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The ISPOR task force defined real-world data as: PowerPoint PPT Presentation


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What Is Meant by "Real-World Data?". The ISPOR task force defined real-world data as: Data used for decision-making that are not collected in conventional randomized clinical trials. Evidence is shaped, while data simply are raw numbers and, alone, are noninformative.

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The ispor task force defined real world data as

What Is Meant by "Real-World Data?"

  • The ISPOR task force defined real-world data as:

  • Data used for decision-making that are not collected in conventional randomized clinical trials

Evidence is shaped, while data simply are raw numbers and, alone, are noninformative

ISPOR = International Society of Pharmacoeconomics and Outcomes Research

Garrison LP, et al. Value Health. 2007;10(5):326-335.


The ispor task force defined real world data as

Limitations of Randomized Clinical Trials

Real-world observational

Traditional interventional

Pragmaticclinical trial

Randomized controlled trial

Prospective observational study

Retrospective observational study

  • Patients treated according to the protocol

  • Actual care that patients receive in clinical practice

  • Extensive inclusion and exclusion criteria

  • All patients must be treated, including those with comorbidities

Garrison LP, et al. Value Health. 2007;10(5):326-335; Nallamothu BK, et al. Circulation. 2008;118(12):1294-1303.


The ispor task force defined real world data as

Limitations of Randomized Clinical Trials

Real-world observational

Traditional interventional

Pragmaticclinical trial

Randomized controlled trial

Prospective observational study

Retrospective observational study

  • Patients treated according to the protocol

  • Actual care that patients receive in clinical practice

  • Extensive inclusion and exclusion criteria

  • All patients must be treated, including those with comorbidities

  • Endpoints are efficacy and safety over a predetermined time period

  • Longer-term efficacy and safety data + economic assessments under typical clinical conditions

Garrison LP, et al. Value Health. 2007;10(5):326-335; Nallamothu BK, et al. Circulation. 2008;118(12):1294-1303.


The ispor task force defined real world data as

Limitations of Randomized Clinical Trials

Real-world observational

Traditional interventional

Pragmaticclinical trial

Randomized controlled trial

Prospective observational study

Retrospective observational study

  • Patients treated according to the protocol

  • Actual care that patients receive in clinical practice

  • Extensive inclusion and exclusion criteria

  • All patients must be treated, including those with comorbidities

  • Endpoints are efficacy and safety over a predetermined period of time

  • Longer-term efficacy and safety data + economic assessments under typical clinical conditions

  • Compare treatment against the standard of care (eg, interferon or glatiramer acetate)

  • Possible to compare multiple interventions

Garrison LP, et al. Value Health. 2007;10(5):326-335; Nallamothu BK, et al. Circulation. 2008;118(12):1294-1303.


The ispor task force defined real world data as

Sources of Real-World Data

  • Pragmatic clinical trials

  • Prospective observational studies and patient registries

  • Administrative claims data

  • Patient surveys

  • Electronic health records/medical chart reviews

  • Government- or third-party-sponsored systematic surveys that assess public health, resource consumption, practice patterns, and clinical trends

Garrison LP, et al. Value Health. 2007;10(5):326-335.


The ispor task force defined real world data as

Clinical and Post-Marketing Experience With Oral Therapies

  • IMS Health. Gilenya Unique Patient Exposures. 2014. Data on file.

  • Henson LJ, et al. CMSC-ACTRiMS 2014. Abstract DX41.

  • http://www.tecfidera.com/pdfs/full-prescribing-information.pdf


The ispor task force defined real world data as

PANGAEA: Documentation of Fingolimod Treated Patients in a Registry

  • Noninterventional registry study for prospective compilation of long-term data on safety and efficacy of fingolimod

  • 4000 patients with RRMS from neurological clinics and practices throughout Germany

  • Recruitment was completed at the end of 2012

  • Safety and efficacy data will be collected over 5-year observation period

  • Pharmacoeconomic data in 800 patients will be collected for 2 years

  • Designed according to the Risk Management Plan of the EMA

PANGAEA

Observational phase

End ofTreatment

Baseline

Fingolimod 0.5 mg, n=4000

Enrolment of patients

EMA = European Medicines Agency; RRMS = relapsing-remitting multiple sclerosis

Ziemssen T, et al. ECTRiMS 2012. Abstract P522.


