CLINICAL

EFFECTIVENESS : Santoso Soeroso

REAL-WORLD DATA Member of
(COLLABORATIVE InaHTA
HOSPITAL BASED HTA)
REAL-WORLD DATA

Real-world data (RWD) in

medicine is data derived from a
number of sources that are
associated with outcomes in
heterogenous patient population in
real-world settings such as patient
surveys, clinical trials, and
observational cohort studies
Real-world data are the data relating to
patient health status and/or the delivery of
health care routinely collected from a variety
of sources. RWD can come from a number of
sources, for example:
Electronic health records (EHRs)
Claims and billing activities
Product and disease registries
Patient-generated data including in home-use
settings
Data gathered from other sources that can
inform on health status, such as mobile devices
REAL-WORLD EVIDENCE
Real-world evidence (RWE) is the
clinical evidence regarding the usage
and potential benefits or risks of a
medical product derived from analysis
of RWD. RWE can be generated by
different study designs or analyses,
including but not limited to, randomized
trials, including large simple trials,
pragmatic trials, and observational
studies (prospective and/or
 SCIENTIFIC VS. REAL WORLD
EVIDENCE
Scientific Real world
Objective Can it work? Does it work?
Purpose Regulatory approval Practice
Design RCT Observational
Setting/condition Ideal Real world
Intervention Fixed Flexible
Compliance High Low to high
Internal validity High Variable
External validity Low to medium High
Duration Usually short Long
Number of data Small to medium Big data

http://medcommsnetworking.com/presentations/shaw_070716.
 METHODS IN HTA
Scientific evidence: Secondary data
 Systematic reviews, meta-analyses of clinical aspects: safety, efficacy / effectiveness

Real world evidence: Primary data

 Clinical data and cost for economic analysis: medical records, interviews with
physicians, patients and families, insurance claims, etc
 Epidemiological and observational studies
 Technical studies (functionality, validation)
 Clinical trials
 Expert (and user) opinion, consensus development
COLLABORATIVE
Real-world data (RWD) and real-world evidence
(RWE) are playing an increasing role in health care
decisions. The health care community is using these
data to support coverage decisions and to develop
guidelines and decision support tools for use in
clinical practice.
Medical product developers are using RWD and
RWE to support clinical trial designs (e.g., large
simple trials, pragmatic clinical trials) and
observational studies to generate innovative, new
treatment approaches.
The use of computers, mobile devices,
wearables and other biosensors to gather
and store huge amounts of health-related
data has been rapidly accelerating. This data
holds potential to allow us to better design
and conduct clinical trials and studies in the
health care setting to answer questions
previously though infeasible. In addition,
with the development of sophisticated, new
analytical capabilities, we are better able to
analyze these data and apply the results of
our analyses to medical product
development and approval.
 PATIENT REGISTRY
A patient registry is an organized system that uses
observational study methods to collect uniform data
(clinical and other) to evaluate specified outcomes for a
population defined by a particular disease, condition,
or exposure, and that serves one or more
predetermined scientific, clinical, or policy purposes
There are two major types of cancer registries:
hospital-based registries and population-based
registries. There are two sub-categories under
hospital-based registries: single hospital registry
and collective registry
 IMPORTANCE OF PATIENT
REGRISTRY
Why are patient registries important? ... This is
where patient registries are so powerful,
particularly for rare diseases. A patient registry
simply stores an individual's personal
information and their medical history, and
registries are usually disease-specific.
 90000

80000

70000

60000

50000

40000
2016 2017
30000

20000

10000

0
2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017

2017: + 25057 patients 106000 ESRD Patients

39 % new patients 20000 do not have access to RRT
 CAPD VERSUS HD
HD
Perlu akses vascular
Datang ke RS 2-3x/minggu
Kehilangan waktu bekerja
Mempertahankan fungsi ginjal sisa
(residual renal function)
Perlu hemodinamik yg stabil
Mortalitas 2-3 tahun pertama lebih
tinggi
CPAD
Tidak perlu
Mandiri di rumah/tempat kerja
Tidak kehilangan waktu
Fungsi ginjal sisa cepat hilang
Pilihan untuk pasien dg gangguan
jantung, EF rendah
Mortalitas 2-3 tahun pertama lebih
rendah
DEFINITIONS (WHO,2011)
Health technology: The application of organized knowledge and skills in the form of
devices, medicines, vaccines, procedures and systems developed to solve a health
problem and improve quality of life. It is used interchangeably with health-care
technology.

