HTA-challenges in the Medical Device Industry
HTA-course, Danish Society for Biopharmaceutical Statistics, May 26 2014
Jeppe Sørensen, International Health Economist
Agenda
1. Introduction to Coloplast
2. HTA challenges for medical devices – same or different?
• Product characteristics
• Licensing requirements
3. Country specific approaches to HTAs and medical devices – examples and
implications for evidence
4. How does Coloplast respond to the challenges? Value based argumentation
• “Traditional” cost-effectiveness analysis
• Disease specific PROMs
• Discreet choice experiments
1. INTRODUCTION
Coloplast’s business areas, typical users and products
Ostomy
Care
Continence
Care
Urology
Care
Wound & Skin
Care
People who have had their
intestine redirected to an
opening in the abdominal wall
People in need of bladder
or bowel management
People with dysfunctional
urinary and reproductive
systems
People with difficult-to-heal
wounds
SenSura® Mio
Launched
in 2011
SpeediCath®
Compact Set
Launched in 2012
Altis®
Single Incision sling
Launched in 2012
New Biatain®
Silicone
Launched in 2013
1. INTRODUCTION
Business areas 2012/13
Ostomy Care
#1
35-40%
global
position
global market
share
42%
4.8
of Coloplast
revenue
billion DKK
annual revenue
7%
Innovative solutions
organic growth
rate
1. INTRODUCTION
Business areas 2012/13
Continence Care
#1
40-45%
global
position
global market
share
35%
4.1
of Coloplast
revenue
billion DKK
annual revenue
7%
Award winning Products
organic growth
rate
2. MEDICAL DEVICES – SAME OR DIFFERENT?
Product characteristics of medical devices influence
conditions for establishing evidence
Reasons why devices are different in relation to RCTs
1. Difficult/impossible to do blinded studies with devices
2. No “steady state” period: Frequent product modifications and
3. Device-Operator Interactions: Efficacy depends on how it is used and RCT
risks demonstrating experience rather than differences
Source: Drummond, M., Griffin, A. and Tarricone, R. (2009), Economic Evaluation for Devices
and Drugs—Same or Different?. Value in Health, 12: 402–404
2. MEDICAL DEVICES – SAME OR DIFFERENT?
Different licensing requirements compared to drugs mean
different conditions for producing evidence
Pharma
• 10-15 years development and clinical trials aiming at a strong regulatory file for
FDA/EMA approval
Medical devices
• Often a CE-mark is the only licensing requirement for disposable medical
devices
• 2-3 years from idea to market
• Fewer and smaller trials
2. MEDICAL DEVICES – SAME OR DIFFERENT?
Example: Evidence on hydrophilic coated vs. uncoated
catheters and occurrence of UTIs
Number of
patients
Institutional data is the best
measure for a difference in UTI rates,
however it overestimates the real life UTI
rates.
UTI / yr
Uncoated
HCIC
Uncoated
HCIC
De Ridder 2005
61
60
6.60
5.28
Cardenas 2011
114
105
8.18
6.47
UTI reduction
21 %
Number of
patients
Patients UTI rates in community is the
most relevant measure for real life picture
of the UTI rate.
Cardenas 2009
UTI reduction
UTI / yr
Uncoated
HCIC
Uncoated
HCIC
23
22
1.65
0.77
53 %
3. APPROACHES TO HTA
Very different funding systems and HTA-requirements – two
examples
3. APPROACHES TO HTA
Example: France – reimbursement system & characteristics
Community Reimbursement system
HAS: The French National Authority for Health
evaluates reimbursement in 2 steps (outside category)
1st step: Medical evaluation
2nd step: Economic evaluation
TECHNICAL & MEDICAL EVALUATION
ECONOMICAL EVALUATION
Committee 1 (CEPP/ CNEDiMTS)
Committee 2 (CEPS)
Key characteristics
• National categories with fixed prices:
 OC: 17 sub-categories
 IC: 3 sub-categories (CD: 8 sub-categories)
 WC: sub-categories based on size
 Application time in category: < 1 week
(only need safety registration in Afssaps)
 MEDICAL SERVICE : YES/ NO
 MEDICAL SERVICE IMPROVEMENT (MSI):

 REIMBURSEMENT PRICE -> LPPR
5 grades
I.
