Pharma Pricing & Market Access

Pharma Pricing & Market Access
Congress 2017
London, 22. February 2017
Thomas Müller
M.D. and Pharmacist, Head of Pharmaceuticals Department
Federal Joint Committee (G-BA)

Seite 3 | © 2017 | Thomas Müller
Substantial
2
Considerable
47
Minor
38
No additional benefit8
87
Reference price group
4
Non-quantifiable
33
Less additional benefit
0
Highest category of additional benefit
/benefit assessment
in accordance with the German Social Code
Book Five (SGB V), section 35a
Erheblich
Beträchtlich
Gering
Kein Zusatznutzen
Festbetrag
Nicht quantifizierbar
Geringerer Nutzen
Status: 02.02.2017
Total.: 211 assessments
 Substantial
 Considerable
 Minor
 No additional benefit
 Reference price group
 Non-quantifiable
 Less benefit

Early scientific advice
with the participation of regulatory authorities
(including international advices)
2011 2012 2013 2014 2015 2016 Total
national
Total 42 82 134 136 190 204 788
European
Total 1 8 9 17 28 10 73
EMA 1 5 3 11 23 10 53
(HAS) SEED/EUnetHTA 0 3 6 6 5 0 20
Participation RA 1 5 12 20 34 30 102
BfArM (Federal Institute for
Drugs and Medical Devices) 1 4 4 9 10 18 46
PEI (Federal Institute for
Vaccines and Biomedicines) 1 8 11 24 12 56

Reimbursement price negotiations in accordance
with§130b SGB V
(Status: February 2017)
• Agreed reimbursement prices1: 106
• Reimbursement prices
determined by arbitration board 1: 21
• Market exits2: 19
• 1Notice: no consideration of negotiation results with re-importers, consideration of pharmaceuticals no longer placed on the market
• 2Notice: no distinction between “Opt-Out” and market withdrawal following set reimbursement pricing, no consideration of hospital use, combination therapies, global market withdrawal

Substantial
1 Considerable
0
Minor
14
No additional benefit
0
Reference price group
0
Non-quantifiable
28
Less additional benefit
0
Erheblich
Beträchtlich
Gering
Kein Zusatznutzen
Festbetrag
Nicht quantifizierbar
Geringerer Nutzen
19.01.2017Status:
Total : 43 assessments
Highest category of additional benefit /benefit assessment
in accordance with SGB V, section 35a - Orphan drugs
 Substantial
 Considerable
 Minor
 No additional benefit
 Reference price group
 Non-quantifiable
 Less benefit

Orphan Drugs: Number of G-BA-resolutions
including extent of additional benefit (2012-2016)
1
2
3
6
13
2
2
3
7
2
1
1
0
2
4
6
8
10
12
14
16
2012 2013 2014 2015 2016
non-quantifiable minor considerable

G-BA-resolutions: Orphan Drugs (chronological since December 1st, 2016)
extent of
additional
benefit
time
considerable
substantial
minor
non-
quantifiable
no additional
benefit
less benefit
Multiplemyeloma
HaemophiliaB
DuchenneMuscular
Dystrophy
Fabrydisease
HaemophiliaB
30–40
20–490
580-660
2300
580-660
790-940Follicularlymphoma
230-500Malabsorption
syndrome
Non-24-Hour
Sleep-WakeDisorder
Multiplemyeloma
Narcolepsy
Hodgkin's
lymphoma
7000
15000-30000
4700-7000
40-60
Chroniclymphoid
leukemia
Primarybiliary
cirrhosis
Multiplemyeloma
Softtissuesarcoma
? ? ? ?

