1

Build a deeper appreciation and understanding of HTA needs into

your wider organization

Stefan HolmstromExecutive Director HEOR

Medical Affairs, Global

Astellas Pharma

2

Outline of presentation

1. Changing environment of Pharma

2. The Challenge

• External

• Internal

3. Integrating HTA needs into organization and internal alignment

4. Conclusion

33

Changes in the healthcare environment

A number of factors are having increasing influence on

healthcare decision making, resulting in pressure and

focus on healthcare budgets.

Pressure on

healthcare

budgets

Ageing

population

& resulting

increase in

chronic illness

Advances in

medical

technology

Slower

economic

growthCost

containment

measures

by payers

Increasing

number &

types of

healthcare

professionals

Increase

in public

expectation

44

5

Defining the challenges

External challenges

The HTA and Payers

Internal challenges

The internal stakeholders and focus

points

6

We all have different focus

ExternalHTA and Payer is focusing on optimizing the use of limited resources/budgets

InternalDevelopment organization is focusing on getting NDA and MAA approvals

Pricing department is focusing on getting the “best possible price” = highest possible price?

Commercial is focusing on getting the biggest market share

Health Economics is focusing on getting an acceptable ICER

Market Access is focusing on getting payer acceptance

7

Regulators versus HTA bodies

“Having an approved NDA/MAA,

qualifies you to talk to me”

Dr. Andrew Walker,

Sr. Economic Reviewer, SMC

88

Payer Need Payer performance measures

R&D delivery

Performance Reduction in symptoms RCT – show efficacy improvements (x-y%)

Effective Usage Clear stop/start rules Identify responders/diagnostics

Value for Money Benefit vs. costs is acceptable Economic Modeling

Human Impact Acceptable QoL for patients Demonstrate effect on QoL and family

Stakeholders Level of pressure from key influencers

Build Constituent need in trial design

Real-world Proof (based on data) that it will deliver real world results

Phase IV open label, Observational, Chart review

HTA & Payer relevant endpoints

99

Our challenges

Safety & Efficacy

Quality

Payers/ HTA

Cost Effective

Budget Impact

Physician and

Patient

acceptance

1010

Dialog with regulators, payers & HTA bodies on endpoints and design

11

Defining the challenges

External challenges

The HTA and Payers

Internal challenges

The internal stakeholders and focus

points

1212

Stakeholder views on the role of a GVD

4

1

2

5

EU5 (n=6) Non EU5 (n=6)

EU5 vs. non EU5 LAF

Yes No

HQ:

“Without the GVD you may have submissions

prepared by people that don’t understand your

strategy behind the positioning of the drug..”

UK-LAF:

“No, it purely depends on the data and

if the data is relevant to the market.”

Can a GVD influence submission outcome?

5

6

7

1

0% 50% 100%

LAF (n=12)

HQ (n=7)

HQ and LAF responses

Yes

No

N/A

With permission of Quintiles Advisory Services, Pim Kooreman, 2015

1313

• HQ: All sections presented in the GVD are equally important (n=7/7; 100%)

• LAF: Some sections are more important than others (n=11/12; 92%)

» Clinical data (n=8)

» Positioning statements (n=5)

» Economical data (n=3)

Stakeholder views on the content of a GVD

DE-LAF:

“Not really missing but not

as in depth as I need it from

a country perspective.”

HQ:

“They are all equally

important, the GVD is

telling a story. “

Are there any sections in the GVD more important than others?

Is there information missing from the GVD?