II.
III.
IV.
V.
Major improvement
Important improvement
Moderate improvement
Minor improvement
No improvement
Decision-makers = physicians

Spotlight clinical data
→ high price
→ premium price
→ price level ?
→ parity/ low price
→ No reimbursement / low price
Cost-minimisation politic for
Healthcare

Spotlight budget impact and
EU prices/ reimbursement
Clinical data not required
 No international reference pricing
 Review every 5th year (longer in reality)
• Brand specific reimbursement possible
 Application time: 6 – 12+ months
 Clinical data required
 International reference pricing
 Review every 5th year (incl reference price)
• Co-payment: none for chronic care (OC/ CC)
 35% non Chronics (but 90% insured)
3. APPROACHES TO HTA
The Coloplast payer landscape – differs across markets
?
Reimbursement/
Value based
Reimbursement/ Procurement/ 100 %CoFixed categories
Tenders
payment
Pricing / feature based
Performance/value based
Page 11
4. VALUE BASED ARGUMENTATION
Example on how health economics is used for value
argumentation in relation to intermittent catheterization
Published article show that by using a
hydrophilic coated catheter, a UTI
reduction of 21 % can be obtained
Urologist and rehab
specialist panel
Health economic
analysis
Findings
Hydrophilic coated catheters are
4% more expensive
than uncoated catheters
HCIC increase QALY by 5%,
increase additional life years by
Consolidated
possible
adverse events
Page 12
4% and decrease the risk of UTI
by 16 % with a lifetime
perspective
Chronic Urinary Retention – HCIC vs. uncoated
No/minor renal impairment
No UTI
Major renal impairment
UTI responding to
initial treatment
No UTI
UTI not responding
to initial treatment
No UTI
UTI responding to
initial treatment
UTI not responding
to initial treatment
No presence of treatment-requiring urinary tract
infections
Presence of treatment-requiring urinary tract infections
that responds well to initial treatment - including 7%
multiple drug resistance.
• Monthly risk at IC: 32,6% (Cindolo 2004, Cardenas 2009 +
2011, De Ridder 2005, Giannantoni 2001, Duffy 1995 & King
1992)
• Risk reductions HCIC vs. uncoated: 10% (meta analysis)
21% (Cardenas 2011 – controlled part), 53% (Cardenas 2009)
UTI not responding
to initial treatment
UTI responding to
initial treatment
Including cases leading to epididymitis, pyelonephritis and
urosepsis.
• Monthly risk: 0,320% (Chai 1995, Perrouin-Verbe 1995, Weld
No/minor renal
impairment
Major renal
impairment
Chronic kidney
failure
2000)
• Risk reduction HCIC vs. uncoated: 10% (Expert assumption)
Background
adverse events
Bladder stones
Kidney stones
Urethral damage
Page 13
0.117%
0.117%
0.189%
(Perrouin-Verbe 1995, Chai 1995)
(assumed the same as bladder stones)
(Perrouin-Verbe 1995, Chai 1995 and Weld 2000)
Chronic kidney failure
No UTI
UTI responding to
initial treatment
UTI not responding
to initial treatment
No or minor renal impairment requiring dietary changes
only
Careful monitoring is needed.
• Monthly risk : 0,020% (1/4 of upper tract abnormalities, Weld 2000)
Dialysis or renal replacement therapy needed.