Annual therapy costs of orphan drugs at
market access
Maximum annual therapy costs according to G-BA-resolutions
0 €
200,000 €
400,000 €
600,000 €
800,000 €
1,000,000 €
1,200,000 €
>1 Mio. € >1 Mio. €

Orphan Drugs:
PLOS One, Hughes et al (Oct 2016)
• Orphan drugs are more profitable compared to Non-Orphans.
• Incentives are effective: market exclusivity, reduced / waived
regulatory fees, protocol assistance, German Act on the Reform of
the Market for Medicinal Products (AMNOG) guarantees patient
relevant additional benefit (below € 50 Mio. annual turnover).
• Cost of Orphan drug clinical trials are less compared to Non-
Orphan trials (average phase III clinical trial: Orphan USD 95 Mio.
vs. Non-Orphan USD 219 Mio.
• Pricing of Orphan drugs 6x higher compared to Non-Orphans:
mean annual therapy cost: TUSD 137 vs. TUSD 21 Non-Orphans.
Despite smaller patient numbers revenue generating potential is
similar to non-orphans.
• Long-term effects on management of research investments ?

Orphan Drugs:
German drug bulletin „Arzneimittelbrief“ August 2016;
JAMA Oct 24, 2016:
„Arzneimittelbrief“:
• Analysis of orphan drugs having received accelerated
regulatory approval in 2004 shows: evidence of efficacy and
safety are still insufficent 10 years following approval
• Clear requirements for complementary clinical trials and
effective sanctions for lacking clinical evidence by EMA and
HTAbodies are needed following EMA regulatory approval
JAMA:“Partial Solution could be adoption of novel regulatory models,
such as limited approval with intensive collection of new clinical
evidence, before a drug becomes universally available. As a further
step, drugs that have not yet shown clinical outcome benefit could be
made available at just the cost of production, or most profits could be
kept in escrow, until adequate trials are completed.“

Highest category of patient relevant additional benefit
/benefit assessment
in accordance with SGB V, section 35a: indication-based
Substantial
1
Considerable
26
Minor
12
No additional
benefit
16
Reference
price group
0
Non-
quantifiable
16
Less benefit
0
Considerable
1
Minor
3
No additional
benefit
19
e.g. Diabetes: 23
Status: 15.12.2016
e.g. Oncology: 71,
including 16 orphan drugs
Status: 02.02.2017

time
considerable
substantial
minor
NSCLC:
Non-small-cell
lung carcinoma
CLL:
Chronic lymphocytic
leukemia
? ? ? ?
NSCLC6300–7700
gastric/gastro-
esophagealjunct.
adenocarzinoma
5900–7900
1)
2)
NSCLC
1)2100–5520
2)4620–6900
1)1200–3300
NSCLC
Renalcell
carcinoma2)max.8%oftotalpopulation
1)3700–9700
2)8130–12130
Multiple
myeloma
Liposarcoma
Melanoma
Melanoma
CLL
?
NSCLC
4700–7000
5900–7900
1)
2)
1a)230-690
1a)270-810
2)2000–3000
1a)1810
1b)810
2)220
1a)35-100
1a)40-120
2)300-450
NSCLC
Colorectal-
carzinoma
CLL
G-BA-resolutions: Oncology (chronological since September 15th, 2016)
extent of
additional
benefit
non-
quantifiable
no additional
benefit
less benefit
Renalcell
carcinoma

0
20000
40000
60000
80000
100000
120000
140000
160000
Melanom NSCLC RCC
Nivolumab vor
Preisverhandlungen
Nivolumab nach
Preisverhandlugen
Pembrolizumab vor
Preisverhandlungen
Pembrolizumab nach
Preisverhandlungen
AnnualtherapycostsinEuro
Considerable
additional benefit
Costs of Immunotherapy (Germany)
1
1 G-BA-resolution January 7th, 2016; indication for substantial additional benefit over Dacarbazin
2 Based on information from Lauer-Taxe regarding reimbursement pricings (in accordance with§78, section 3a AMG; calculations standardized
to one year)
3 G-BA-resolution February 4th, 2016; hint for substantial additional benefit over Ipilimumab and indication for substantial additional benefit over
a patient-individualized therapy according to the treating physician‘s choice (Ipilimumab)
4 G-BA-resolution October 20th, 2016; non-squamous epithelial histology; indication for substantial additional benefit over Docetaxel
5 G-BA-resolution October 20th, 2016; indication for substantial additional benefit over Everolimus
2 2 23 2 4 5
Considerable
additional benefit
Considerable
additional benefit
Pembrolizumab – prior to
price negotiations
Nivolumab – following
price negotiations
Nivolumab – prior to
price negotiations
Pembrolizumab – following
price negotiations

