Budget impact is the main driver for negative recommendations issued by ANM in Romania, with half of rejected cases due to high budget impact. Improved budget impact methodology is needed to differentiate high cost products from those lacking price comparators. ANM rejected nearly half of products due to high perceived budget impact despite no calculations, suggesting a need for distinct assessment criteria for products without comparators and consideration of long-term costs.

1. Conclusions
Budget impact is indeed a main driver for negative recommendations issued by ANM, suggesting Romania is more budget impact-sensitive compared to other markets. The study demonstrated that in half of the cases,
ANM was not able to choose an appropriate price comparator for NMEs; hence these were also rejected on the basis of budget impact. Therefore, improved robustness in the budget impact methodology is required in
order to differentiate those high-budget impact products from those simply lacking a price comparator.
We suggest a distinct scoring system for drugs with no available comparator and/or having a distinct category for such drugs. In addition, the relevant authorities may also ensure consistency between the choice of comparators
by taking the comparators from other countries as a reference. Furthermore, Romania may consider the costs of patient management, including non-drug therapies, as a cost reference for future budget impact analysis.
For additional information, please contact: cornelia.roibu@gfk.com © GfK 2016
Health Technology Assessments (HTAs) are comprehensive
evaluations incorporating a range of parameters, such as the social,
economic and ethical issues of a medicine, medical device or clinical
procedure. They are intended to provide support for key decision-makers
with regard to the appropriate use of a new technology, their impact
and efficient allocation of national or regional healthcare resources.1
The Romanian HTA system is rather new, having been formally
initiated as a fully operational unit within the Ministry of Health (MoH)
in 2013 and subsequently revised in 2014.2,3
The latest HTA process
is the responsibility of the Romanian National Medicines Agency
(ANM). As of 2016, the HTA process called quick-HTA, is based
on a scorecard system which determines inclusion of a medicine
in the reimbursement list.
The evaluation criteria for a New Molecular Entity
(NME) include:4
▪▪ Decision from Haute Autorité de Santé (HAS), the National
Institute for Health and Care Excellence (NICE), the
Institute for Quality and Efficiency in Healthcare (IQWiG)
and/or the Federal Joint Committee (G-BA)
▪▪ maximum number of points (30) granted, if all three
bodies issue a positive assessment
▪▪ Number of European Union (EU) countries that
granted reimbursement
▪▪ maximum number of points (25) granted if at least
14 of 27 EU countries granted reimbursement
▪▪ Real-world evidence
▪▪ maximum number of points (45) granted if the
manufacturer provides collected real-world evidence
for a period of at least one year
▪▪ Budget impact in Romania
▪▪ maximum number of points (30) granted if the annual cost
of the new therapy is at least 5% less expensive compared
to the annual cost of treatment with the comparator
Three outcomes are possible based on the number
of points:
▪▪ Unconditional inclusion – the new therapy obtains a
score > 80 points
▪▪ Conditional inclusion – the new therapy obtains a score
between 60 and 79 points
▪▪ Exclusion from the reimbursement list – the new therapy
obtains a score <60 points
The present analysis will examine the relationship between the
evaluation criteria and HTA outcomes and will discuss the main
reason for HTA rejection of oncology drugs in Romania.
▪▪ Four assessments were given a positive recommendation in
Romania, but not by NICE, albeit having positive outcomes
from HAS and IQWiG/G-BA.
▪▪ 10 assessments were following the recommendation of at
least one of the HTA bodies, classified as “other”.
▪▪ Detailed analysis of all 15 negative assessments revealed underlying
factors. As seen in Table 3, only 20% of assessments follow the
recommendation given by NICE, whereas 100% of negative
assessments are in discord with the HAS positive recommendation.
▪▪ As shown in Figure 2, out of the 15 negative assessments, eight
assessments, which equates to 53%, were due to high-budget
impact that was numerically validated.
▪▪ As seen in Table 2, out of 37 oncology product assessments
published between January 2014 and June 2016, 15 received a
negative recommendation by the ANM, albeit receiving a positive
recommendation by at least one of the three national bodies:
HAS, NICE and/or IQWiG/G-BA.
▪▪ Figure 1 shows the ANM oncology product assessments
separated by outcome. Out of the 22 positive assessments:
▪▪ Eight assessments follow the recommendations of the other
three HTA bodies (HAS, NICE, and/or IQWiG/G-BA).
▪▪ From all of the ANM oncology assessments since 2014, 40% have
been given a negative recommendation by ANM, and therefore
reimbursement was rejected in Romania.
▪▪ Out of these negative assessments, 53% were rejected on the basis
of proven high-budget impact, suggesting this is indeed an important
factor in the quick-HTA system.
▪▪ Nearly half of the negative recommendations (47%) were not given
any points for the “budget impact” category. As such, these were
considered to be of high-budget impact, even though no calculations
were performed due to the lack of a suitable price comparator.
Background
Results
Results - Overall
Objectives
Romania is known to have one of the lowest indexes for health
expenditure as percentage of gross domestic product (GDP) among
EU countries.5
As such, we hypothesized that reimbursement for a
new medicine may strongly be dependent on the budget impact this
imposes on the country. This research examines whether budget
impact is the main reason for rejection of oncology drugs in Romania.