• Monthly risk : 0,0035% (UK renal registry, Lawrenson 2001)
4. VALUE BASED ARGUMENTATION
Full overview of the CEM CUR Model
Data input
Modelling
No/minor renal
impairment
Major renal
impairment
Output
Chronic kidney
failure
Results
Urinary Tract Infections
No UTI
UTI responding to
initial treatment
Adverse Events
Cost Data
Page 14
UTI not responding
to initial treatment
4. VALUE BASED ARGUMENTATION
Output
Data input
Modelling
Output
Mean cost
Mean QALY
Mean LYG
UTIs
Adverse Events
Page 15
ICER
Cost-effectiveness ratio
depends on local costing data
4. VALUE BASED ARGUMENTATION
Limitations in conventional CUAs in relation to effects of
medical devices – defining and measuring QOL
Page 16
4. VALUE BASED ARGUMENTATION
Example: SpeediCath Compact and quality of life
QoL
Intermittent Self-Catherisation Questionnaire (ISC-Q)
HRQoL
SF-36
EQ-5D
• Validated instrument accepted for publication (Pinder 2013)
• Total score 0-100 based on 24 questions in 4 domains:
• Ease of use, convenience, discreteness and psychosocial wellbeing
Disease QoL
Qualiveen
Compact trial results on quality of life (ISC-Q)
C-IQoL
Compact: 17 point improvement out of 100
ISC-Q
Randomised controlled trial of 118 neurogenic users from 5 countries using
Compact 6 weeks vs. non-Compact for 6 weeks (Chartier-Kastler 2013).
But what is better quality of life worth ?
Page 17
4. VALUE BASED ARGUMENTATION
One way is to base willingness to pay on a discreet choice
experiment
Attribute
Catheter A
Catheter B
Apply gel to the catheter to lubricate it
The catheter is pre-coated with lubricant
Convenience
Catheter coating
and needs no preparation
Risk of infection
No urinary tract infections
You will have 1 urinary tract infection
Ease of insertion
Moderately easy to insert
Difficult to insert
£XX per month
£XX per month


Willingness to pay
Which catheter do you prefer?
Page 18
4. VALUE BASED ARGUMENTATION
Example of WTP results for specific catheter attributes
• WTP puts a value on the
benefit of certain features to
the users
• Can be used to link an
improvement in quality of life to
monetary units
• In Coloplast WTP is used to
describe user-perceived value
of various aspects (i.e. coating,
risk of infection, convenience
etc.)
• A regular cost-minimisation
analysis only counts costs –
WTP counts the value for the
users
Page 19
Attribute
Level
£/Cath.
Convenience
Small and compact.
Moderately sized.
Not compact and can be bulky.
XX
XX
-
Catheter
coating
Pre-coated, needs no preparation.
Add water and wait ~30 seconds.
Apply gel.
XX
XX
-
Risk of
infection
UTI next 12 months:
No urinary tract infections
1 urinary tract infection
3 urinary tract infections
XX
XX
-
Ease of
insertion
Easy
Moderately easy
Difficult
XX
XX
-
4. VALUE BASED ARGUMENTATION
Existence of evidence within the field of stoma care is very limited
Stoma appliance trials registered at ClinicalTrials.gov
Details:
Sponsor
Trials
RCT
Blinded
Finished
Coloplast
15
13
0
14
Subjects in trials: <6,000 in total – 2 trials accounts
for ~5,000
ConvaTec
7
1
0
5
25 trials registered – 15 by Coloplast
Hollister
2
0
0
2
15 RCTs registered – 13 by Coloplast
Independent
1
1
0
1
Total trials
25
15
0
22
0 blinded trials registered – Not possible to blind
stoma appliance trials
International perspective on evidence gap
Use of Willingness-to-pay study
Use of less valid study designs
The DialogueStudy
- >3,000 subjects included
- Improved Quality of Life
Combining willingness-to-pay study with costminimisation analysis (niklas.hedberg@tlv.se)
10 May 2013
Assessment of SAS premium evidence
Page 20
- Improved leakage level
- Improved skin condition
Half of all patient ever studied in stoma care
Any questions or comments ?
Page 21
Jeppe Sørensen
International Health Economist, Coloplast
dkjsor@coloplast.com
Page 22
22 May, 2014
dksfjsldkj
Page 23