Medical device: An article, instrument, apparatus or machine that is used in the

prevention, diagnosis or treatment of illness or disease, or for detecting, measuring,
restoring, correcting or modifying the structure or function of the body for some health
purpose. Typically, the purpose of a medical device is not achieved by pharmacological,
immunological or metabolic means.

Medical equipment: Medical devices requiring calibration, maintenance, repair, user

training, and decommissioning − activities usually managed by clinical engineers.
Medical equipment is used for the specific purposes of diagnosis and treatment of
disease or rehabilitation following disease or injury; it can be used either alone or in
combination with any accessory, consumable, or other piece of medical equipment.
Medical equipment excludes implantable, disposable or single-use medical devices.
PENYUSUNAN ROADMAP PENGAWASAN ALAT KESEHATAN DAN PKRT, KEMENKES, 28-29 JULI 2016
HTA
Medical
Procedures

Health
Technology
Assessment

Pharmacy Medical
Devices

PENYUSUNAN ROADMAP PENGAWASAN ALAT KESEHATAN DAN PKRT, KEMENKES, 28-29 JULI 2016
ALTHCARE TECHNOLOGY LIFE CYCLE

PENYUSUNAN ROADMAP PENGAWASAN ALAT KESEHATAN DAN PKRT, KEMENKES, 28-29 JULI 2016
HTA MEDICAL DEVICES

Health technology assessment and diffusion of health technologies

Sumber: WHO, 2011 PENYUSUNAN ROADMAP PENGAWASAN ALAT KESEHATAN DAN PKRT, KEMENKES, 28-29 JULI 2016
 SPECTRUM OF HEALTH TECHNOLOGY
ASSESSMENT

Assessment Evaluative
Activities Research

Literature Meta-
Review Standardised analysis
Evaluation
Analysis
Criteria
Modeling Randomised
Expert
Controlled
Consensus
Trials
Judgement
Primary data
HOSPITAL-BASED HTA ORGANIZATIONAL MODELS
( ADAPTED FROM : HOSPITAL-BASED HEALTH
TECHNOLOGY ASSESMENT WORLD WIDE SURVEY,
ISG HOSPITAL BASED HTA, HTAI 2007 )

Organizational Focus on Clinical practice Focus on Managerial

complexity decision Making

High (Q3) (Q4)

(Team-group-unit) ‘Internal ‘HTA Unit’ model
RS PEND/RS >200 Committee’ Model
TT/ORGANISASI
KESEHATAN

Low (Q1) (Q2)

(Individual) ‘Ambassador’ Model ‘Mini-HTA’ Model
RS KELAS C/D
 AMBASSADOR MODEL
(Q1):
Ambassador Model (Q1): clinicians recognized
as ‘opinion leaders’ play the role of ambassadors
of the HTA “message” inside the HCOs. They
may not take part to
assessments but play a key role in results
diffusion within hospitals. In this quadrant fits
the case of the Swedish ambassadors (Rehnqvist,
2005).
MINI HTA (Q2)
Mini HTA (Q2) : it is the case of
Danish mini-HTA, in which single
professionals participate in the
assessment process collecting data at
organizational level to inform decision
makers at an higher level (Ehlers et al.,
2006).
INTERNAL COMMITTEE
(Q3):
Internal Committee (Q3): evidence is produced
by multidisciplinary groups (called internal
committees) representing different perspectives
and taking the responsibility of reviewing
evidences to issue recommendations useful
hospital-wide. Usually documents are produced
on a peer-to-peer basis by professionals, who
rarely work full time on HTA. The model is
commonly used in HMO in the U.S.A. and in
some Canadian hospitals (Menon & Marshall,
1990)
HTA UNIT (Q4):
HTA Unit (Q4): formal organizational
structure based on specialized HTA
personnel working on a full time basis
inside the Unit. This model represents
the highest degree of structure for
hospital HTA (McGregor & Brobhy,
2005; Catananti et al., 2005).
HTA: ATTRIBUTES / ASPECTS TO BE
ASSESSED