0
20000
40000
60000
80000
100000
120000
140000
160000
Pembrolizumab vor
Preisverhandlungen
Pembrolizumab nach
Presiverhandlungen
aim:±€27800
Costs of Immunotherapy (Germany)
e.g. Pembrolizumab (Melanoma)
JahrestherapiekosteninEuro
reimbursement
rejected
aim:±€50000
negotiations
terminated
§
#
1 G-BA-resolution February 4th, 2016; hint for substantial additional benefit over Ipilimumab and indication for substantial additional benefit over
a patient-individualized therapy according to the treating physician‘s choice (Ipilimumab)
2 Based on information from Lauer-Taxe regarding reimbursement pricings (in accordance with§78, section 3a AMG; calculations standardized
to one year)
3 http://www.apmhealtheurope.com; Merck & Co aiming to make Keytruda available in France 'as soon as possible'; October 13th, 2016
4 http://www.apmhealtheurope.com; UK's NICE rejects MSD's Keytruda in advanced lung cancer; October 4th, 2016
§cost per QALY > £ 50 000 (> € 55 550 )
# cost limit per QALY: £ 20 000 - 30 000 (€ 22 255 - 33 383)
1 2 3 4 34
negotiations
ongoing
Pembrolizumab – prior to
price negotiations
Pembrolizumab – following
price negotiations
AnnualtherapycostsinEuro
negotiations
terminated

time
470.000
78.000–170.000
a)374.000–918.000
a)20.000
b)200
Type1diabetesmel.
Type2diabetesmel.
Type2diabetesmel.582.000-800.000
Prophylaxisofstroke/
systemicembolisms/
phlebothrombosis/
pulmonaryembolisms
926.000-1.093.000
241.000
Heterozygous
hypercholesterolemia
Homozygous
a1)270.000
a2)1.750
a3)1.500
b)60-70
Heterozygous
a)270.000
b)1.750
c)1.500
Heartfailure
b)176.000–432.000
a)220.000
b1)
c2)
b2)
c1)
Prevention
atherothrombotic
events
105.000
1,0–1,4Mio.
c)550.000-760.000
a)523.000
b)635.000
550.000-750.000
extent of
additional
benefit
substantial
considerable
minor
non-
quantifiable
no additional
benefit
less benefit
G-BA-resolutions: Metabolism/Diabetes/Cardiovascular (chronol. since July 16th, 2015)
Type2diabetesmel.
Type2diabetesmel.
Type2diabetesmel.
Type2diabetesmel.

• Section 35: The issue of resistance for the treatment of bacterial
infectious diseases (antibiotics) shall be given special consideration.
Reserve antibiotics can be exempted when defining reference price
groups.
• AM-NutzenV: The issue of resistance shall be taken into account in
assessing the additional benefit of antibiotics.
AM-VSG draft key points (1)

• Evidence transfer for pediatric marketing authorizations shall be
examined for patient groups or subindications covered by the marketing
authorization, but which are not (or not sufficiently) represented in the
study population and for which marketing authorization has been
granted based on evidence transfer. The G-BA can recognize additional
benefit if the transfer of evidence conforms to and can be justified by the
standard of current scientific knowledge.

• Pharmaceuticals without additional benefit shall be banned from
reimbursement to patient groups when the resolution in accordance
with the German Social Code, Book Five (SGB V), section 35a is
passed, if this is necessary to ensure adequate care for individual
patient groups. Pharmaceutical companies can submit a petition to the
G-BA.
• Consideration of new evidence upon request during proceedings shall
be streamlined; repeated proceedings can be initiated one year after
the resolution is passed.