▪▪ HTA assessments of oncology drugs by the ANM were examined
since the latest system was set in place, i.e., during the period
January 2014 - June 2016.
▪▪ As illustrated in Table 1, 37 oncology assessments were
published during this timeframe.
▪▪ The 37 oncology assessments were analyzed by outcome
positive and negative to assess the proportion of negative
recommendations granted by the ANM.
▪▪ The reason for the negative ANM assessment was divided into
three criteria: HTA outcomes, reimbursement in other countries
and budget impact.
▪▪ The negative assessments that were rejected on the basis of the
“budget impact” category were further analyzed and subdivided
into two new categories: calculated high-budget impact and
choice of comparator.
Methods
1. WHO Health Technology Assessment. Available at: http://www.who.int/medical_devices/assessment/en/. Accessed August 30, 2016.
2. Radu C-P, Pana P. Key aspects regarding the introduction of health technology assessment in Romania. Management in health XVII/2/2013;
pp. 4-7.
3. Romanian Quick-HTA Development in 2013. Accessed August 30, 2016.
4. Law Nr.861 valid from 23.07.2014 Ministry of Health. http://www.anm.ro/anmdm/_/ORDINE/OMS%20861_2014.pdf. Accessed August 30,
2016.
5. The World Bank. World Health Organization Global Health Expenditure database. Available here: http://data.worldbank.org/indicator/SH.XPD.
PUBL.ZS. Accessed September 1, 2016.
Year of ANM assessment Total of oncology product assessments
2014 26
2015 8
2016 3
Total 37
Drug name ANM decision
abiraterone Rejected
abiraterone Recommended
aflibercept Recommended
axitinib Recommended
azacitidine Recommended
azacitidine Recommended
bendamustine Rejected
bendamustine Recommended
bevacizumab Rejected
bosutinib Recommended
brentuximab Recommended
cabazitaxel Rejected
crizotinib Recommended
dabrafenib Recommended
degarelix Rejected
dutasteride + tamsulosin Recommended
enzalutamide Recommended
everolimus Rejected
everolimus Rejected
Drugs that received
a negative assessment
by ANM
HAS assessment NICE assessment
IQWiG/G-BA
assessment
Reimbursement
in other
countries
ANM high budget
impact
abiraterone Yes recommended Not NICE-assessed Yes recommended
Only 12
countries
No comparator is
available
bendamustine Yes recommended Yes recommended Not assessed Max points Yes
bevacizumab Yes recommended
No not recommended
by NICE
Not assessed Max points Yes
cabazitaxel Yes recommended
No not recommended
by NICE
Yes recommended Max points Yes
degarelix Yes recommended
Not NICE assessed,
but SMC recommends
Not assessed Max points Yes
everolimus Yes recommended
No not recommended
by NICE
Not assessed Max points No comparator
everolimus Yes recommended Not NICE-assessed Not assessed Max points
No comparator
was suggested
fotemustine Yes recommended Not NICE-assessed Not assessed Only 9 countries Yes
gefitnib Yes recommended Yes recommended Not assessed Max points Yes
lapatinib Yes recommended Not NICE-assessed Not assessed
Only 12
countries
Choice of
comparator
not considered
appropriate
regorafenib Yes recommended Not NICE-assessed Yes recommended Max points
Choice of
comparator
not considered
appropriate
silodosin Yes recommended Not NICE-assessed Not assessed Max points Yes
trastuzumab Yes recommended Yes recommended Not assessed Max points Yes
ulipristal acetate Yes recommended Not NICE-assessed Not assessed Max points
No comparator is
available
vismodegib Yes recommended Not NICE-assessed Yes recommended Only 9 countries
No comparator is
available
ANM assessments with a calculated negative budget impact Y
ANM assessments with lack of a suitable comparator N
Drug name ANM decision
everolimus Recommended
fotemustine Rejected
gefitinib Rejected
ibrutinib Recommended
ibrutinib Recommended
ipilimumab Recommended
lapatinib Rejected
olaparib Recommended
panitumumab Recommended
pazopanib Recommended
pazopanib Recommended
regorafenib Rejected
silodosin Rejected
sorafenib Recommended
trastuzumab Rejected
ulipristal acetate Rejected
vemurafenib Recommended
vismodegib Rejected
Positive ANM assessments Recommended
Negative ANM assessments Rejected
Table 1: Number of oncology product assessments by ANM (2014 to 2016)
Table 2: ANM decisions by oncology product
Table 3: Details on the negative ANM assessments by criteria
Figure 1: ANM assessments by
relative outcome
Figure 2: Justifications for the ANM budget
impact category among rejected drugs
15 8
4
10
22
Negative
assessments
ANM outcome
consistent with
other HTA
bodies outcome
Positive ANM
outcome but
negative NICE
recommendation
Other
Negative
assessments Positive
assessments
53%
47%
Calculated
high-budget
impact
Choice of
comparator
Budget impact as a main reason for HTA
rejection of oncology drugs in Romania
Cornelia Roibu, Laurent Pacheco (GfK, London, UK)
Value in Health, 2016; Vol 19, Issue 7 – Presentation code: PCN279
ISPOR 19th Annual European Congress | October 29-November 2, 2016 | Austria Center Vienna, Vienna, Austria