Access to Innovative Therapies - Aspen Institute Romaniaaspeninstitute.ro/wp-content/uploads/2018/10/Panel... · 3 •~60% of NMEs in 2015 were priced at a discount to price comparator,

Copyright © 2017 IQVIA. All rights reserved.

Conference Presentation

23rd October 2018

Access to Innovative Therapies

1

Global Trends

2

New wave of innovation is apparent through an explosion of approvals for NMEs in the last few years…

EMA NME (new molecular entity) approvals by therapy area

0

15

30

4541

29

2013

15

2012 20152014

Num

ber

of

pro

ducts

14

2011

5

Musculo-Skeletal

Dermatology

Genito-Urinary

Cardiovascular

Alimentary

Nervous system

Respiratory

Anti-Infectives

Blood

Antineoplastic

Source: IQVIA HTA Accelerator

3

• ~60% of NMEs in 2015 were

priced at a discount to price

comparator, vs. ~20% in 2012

• Oncology products account for

the highest proportion of list

price premiums in 2015 NME

cohort

… however new treatments are experiencing growing price pressure from payers as they try to contain budgets

Comparative price premiums of EMA New Molecule Entities (NMEs)

0%

20%

40%

60%

80%

100%

20152014201320122011

-5 ~ 5%

-6 ~ -20%6 ~ 20% -36 ~ -99%

-21 ~ -35%21 ~ 35%

36 ~ 99%

>100%

Source: IQVIA HTA Accelerator

4

Similarly, Orphan Drug approvals surge in Europe…

10

18

20

14

7

11

54

10

6

0

5

10

15

20

2017*20162015201420132008 2012201120102009

Orphan drugs in Europe with European Market

Authorisation by approval year (2001-16) – 135 in total

Source: European Medicines Agency October 2017; *to Oct 2017 only

• 146 medicines were reviewed by

the EMA for orphan designation

since 2001-2016.

• As of 2016:

• 90 are approved Orphans

• 45 have been withdrawn

• 11 were refused designation

• 91% (82) of approved Orphan

drugs in 2016 have sales in Europe

in MIDAS

5

Source: IQVIA HTA Accelerator

… but restricted or negative outcomes are becoming more common in orphan drug assessments

* Positive = full access as per label; Restricted = access but with label restrictions; negative = no access granted

** In DE, Positive = “minor” or “major additional benefit” rating, Restricted = “non-quantifiable additional benefit”

rating, no negative ratings due to OD law mandating that drugs with OD designation automatically receive an

“additional benefit” rating

Orphan Designated Drug HTA Outcomes

(2013-2016)

22% 29% 27% 24%

43%31%

22%

14%

43%

89%72%

29%42%

78%62%

100%

57%

11%0%

20%

40%

60%

80%

100%

2013-

2016

2006-

2012

2013-

2016

6%

2006-

2012

2013-

2016

2013-

2016

2006-

2012

2006-

2012

Restrictive outcome Negative outcomePositive outcome

n = 18 50 14 56 5 32 9 28

6

Pipeline by Technology Class*

Phase II to Reg

5%

Cell therapy2%

Gene therapy6%

Antibiotic/-bacterial

3%

Other recomb. proteins

5%

Other proteins/peptides

10%

Other/Non specified

13%

55%

Vaccines/antivirals

mAb/mAb

drug conjugated

Cell and gene therapies by phase

Phase II to Reg

43

61Cell therapy

RNAi 19

Antisense/

oligonucleotide

6

Other

gene therapies53

6Exon skipping

Hydrolase

Nucleoside

analogue

20

Oncolytic virus

13

Phase III Registered

Phase II Pre-registration

Immuno-Oncology and Cell & Gene therapies are at the forefront of the new wave of technologies

Source: IQVIA R&D Focus October 2017; Thought Leadership analysis; active phase only

*Not specified excluded (717 products)® 2018 IQVIA Commercial – Pharma Trends 2018 Source: IQVIA R&D Focus October 2017; Thought Leadership analysis

7

Immuno-Oncology PD-1 and PD-L1 Inhibitor Uptake in the United States

Immuno-Oncology’s remarkable clinical profile offers unique opportunity through rapid indication expansion

