of both targeted cancer therapies and chemotherapy drugs has increased by high 
double digit rates. From 2005 to 2008, the total prescriptions for targeted cancer 
therapies and chemotherapies increased by 66% and 30%, respectively. While the 
sales of both types of these drugs are expanding, the majority of sales growth is 
attributed to an increasing uptake of targeted cancer drugs. The sales share of targeted 
cancer therapies in the US oncology market increased from 36% in 2004 to 56% in 
2008. Among targeted cancer therapies, majority (more than 75%) of uptake belongs 
to monoclonal antibodies. CONCLUSIONS: The usage and sales trends show a sig-
niﬁ cant increase in the use of cancer drugs. The high usage of targeted cancer therapies 
versus chemotherapies shows the rapidly changing nature of cancer treatment 
regimen.
PCN153
HOW DO HTA AGENCIES RECOGNIZE AND REWARD INNOVATION? 
CASE STUDIES IN BREAST CANCER AND COLORECTAL CANCER
Shah K1, Mestre-Ferrandiz J1, Towse A1, Nash-Smyth E2, Ball D2, Grainger D3
1Ofﬁ ce of Health Economics, London, UK; 2Eli Lilly & Company, Indianapolis, IN, USA; 3Eli 
Lilly & Company, Sydney, Australia
OBJECTIVES: This paper examines how different payers and health technology 
assessment (HTA) agencies recognize and reward innovation, using treatments for 
breast and colorectal cancer as case studies. METHODS: Breast and colorectal 
cancer were chosen given the extent of clinical advancements to date and supporting 
publicly available data. Sixteen cancer medicines across these two tumor types were 
considered. For each medicine/indication, the reimbursement decision and the reasons 
behind it were obtained from assessment reports published by the respective agencies 
in Australia, Canada, England and Wales, France, and Scotland. RESULTS: Seventy-
seven decisions were reviewed (39 and 38 for breast and colorectal cancer, respec-
tively). Twenty-four (62%) and 16 (42%) were positive for breast and colorectal 
cancer, respectively, while 21% and 45% were negative. In general, HTA agencies 
appear to consider advancements in breast cancer treatments as representing good 
uses of health-care resources with some assessments yielding less positive or more 
restrictive results. The majority of appraisals for newer colorectal cancer treatments 
have failed to receive positive recommendations. CONCLUSIONS: We identiﬁ ed some 
broad areas where differences of approach have led to different decisions. These relate 
to the: 1) acceptability of surrogate end points (e.g., progression-free survival) in cases 
where improvement in overall survival has not been established; 2) extent to which 
agencies formally consider input from clinical and patient representative organizations 
as part of their decision-making process; 3) methods used to assess medicines where 
pivotal trial did not use a comparator reﬂ ecting standard therapy; and 4) mechanisms 
for re-review or adopting performance-based risk-sharing arrangements following 
rejection due to uncertain clinical and/or cost-effectiveness. Addressing these issues 
may improve the likelihood of innovative medicines meeting reimbursement require-
ments, for breast and colorectal cancer as well as other therapeutic areas, thereby 
increasing the overall health beneﬁ t from pharmaceutical development that accrue to 
patients.
PCN154
A COMPARISON OF HTA RECOMMENDATIONS FOR CANCER 
TREATMENT TECHNOLOGIES PUBLISHED BY AGENCY FOR HEALTH 
TECHNOLOGY ASSESSMENT (AHTAPOL) AND NATIONAL INSTITUTE 
FOR HEALTH AND CLINICAL EXCELLENCE (NICE)
Kiljan A, Kolasa K, Hermanowski T
Medical University of Warsaw, Warsaw, Poland
OBJECTIVES: The objective of this study was to compare HTA recommendations 
for cancer drug technologies issued by both Agency for Health Technology 
Assessment (AHTAPol) in Poland and National Institute for Health and Clinical 
Excellence (NICE) in the UK. METHODS: The review of HTA recommendations 
concerning cancer technologies published online in the period August 2007–June 2010 
(AHTAPol) and March 2000–June 2010 (NICE) was performed. The classiﬁ cation of 
HTA recommendations based on Raftery’s approach labeling them as positive, posi-
tive with major or minor restriction, and negative was conducted. Negative guidance 
was categorized as clinical or nonclinical. Reasons for HTA recommendations for drug 
technologies appraised by both AHTAPoL and NICE were compared. Contradictory 
and noncontradictory recommendations were identiﬁ ed as well. RESULTS: A total of 
149 drug technologies were appraised by AHTAPol, of which 39 concerned cancer 
technologies (seven resubmissions). NICE published 136 drug appraisals, of which 59 
related to cancer technologies (12 resubmissions). In total, 12 cancer drug technologies 
were appraised by both AHTAPoL and NICE. Among them, there were nine contra-
dictory and three noncontradictory pairs of guidance (two negative and one positive). 
In the group of drug technologies appraised by both agencies, there were 42% and 
67% positive HTA recommendations issued by AHTAPoL and NICE, respectively. 
Negative recommendations based on nonclinical reasons prevailed in Poland (58%). 
At the same time, there were as many positive recommendations with major restric-
tions (33%) as negative recommendations based on nonclinical reason (33%) in the 
UK. The positive guidance without restriction constituted 8% and 17% of all apprais-
als for cancer drug technologies published by AHTAPol and NICE, respectively. 


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PCN149 DECIDING UPON NEW AND EXPENSIVE TECHNOLOGIES IN HEALTH CARE: REAL OPTIONS ANALYSIS IN PROTON THERAPY

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