The ispor task force defined real world data as

PANGAEA 24-Month Interim Results: ARR by Previous Therapy

ARR = annualized relapse rate; IFN = interferon

Ziemssen T, et al. AAN 2014. Abstract P3.152.


The ispor task force defined real world data as

Days Off Treatment: Adherence With Fingolimod

During the last 14 days, how often did you not take your medication?

Number of Days Off Treatment During Last 14 Days

A comparison between PANGAEA and PEARL shows better compliance on fingolimod than on first-line therapy.

DMT = disease-modifying therapy

Ziemssen T, et al. ECTRIMS 2012. Abstract P302.


The ispor task force defined real world data as

MSBase –Fingolimod vs BRACE Therapies:

Study Overview[a]

Objective: Assess comparative effectiveness on time to first relapse and to treatment discontinuation in patients with a history of relapses and switching from a BRACE therapy (IFN-β or glatiramer acetate) to fingolimod or another BRACE therapy

Propensity scores* were used to create balanced cohorts of patients

Patients switching from a BRACE therapy to either fingolimod or another BRACE therapy, with at least one relapse in the prior 12 months

(n=518)

BRACE cohort(n=243)

Fingolimod cohort(n=275)

Unmatched cohorts

1:1 propensity matching of patient characteristics:

sex, age, disease duration, EDSS score,

pre-baseline treatment and relapse activity

Matched cohorts

BRACE cohort(n=208)

Fingolimod cohort(n=208)

*Propensity score matching is a statistical technique used to match patients in observational studies[b]

BRACE = betaferon, rebif, avonex, copaxone, extavia; EDSS = expanded disability status scale;

IFN-β =interferon beta

a. Spelman T, et al. ECTRiMS 2013. Abstract P1096.

b. Rosenbaum PR and Rubin DB. Biometrika 1983;70:41–55.


The ispor task force defined real world data as

MSBASE – Fingolimod vs BRACE Therapies:

Proportion of Patients Not Relapsed After Switching

In patients with a history of relapses on BRACE treatment, fingolimod reduced the relapse rate compared with BRACE therapies.

Time to first relapse and corresponding risk reduction over 12 months in patients with history of relapse on DMT treatment*

45%reduction vs BRACE

(P < .01)*

*Study cohorts were restricted to patients who switched from a BRACE therapy to either fingolimod or to (another) BRACE therapy and had at least 1 relapse in the prior 12 months or at least 2 in the prior 24 months.

Spelman T, et al. ECTRiMS 2013. Abstract P1096.


The ispor task force defined real world data as

MSBASE – Fingolimod vs BRACE Therapies:

Discontinuation Rate After Switching

In patients with a history of relapses on DMT treatment, fingolimod reduced the risk of discontinuing treatment compared to BRACE therapies

Time to discontinuation and corresponding risk reduction over 12 months in patients with history of relapse on DMT treatment*

78%reduction vs BRACE

(P< .01)*

*Study cohorts were restricted to patients who switched from a BRACE therapy to either fingolimod or to (another) BRACE therapy and had at least 1 relapse in the prior 12 months or at least 2 in the prior 24 months.

Spelman T, et al. ECTRiMS 2013. Abstract P1096.


The ispor task force defined real world data as

MSBASE – Real-World Data Comparing Natalizumab vs BRACE Therapies: Study Overview

"Stay or Go" analysis of 2 data sets using propensity matching

All

MSBase

TOP

Patients

3976

694

4670

Original sample

  • Selection criteria:

  • Patients on any BRACE therapy who had experienced on-treatment relapse during the prior 12 months and who:

    • Continued their BRACE therapy or

    • Switched to natalizumab

3821

694

4515

Satisfying entry criteria

Without missing values in the covariates

3743

694

4437

569

569

1138

Successfully matched

Completeness of matching 569/694 = 82%

TOP = Natalizumab Observational Program

Spelman T, et al. AAN2013. Abstract P01.211.


The ispor task force defined real world data as

MSBASE – Real-World Data Comparing Natalizumab vs BRACE Therapies: Proportion of Patients Not Relapsed

Propensity Matched

HR for BRACE vs natalizumab,

2.73; P <.001

Spelman T, et al. AAN2013. Abstract P01.211.


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