® 2018 IQVIA Commercial – Pharma Trends 2018

0

200

400

600

800

1,000

1,200

1,400

Nov 2

014

Dec 2

014

Jan 2

015

Feb 2

015

Mar

2015

Apr

2015

May 2

015

Jun 2

015

Jul 201

5

Aug 2

015

Sep 2

015

Oct 2015

Nov 2

015

Dec 2

015

Jan 2

016

Feb 2

016

Mar

2016

Apr

2016

May 2

016

Jun 2

016

Jul 201

6

Aug 2

016

Sep 2

016

Oct 2016

Nov 2

016

Dec 2

016

Jan 2

017

Feb 2

017

Mar

2017

Exte

nd

ed

Un

its T

ho

us

an

ds

Non-Squamous NSCLC

BRAF V600 Wild-Type

Melanoma

(combination with

ipilimimab)

pembrolizumab approvals

nivolumab approvals

atezolizumab approvals

avelumab approval

Squamous

NSCLC

Renal Cell

Carcinoma

BRAF V600

Wild-Type

Melanoma

MelanomaNSCLC

PD-L1+

Melanoma

(first line)

Bladder

Cancer

Melanoma across

BRAF status

(combination with ipilimimab)

Hodgkin

Lymphoma

Head and

Neck Squamous

Cell CarcinomaBladder Cancer

Head and

Neck Squamous

Cell Carcinoma

NSCLC

(first line)

Hodgkin

Lymphoma

Merkel

cell

carcinoma

Source: U.S. FDA, QuintilesIMS, National Sales Perspectives, Feb 2017; QuintilesIMS Institute, Apr 2017

8

A new wave of Immuno-oncology drugs is in the pipeline for virtually all cancer types

4-1BB Agonist

Anti-CSF-1R

Anti-KIR

Anti-LAG-3 mAb

Anti-M-CSF mAb

Anti-PD1

Anti-PD-L1

Anti-TIM3 mAb

CAR-T cell therapy

IDO-1 inhibitor

Phase 1

Phase 3

Phase 2

Marketed PD1/PDL-1 inhibitors have been explored across most of the existing tumor types. Next generation of I-O MoA

(such as IDO, Anti-CSF-1R) have already started to expand their reach across multiple tumors

Notes: MoAs such as OX-40, Anti GITR, TIGIT, are in phase 1 trials for advanced solid tumours and have not been included here; urothelial cancer included within bladder cancer; Pfizer’s phase I CCR2 antagonist, CD137 agonist and CD19 CAR-T

are not included here.

Snapshot of immuno-oncology drugs pipeline

9

Cell & Gene therapies, on the other hand, offer unique value across large number of under- or un-treated diseases

Number of pipeline candidates by Top 10 companies

Phase II to Reg

4

5

5

6

8

9

19

2

1

3

3

4

2

GSK

AGTC

Sanofi

Novartis 5

5

Celgene 5

Kite Pharma

Sangamo

Therapeutics

J&J

Alnylam

Ionis

Cell therapy

Gene therapy

Major therapeutic areas of pipeline products

Phase II to Reg

5%Metabolic disease

Ophthalmology6%

Infectious diseaseHaematology

Genetic disorder

12%8%

8%

Oncology34%

6%CNS

6%Musculoskeltal

4%

Cardiology11%

Other

Source: IQVIA R&D Focus October 2017; Thought Leadership analysis

10

Oncology alone, should pipelines materialize, will triple in value, effecting extreme budget pressure…

Source: IQVIA, ARK R&D Intelligence, Feb 2017; IQVIA Institute, Mar 2017; IQVIA MIDAS QTR restricted MAT Q3 2017

New Active Substance by Cancer Type by 2024

11

As treatments evolve and grow more complex, they present all stakeholders with many risks and uncertainties

Stakeholders question whether oncology products will work as

promised and seek to avoid paying for, prescribing, or using

ineffective therapies

Uncertainty about clinical

impact or value

Uncertainty about budget

impact or cost

Uncertainty about

patients

Affordability

Transaction and

monitoring costsProducts, agreements, or contracts that impose substantial

monitoring requirements will be met with skepticism.

Providers and payers want to know which patients to treat, in

which patients a response is most likely, and how long patients

will remain on therapies

Still scarred from HCV, health systems worry that treatment

volumes will exceed expectations and seek to limit cost exposure

Even cost-effective products encounter limits. Payers and

patients must keep expenditure below a certain level.

Payer PatientHCP

12

In the end, accelerated access to innovation will require a delicate balance vs. budget impact and within a complex policy environment

Containing budget impactFacilitating access to innovation

Within an environment of policy

changes/uncertainty

Payer Balance

Contact information

Sorin Petcu

Country Manager, IQVIA Romania

Email: [email protected]

Tel: +40 722 648 046