reference model for economic evaluation: industry perspective · – better characterization of the...

Reference Model for Economic Evaluation: Industry Perspective

Christopher LeibmanVP, HEOR – Global Market Access

Biogen

Disclosure and Acknowledgements C. Leibman is an employee of Biogen

Views and opinions are Leibman’s, not Biogen’s

Acknowledgements: Michele Potashman, Ravi Singh, Samantha Budd Haeberlein, Eric Hall, Tom Koenig, Robin Thompson

Outline IPECAD 2011 - Let’s see how far we’ve come…

IPECAD 2015 – Situation and near-term– Key attributes for a ‘reference’ model

IPECAD circa 2020+– Preparation for transformation

Conceptual Framework Focused on AD, not pAD

McLaughlin T, Feldman H, Fillit H, Sano M, Schmitt F, Aisen P, Leibman C, Mucha L, Ryan JM, Sullivan SD, Spackman DS, Neumann PJ, Cohen J, Stern Y. Alzheimer’s & Dementia 6(2010) 482-493.

AD Models: Moving Towards Models 2.0 Much of this has been driven by:

– Lack of good data (measures, trial length)

– Limited inclusion of broader measures that might incorporate impacts of multiple symptoms into clinical trials

Future Models– More realistic and compelling evaluations of various interventions

– Better characterization of the disease – conceptual framework

– Better assess the full range of costs and benefits across interventions

– Better evaluate the incremental costs and benefits

Cohen JT, Neumann PJ. Alzheimer’s & Dementia 4(2008) 212-222.Green C. Pharmacoeconomics 2007; 25(9): 735-750.

AD: Alzheimer’s Disease

IPECAD 2015: ‘Reference’ Reality in AD Where have ‘reference models’ been successful

Attributes for reference success:– Population characterization stability – clear definitions

– Robust/Agreed data on disease progression (and costs)

– Evidence expectations clear – aligned endpoints / staging

– Stable period of clinical practice and clear treatment pathways

– Recent assessments to draft from:• Previous model variation has been honed - ‘survival of the fittest’

• Fit for near-term purpose – recent series of evaluations lead to anticipation of upcoming evaluations

• Consortium interests align (nice to have – but not required)

Population Continuum Evolving

~1.6M ~2.3M ~1.4M

5.3 M - Diagnosed with Alzheimer’s disease in the US

Cognitive and functional decline fulfilling dementia

SevereSevereModerateModerateMildMild

US Census Bureau 2014, * Jansen et al JAMA 2015, ⱡ Roberts et al Clin Geriatr Med 2013, Petersen et al Curr Alz Res 2009, Mitchell et al Acta Psych Scand 2009# Petersen et al Neurol 2012, Whitwell et al Arch Neurol 2012, Plassman et al Ann Int Med 2008, Manly et al Arch Neurol 2005

Population Continuum Evolving10-15%ⱡ Conversion/year

MCI due to AD / Prodromal

MCI due to AD / ProdromalAt risk PopulationAt risk Population

~1.6M ~2.3M ~1.4M ~13M ~3.8M

27%* of over 65’s - US at risk population

~8%# of over 65’s - US amnestic MCI pop.

5.3 M - Diagnosed with Alzheimer’s disease in the US

Amyloid positive, ‘cognition normal’


Subjective memory decline


Early AD

US Census Bureau 2014, * Jansen et al JAMA 2015, ⱡ Roberts et al Clin Geriatr Med 2013, Petersen et al Curr Alz Res 2009, Mitchell et al Acta Psych Scand 2009# Petersen et al Neurol 2012, Whitwell et al Arch Neurol 2012, Plassman et al Ann Int Med 2008, Manly et al Arch Neurol 2005

Targeting of the Disease Continuum







Targeting of the Disease Continuum







MCI/Mild MMSE 22-30, CDR 0.5 to 1.0

Mild MMSE 20-26

Asympt. MMSE 25-30,

CDR 0

MCI/Mild MMSE 21-28

MCI due to AD/portion of Mild MMSE 24-30, CDR 0.5

Mild (No MMSE)

MCI/Mild ≥ 20

Mild-Moderate (No MMSE)Prodromal (No MMSE)

*Prodromal and Mild definitions on MMSE overlap from 24-26

Early AD, CDR 0 to 0.5 (No MMSE)

Aβ plaque mAbAβ soluble mAb

BACE

Solanezumab

MK-8931

Aducanumab

Gantenerumab

AZD3293

JNJ-54861911

BAN-2401

E2609

Inve

stig

atio

nal C

ompo

unds

Evidence expectations clear?

Outcome of interest clarity

Economic flow by stage (population)

Patient and care-provider or support inclusion

Disease continuum and model construct: new stages, thresholds

Need for a fit for purpose evolution

IPECAD 2020…We can’t wait!AD starts many years prior to onset of symptoms

SevereSevereModerateModerateMildMildMCI due to AD / Prodromal


Jack, et al. Lancet Neurol (2013)

There is work to be done!

Population continuum and model structure versus…

Data challenges - Prioritization of gaps?

Population flexibility

Inclusion of other costs and benefits – appropriateness

Thinking with the future in mind:– Movement to non-symptomatic patients – use of surrogates

– Relevance of biomarkers – enrichment vs. outcomes

“Would you tell me, please, which way I should go from here?” Alice asked the Cheshire Cat.“That depends a good deal on where you want to get to,” said the Cat.

“I don’t much care where…” said Alice.

“Then it doesn’t matter which way you go”, said the Cat.

“…so long as I get somewhere”, Alice added as an explanation.

“Oh, you’re sure to do that,” said the Cat, “if you only walk long enough”.

Where do we go from here?

Theme 1: Outcome definition

Theme 2: Access to quality data

Theme 3: Use of data

Theme 4: Patient engagement

WP 2: Outcome definition and

mapping of RWE data sources

WP 3: Integrationstrategy of RWE

data

WP 4: Modelingand simulation

WP 5: HTA – EMA Integration

Set of target outcomes

Searchable web catalog of available data sources

Evaluation of suitability for use in natural history / effectiveness

Evaluation of suitability for combining data sources

Integration strategy (incl. demonstration)

Digital alternatives (incl. demonstration)

Statistical functions to predict outcomes

Drivers of outcome variation

Comparisons of methodologies to model archetypes

WP 1: Project management

WP 6: Training and CommunicationWP 7: Legal and Ethics

IMI: Real World Outcomes Across the AD Spectrum (ROADS) to Better Care Consortium

Thank You!

[email protected]

reference model for economic evaluation: industry perspective · – better characterization of the...

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