7 5

5 1

0% 20% 40% 60% 80% 100%

LAF (n=12)

HQ (n=6)

HQ and LAF responses

Yes

No

Ambiguous

With permission of Quintiles Advisory Services, Pim Kooreman, 2015

14

Outline of presentation

1. Changing environment of Pharma

2. The Challenge

• External

• Internal

3. Integrating HTA needs into organization and internal alignment

4. Conclusion

1515

HEOR Key activities to maximize product value

10% 20% 30% 40%

Drug discovery today 02/2012; 17(11-12):615-22

1616

Pyramid of refinementFinding the balance with your intrinsic data & results and the HTA/Payer needs

Blue Sky

Testing your message hypothesis, endpoints,

PRO’s and models

Delivering final Value Propositions and

HE model

RWE and

refined Value

POC

Launch

LCM

ph2

ph3

1717

The Global Value Alignment model

(Phase II) Phase IIIARegulatorysubmission

HTA/ Reimbursement

submissions, Phase IIIb/IV

To LCM

Value Arguments

Early Evidence Generation

Value dossier Country dossiersEarly Value Dossier

(POC)

Late Phase Evidence Generation/Value Dissemination

Data examples Data examples

Global Value Team

1818

Global Value Team

Chair

Region

Region

Region

Global

Strategic

Alignment

1919

Integrated focus for optimal Market Access

HTA strategy

Reimbursement

strategy

Brand support

LCM and

Regional supportGuidance

Competitors and

RWE

R&D Focus

Development focus

R&D Commercial

Regions/

Affiliates

Strategic

support

2020

Integrated focus for optimal Market Access

HTA strategy

Reimbursement

strategy

Brand support

LCM and

Regional support

Guidance

Competitors and

RWE

R&D Focus

Development

focus

Multidisciplinary approach

Market Access

Governmental

Affairs/PolicyClinical

Medical

HEOR

CommercialRegulatory

Statistics

R&D Commercial

Regions/

Affiliates

Strategic

support

KAM

Pre Clinical

2121

Global Value Team

Chair

Region

Region

Region

Global

Strategic

Alignment

2222

What does good look like?

• GVD Structural optimization

• Regional and Affiliate input

• Focus on Medical Affairs Core Medical Team

• Endpoint selection

• Guidance

• Focus on development structure Core Team

• Methodology excellence

• HTA Expert networks

• Early Scientific Advice

• PRO and QoL guidance

• Instrument control

• PRO Expert network

PRO’s HTA

Global VDEarly

Development

2323

Value chain, working by stage

Launch readiness Post approval supportLocal level

engagement

Strategic

and

tactical

activities

• HEOR input into

Development studies

(Phase 2 / 3 studies)

• Preparation of Global

Value Dossier

• PRO assessment and

input

• Landscape assessment

(e.g. unmet need, disease

burden, competitor

analysis)

• Engagement with

customers (e.g. product

presentations)

• Adjust Global Value

Dossier to country needs

• HEOR projects:

− disease registries

− External database

analyses

− Post hoc analyses

− Big data analyses

• Adjust Global Value

Dossier to region and

country needs

• Alignment and governance

of HEOR activities

ApprovalEarly decision

Involve-

ment

Global,

(with region input)

Region,

with Global coordination

Country execution

(with region support)

Global Value team coordination

24

Key recommendations

• Tailor global market access strategy based on product qualities

• Breakthrough/first in class vs. me too vs. follower

• Be pro-active and understand setting-specific requirements and payer mindset

• engage with key stakeholders early on (regulators, HTAs, patients); not always easy, but necessary

• Ensure global alignment with development team members (statistics, project lead, medical, regulatory, HEOR, commercial, etc.) as well as continuous regional and policy input

25

Key recommendations

• Ensure Value Dossier is available at NDA/MAA approval

• Enhance skills and capacity internally to understand client needs:

• Health systems

• Pricing, Health economics and outcomes

• Patient advocacy

• Regulatory

26

CONCLUSION

2727

Conclusion

● Planning for evidence generation should begin in early phase and be regarded as a “living” process that can be updated to account for new internal data and new external pressures throughout the product lifecycle

• Identify unmet information needs

• Align evidence with product value

proposition

• Build implementation plan

• Health Care Professionals

• Patients & Caregivers

• Regulators/Health Authorities

• Payers

P r o d u c t L i f e C y c l e

Integrated,

Holistic

Evidence

Generation

Develop credible evidence…

…when it is needed

…for appropriate audiences…

Phase II Phase III Phase IV Loss of Exclusivity (LOE)

28

To accomplish great things,

we must not only act,

but also dream;

not only plan,

but also believe

- Anatole France