Why RWE Matters to Payers: Incorporating RWE in Health Economic Analysis to Maximise Value

Amanda Cole, [email protected]

Why RWE Matters to Payers: Incorporating RWE in Health Economic Analysis to Maximise Value

7th DecemberHealth Economics Global Congress 2015

Why RWE matters for payersDecember 2015 2

Content

1. RWD and RWE – what and why?• What is RWD/RWE• Why might it be important for payers?

2. Do payers live in the ‘real world’?• Managed entry agreements (MEAs) to address uncertainty

and maximize value for payers

3. Challenges• Implementation• Governance• Methods to deal with messiness


Preview: Five global challenges1

The environment for global R&D is evolving

Challenge 1: Increasing importance of specialised and stratified medicines

Challenge 2: Rising drug development costs

Challenge 3: Closer benefit/risk monitoring by regulators over a medicine’s lifecycle

Challenge 4: Increase in demand for RWE and relative effectiveness by payers, and regulators

Challenge 5: Disconnect between regulators and payers/HTA bodies evidence needs

1 Mestre-Ferrandiz, Towse, Pistollato and Lewis. 2014. Securing a future for innovative medicines: a discussion paper. London: ABPI.


1. RWD and RWE – what and why?


1. RWD and RWE – What and why?

?


• Real world data (RWD) • Collected outside of an experimental clinical trial

setting: reflects outcomes in the ‘real world’.• Electronic health records, clinical registries,

surveys, observational data from cohort studies, etc. (messy data)

• Real world evidence (RWE)



1. Understanding outcomes in the ‘real world’• Efficacy Effectiveness• Representative populations (co-morbidities,

spectrum of ages, treatment delivery less controlled etc.); additional insights e.g. adherence, costs.



2. Evolving R&D pipeline• Declining R&D investment in drugs - often

attributed to rising cost of developing new products

• More specialised and stratified medicines – Highly targeted, small populations, high

uncertainty


2 Daniel et al. (2015) Health Affairs, 34 (2) pp. 319-3273 Scannell et al. (2012). Nature reviews Drug discovery, 11 (3) pp. 191-200

Source: Daniel et al (2015)2:

Annual new drug approvals by FDA and Industry spending on R&D, 1994-2013

Reduced R&D productivityNumber of new drugs approved per $billion halved every 9 years since 19503


3. Timing of decision-points for regulators and payers are changing

• Regulators: Accelerating access to medicines that have the potential to address a high unmet need (uncertainty vs. urgency)– E.g.: EAMS (UK), ATU (France), BTD (U.S)

• Payers: Making decisions based on a limited evidence base.


Adaptive pathways


1. Understanding outcomes in the ‘real world’2. Evolving R&D pipeline3. Timing of decision-points for regulators and

payers are changing

USING RWE TO MANAGE UNCERTAINTY



2. Do payers live in the ‘real world’?


• In England the National Institute for Health and Care Excellence (NICE) produces evidence-based guidance to the NHS (payer).

• Decisions based on robust clinical and cost-effectiveness evidence

• [not selected if it is expected that there is insufficient evidence]

• Yes• Optimised • No• Only in research (we’re too uncertain)

2. Do payers live in the ‘real world’?

Managed entry agreements


• Managed entry agreements (MEAs)• Performance-based risk-sharing arrangements (PBRSA), outcomes-based

schemes, pay-for-performance, patient access schemes...

• Formal utilisation of RWD to manage the entry of a product. Can be in many forms, for example:

• commitment to collecting confirmatory evidence of effectiveness in real-life settings

• linking reimbursement explicitly to patient outcomes

• capping expenditure • Ensures the payer receives value for money

2. MEAs


Source: University of Washington PBRSA database. May 2015

2. MEAs


2. MEAs• MEAs to address uncertainty

and maximise value for payers4

Clinical uncertaintyValue for moneyBudget impact

4 Garrison, L.P., Towse, A., et al. (2013). (5) pp. 703-719


• Reimbursement based on outcome - ‘Rebate’ for non-responders

• Manufacturer pays for the medicine beyond a certain number of doses

• Reimbursed for a specified period of time whilst further evidence / RWD are collected

Performance-linked reimbursement

Coverage with evidence development

Outcomes guarantee

Dose capping

RE

AL

WO

RLD

DAT

A2. MEAs


3. Challenges

Implementation, Governance, Methods


• Substantial administrative burden• Can be complicated to negotiate with providers• Capacity for data collection

• Electronic health records, registries, patient identifiers for data linkage5

• Coverage with evidence development:• sufficient time to address resolvable uncertainty • Not straightforward to ‘de-list’ a drug (an ‘acquired

right’?)

3. Challenges: Implementation

5 Chapman & Karlsberg-Schaffer (2015). OHE Consulting report: https://www.ohe.org/publications/assessing-use-multi-indication-medicines-review-current-data-capabilities-uk

https://www.ohe.org/publications/assessing-use-multi-indication-medicines-review-current-data-capabilities-uk




• Who manages (and pays for) data collection / analysis?

• Information governance• Data protection: patient consent• De novo versus routinely-collected data• Audit or research?

3. Challenges: Governance6

6 Cole, Garrison, Mestre-Ferrandiz & Towse (2015) OHE Consulting Report. London: https://www.ohe.org/publications/data-governance-arrangements-real-world-evidence

https://www.ohe.org/publications/data-governance-arrangements-real-world-evidence




3. Challenges: Methods• Methodological challenges in dealing with

messy RWD• Incomplete data• Confounders and biases• Quality of data

• Precedent set by medical technologies evaluation?


• Understanding outcomes in the real world• Being responsive to an evolving R&D pipeline• There is an expanding experience with regulatory

mechanisms that allow earlier access to medicines in development Payers need to be on board

• HTA moving from Yes/No Agreements• MEAs can explicitly link payment with value• There are implementation, governance and

methodological challenges to the implementation of MEAs.

ConclusionWhy RWE Matters to Payers


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Why RWE Matters to Payers: Incorporating RWE in Health Economic Analysis to Maximise Value

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