Health technology assessment (HTA) criteria in the light ...old.iss.it/binary/scf1/cont/Dario_Scapola.pdf · and companies to create earlier access in an “Adaptive Licensing”

Health technology assessment (HTA) criteria in the light of

current R&D trends

Dario ScapolaMarket Access Director - Roche S.p.A.

Background: Technological & societal developments

Improved understanding of disease biology and availability of big data.....

........are leading to an increased efficacy in pharmaceutical research......

.......which coincides with a demographic change and economically volatile situation

These developments require:

A rapid formulation and adoption of solutions - to allow patients full access to new treatments

+ + + = ?

We are in the transition from the “blockbuster model” …

Patients with same syndrome

One-size-fits-all approach

.. to a Personalised Healthcare model

Group of patients with the same syndrome

Opportunity: Targeted therapy

Good news and a challenge: Our improved understanding of cancer biology challenges our traditional business model

Potential driver mutations (NSCLC) Overlapping Biomarkers (NSCLC)

Met Low

(45%)

PIK3 Mutation

PTEN Loss (12%)

PDL1

(28%)

Napi3B

(73%)

KRAS

Mutation

(25%)

Met High

(55%)

3 Challenges for Patient Access in the Future

1. How to speed up collaboration between patients, payers, regulators and companies to create earlier access in an “Adaptive Licensing” setting?

2. How to pay for new combinations of therapies and technologies? Example: Pricing definition for Combination Therapies

3. How to define the value of use for existing therapies and technologies for different diseases? Example: Value Based Pricing for Multi-Indication Products

Traditional paradigm with regulatory focus necessary

but not sufficient

Payers need to understand comparative effectiveness of

medicines, not only benefit/risk.

Evidence must be gathered from a whole variety of different

sources including Real World Data.

Accelerated approval leaves less time to generate evidence for

all our stakeholders.

Meaningful comparative effectiveness data must be generated

and communicated.

Access Evidence must be addressed early and often to be able

to effectively meet HTA requirements.

Increasing payer reliance on

HTA to understand clinical and

economical value

Changing payer evidence

requirements

Focus on the importance of

outcomes for patients and other

evidence sources

Increasing competition

Opportunities to accelerate

approval in areas of particular

unmet patient need

Trend What this means for us

HTA: Health Technology Assessment

On balance, regulators and payer evidence requirements are diverging,

not converging

Regulators ‘evolving approval’ of

safety, efficacy and quality

•FDA breakthrough – single arm

lighter weight trials possibly sufficient

•Adaptive licensing (AL) – EMA are

working on how AL could be achieved,

mindful of payer

Becoming more flexible

and adaptive

Becoming more stringent

on evidence of incremental

benefit

Regulators Payers

Payers often will not extrapolate

clinical endpoints to patient benefit or

to populations outside clinical trial

Evidence some payers will consider:

• Coverage with evidence development

i.e. Real World Evidence

• Pay for performance arrangements

EMA: European Medicines Agency; FDA: Food and Drug Administration

Requirements:

FDA/EMA versus HTA

REGULATORS (EMA, FDA) PAYERS (HTA)

Risk/Benefit profile Value compared to existing

alternatives

Surrogate / Intermediate are usually

primary endpoints in clinical research

Final outcome first (mortality and

quality of life) Intermediate (avoided

events) and surrogate afterwards

Economic impact not

considered

Crucial role played by the economic

impact: value for money and

budget impact

Standard criteria Different approaches across countries and

sometimes within countries

Adaptive Pathways will Require New & Early Interactions with Our

Stakeholders

• Early payers involvement is needed to

avoid fast regulatory approval and

delayed access to patients

• Are we ready for adaptive

reimbursement?

• RWE should be accepted

• Role of patient advocacy in RCT design

should improve





Combination Treatment:Example of a Managed Entry Agreement

In Italy, for the first time, the cost of the combination was not based on the sum of

each drug price, but a total therapy cost was negotiated

Savings

for NHS

Costo/totale

ciclo terapia

ciclo A+BBiologico B

Costo/Ciclo

Biologico A

Costo/Ciclo

30% savings on list

prices

combination

Prices combination Negotiated cost per

treatment cycle





A medicine’s value may differ between indications

Indication D

Pe

rce

ive

d b

en

efi

t o

f

mo

lec

ule

Lower

Higher

Ind

icati

on

BBenefit of a molecule may be perceived differently

because

• Different incremental health gains („relative

effectiveness“) in the various indications

• Unmet need in the various indications perceived

differently

• Different competitive situation (“relevant treatment

alternatives”) in the various indications.

• It is important to note that different indications are

usually launched sequentially which has

implications for potential price adjustments.

Ind

icati

on

C

Ind

icati

on

A

Different indication prices reflect different

values for indications

Value Based Pricing allows

payments based on different

unmet needs and willingness to

pay

Take home messages

• FDA/EMA and HTA requirements are diverging not converging. Traditional

development paradigm with regulatory focus necessary

but not sufficient.

• A Value based strategy must be defined from the start of a drug development to address

HTA requirements. Product perceived value will ‘drive’ Market

Access.

• AIFA Criteria for innovation will be a model of product evaluation for any new

applicants. The ‘NEW’ must have a measurable value in term of improving patient

condition and healthcare system quality.

Doing now what patients need next

Documents

Health technology assessment (HTA) criteria in the light ...old.iss.it/binary/scf1/cont/Dario_Scapola.pdf · and companies to create earlier access in an “Adaptive Licensing”