Safety
Efficacy
Effectiveness
Economic
Socio-cultural
Ethical
Legal-Institutional
Religious
TYPES OF ECONOMIC ANALYSIS

Cost-of-illness analysis (CIA): a determination of the economic impact

of an illness, e.g., thalassemia, DM.
Cost-minimization analysis (CMA): a determination of the least costly
among alternative interventions that are assumed to produce equivalent
outcomes.
Cost-effectiveness analysis (CEA): a comparison of costs in monetary
units with outcomes in quantitative non-monetary units, e.g., reduced
mortality or morbidity.
Cost-benefit analysis (CBA): compares costs and benefits, both of which
are quantified in common monetary units.
Cost-utility analysis (CUA): a form of CEA that compares costs in
monetary units with outcomes in terms of their utility, usually to the
patient, measured, e.g., in QALYs
Budget impact analysis (BIA): determine the impact of implementing
HT on designated budget.
COST-UTILITY ANALYSIS

• Berapa penambahan masa dan kualitas hidup?

• Berapa biaya diperlukan untuk menambah 1
QALY?
• Perspektif pemberi layanan
• Perspektif sosietal
• Tiap negara harusnya punya threshold berapa
yang dianggap cost effective. Bagi yang belum
punya, WHO sementara menganjurkan maks 3 x
GDP per kapita
 STUDI BEVASIZUMAB /
KPTK
Scientific evidence: safety, efficacy, effectiveness
Real world evidence:
 Keterbatasan
 Dengan data yang ada, penambahan bevasizumab :
 Menambah PFS 2 bulan (ICER 530 juta/tahun PFS)
 Menambah OS 1 bulan (ICER 1000 juta/tahun OS)
 Cost utility analysis:
 Perspektif pemberi layanan : 837 juta / QALY
 Perspektif sosietal : 890 juta / QALY
(Catatan: Dengan threshold 3 x GDP = 3 x 60 juta =
180 juta. Sangat tidak cost-effective).
 BIA: Penambahan beva memerlukan tambahan dana 15 T untuk 5
tahun ke depan.
 NORSTENT : DRUG ELUTED STENT VS BARE
METAL STENT
NORSTENT compared DES with BMS in a pragmatic, inclusive, non–industrysupported
randomized trial. Of the 2063 patients undergoing percutaneous oronary intervention in Norway
from 2008 to 2011, a total of 12425 met the inclusion criteria, and 9013 (72.5%) were randomly
assigned to receive either DES or BMS. After 6 years of follow-up, there were no significant
between-group differences in the primary composite outcome of death from any cause or
nonfatal spontaneous myocardial infarction, nor were there differences in the individual end
points of myocardial infarction or unstable angina. Measures of quality of life did not differ. The
need for any repeat revascularization was low in both groups. During 6 years of follow-up, any
repeat revascularization occurred in 19.8% of patients in the BMS versus 16.5% of patients in the
DES group (3.3% absolute
risk reduction; P<0.001). The difference was driven, as expected, by less restenosis with
DES. Based on these figures, the number needed to treat with DES to prevent 1 revascularization
is 30.
In summary, during 6 years of follow-up, BMS
delivered the same hard outcomes
and the same quality of life as DES, and, for 96.7%
of the patients, BMS also delivered the same results for
repeat revascularization. NORSTENT thus showed
that the vast majority of patients had the same long-
term results regardless of whether they received BMS
or DES.
The main results of NORSTENT were robust across
subgroups defined by sex, age, medical history, clinical
presentation, and lesion complexity.
 LESSON LEARNED
Dari studi penggunaan obat:
 Perlu review utilisasi sesuai indikasi
 Persyaratan sering tidak terpenuhi # Audit
# Verifikator
 Pemberian obat bervariasi
 Tulisan sulit dibaca
# Electronic medical records
 Rekam medis tidak lengkap
Dari pengalaman negara lain:
 Taiwan: Tidak melakukan HTA pada teknologi yang tidak
dijamin UHC negara-negara maju
 Untuk sementara kami akan melakukan hal yang sama
HTA could potentially impact on Health Technology uptake and
expenditure when realized within hospital setting
HTA Stimulating collaboration and being a useful tool for budget
control and planning

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