• Benefit assessment of pharmaceuticals with active ingredients that are
not new can be initiated in exceptional cases and if certain prerequisites
are met.
• Accompanying diagnostics: doctors’ fee schedule shall be revised soon
after the resolution.

• Machine-readable versions of the resolutions shall be published within
one month after passing (6 months after Rules of Procedure are
amended for older resolutions).
• AMNOG resolutions shall be implemented into the physician's
information system. Details, in particular on cost-effectiveness
requirements of prescriptions of pharmaceuticals as compared to other
treatment options, shall be defined in a regulation passed by the
Federal Ministry of Health.

• No dossier or documents (“additional benefit has not been proved”)
leads to a lower reimbursable price than appropriate comparator.
• Pharmaceuticals without additional benefit but with several alternatives
for the appropriate comparator: reimbursable price shall (formerly:
“must”) not lead to greater costs than the most economical alternative.

• The reimbursable price agreed must not be listed publicly. It may be
shared only with those institutions that require this information in order
to fulfil their statutory obligations. The Federal Ministry of Health shall
be authorized to pass regulations, with the consent of the Federal
Council in mutual agreement with the Federal Ministry of Economics,
detailing the invoicing of the reimbursable price.
• The price moratorium shall be continued.
• If revenues from a pharmaceutical exceed €250m in the first year of
market authorization, the reimbursable price shall apply from the month
following (not only from month 13).

Cross-Border Healthcare Directive (2011/24/EU)
provides a detailed legal framework focused on
three main areas:
• rules concerning the reimbursement of costs of cross-border
healthcare
• responsibilities of the Member States with regard to cross-
border healthcare
• cooperation between healthcare systems
EU Objectives in HTA Article 15 Directive 2011/24:
• Support cooperation between national HTA Authorities
• Support MS in the provision of objective, reliable, timely,
transparent , comparable and transferable information […] to
enable effective exchange of information
• Avoid duplication of assessments

Cross border health directive provides legal basis for HTA
network
EUnetHTA JA3 runs under the Third EU Health Programme
(2014-2020), legal basis: Regulation (EU) No 282/2014
HTA Network (political-strategic)
EUnetHTA (scientific-technical)
HTA Network and EUnetHTA

EUnetHTA timeline
EUnetHTA
Collaboration
Joint Action 1 Joint Action 2 Joint Action 3
Putting into
practice
Strengthening
practical
application
Turning pilots
into standard
practice
Develop a sustainable
model for cooperation
2006 2016 - 2020
EUnetHTA
Project
Inception

Project Coordinator:
The Dutch National Health
Care Institute (ZIN)
EUnetHTA JA3 Participants
79 partners consisting of
national, regional and non-
for-profit agencies that
produce or contribute to
HTA

Work Package Organization
Work package 1 Network Coordination
Work package 5
Evidence generation
(e.g., Early Dialogues)
Work package 7
National
implementation
Work package 2
Dissemination
Work package 3
Evaluation
Work package 6
Quality management
Scientific guidance
Tools
Work package 4
Joint production
Pharmaceuticals
Health Technologies

EUnetHTA Early Dialogues – status quo
Launch of multi-HTA
Early Dialogues on
January 26th 2017
http://www.eunethta.eu/news/call-expression-interest-multi-hta-early-dialogues-
pharmaceuticals
Call available on
EUnetHTA website:

• Procedure for Early Dialogues together with the EMA
(parallel Early Dialogues) currently under review
• Goal: merging of PSA (EMA plus few selected HTABs) and
EUnetHTA parallel Early Dialogues (EMA plus permanent
working party of HTABs, collaborative approach)

• Inception impact assessment launched, together with various
surveys
The future of HTA in the EU

• Portrayal of five policy options (mix and match possible)

German position towards the future of HTA in
the EU
• cooperation on HTA only voluntary
• building a lasting platform for exchange and
cooperation at scientific and technical levels
• ‘reuse’ of other HTA’s national (regional) reports
• Potential for EU ED replacing national one under
certain conditions

www.g-ba.de
arzneimittel@g-ba.de

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