introduction to outcomes research

THE POWER OF REAL-WORLD INFORMATION

TCD OUTCOMES RESEARCH © 2016 | 1



OBJECTIVES

It is NOT to create OR specialists ….

Use a product/industry life-cycle perspective ….

Using OR smartly ....



• Framing Outcomes Research (OR)

• Techniques

• Case studies

• Application

CONTENTS:



1. FRAMING OUTCOMES RESEARCH

In your view:

• Why are you here today?

• What is OR?

• Where, if at all, should OR reside in

your organization?

FRAMING OR

DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS

| 6TCD OUTCOMES RESEARCH © 2016

• New questions from regulators, geographies, insurers, payers, patients, providers

• Disruptive new technologies: New pressures on price (NDOH Pricing Committee)

• New pressures on illustrating value

• Internal and external validity

MARKET ACCESS ANDREIMBURSEMENT CHALLENGES

FRAMING OR



EVOLUTION OF EVIDENCE

Dynamic Solutions to Dynamic QuestionsTM

Source: http://www.quintiles.com/~/media/library/presentations/the-emerging-role-of-real-world-studies-and-registries.pdf

FRAMING OR


| 8TCD OUTCOMES RESEARCH © 2016 Dynamic Solutions to Dynamic QuestionsTM

FRAMING OR

DRUG DEVELOPMENT CYCLE



FRAMING OR

COMMERCIALIZATION CYCLE

High-value vs. non-high-value

assets



FRAMING OR

DYNAMIC SOLUTIONS TO DYNAMIC QUESTIONS™



FRAMING OR

THEREFORE …

• What is OR? • What makes it different?

• How can OR contribute towards policy?

?



FRAMING OR

• Outcomes Research “seeks to understand the end results of particular health care practices and interventions” on individual patients and populations

• Often includes:

» Clinical impact

» Economic impact

» Financial impact

» Funding impact

» Market impact

» Public health impact

Clinical impact

Economic impact

Financial impact

Funding impact

Market impact

Public health impact

WHAT IS OR??



FRAMING OR

1. The Question It AsksA hallmark of outcomes research is the breadth of issues it addresses. Outcomes research touches all aspects

of health care delivery, from the clinical encounter itself to questions of the organization, financing and

regulation of the health care system. Each of these factors plays a role in the outcome of care, or the ultimate

health status of the patient. Understanding how they interact requires collaboration among a broad range

of health services researchers, such as physicians and nurses, economists, sociologists, political scientists,

operations researchers, biostatisticians and epidemiologists.

2. The Setting It StudiesOutcomes research evaluates the results of the health care process in the real-life world of the doctor’s office,

hospital, health clinic and even the home. This contrasts with traditional randomized controlled studies,

funded mainly through the National Institutes of Health, which test the success of treatments in controlled

environments. These are called efficacy studies. Research in real-life settings is called effectiveness research.

WHAT MAKES IT DIFFERENT?



FRAMING OR

3. The Health Status Measures It UsesTraditionally, studies have measured health status, or health outcomes, in terms of physiological measurements

– through laboratory test results, complication rates (e.g. infections) or death. These measures alone do not

adequately capture health status. A patient’s functional status, well-being, and satisfaction with care must

compliment the traditional measures.

4. The Methods It UsesConstraints of money and the time it takes to conduct large, long term clinical trials is a major challenge of

outcomes research. One strategy of overcoming this barrier is to use existing computerized databases, such as

those available from Medicare, Medicaid, private insurance companies, and major employers. Another strategy

is to use simple questionnaires that patients can use to evaluate their own health and health care. Outcomes

research also uses meta-analysis, a technique to summarize comparable findings from multiple studies.

These approaches complement the more standard research strategies involving the collection and analysis of

information from medical records and other clinical and management data.

WHAT MAKES IT DIFFERENT?



FRAMING OR

CONTRIBUTION TO POLICY

• Global policy trends …

• Country policy

• Funder policy

• Clinical policy

Bridge the gap between clinical and commercial functions, throughout the product life-cycle. By infusing insights from the marketplace in the form of real-world data into the development process, companies can better align their development efforts with unmet patient needs and accelerate time to market with the right evidence package. In the post-marketing realm, collecting more data on real patients in a real-world setting is becoming the new mandate.



FRAMING OR TECHNIQUES

2. OUTCOMES RESEARCH TECHNIQUES




TECHNIQUESOur armamentarium:

• Systematic review and Meta-analysis

• Health economics

• RWD analysis

• Observational studies

• Comparative effectiveness trials

• Patient reported outcomes

• Phase IIIB and IV studies




TECHNIQUES: SCIENTIFIC METHODOLOGY

Just because it is OR, does not mean we do not need scientific rigor …

The research question: Why are red cherries black?

Hypothesis: Because they’re green.

• Protocol development, PI and GCP

• Regulatory

• Ethics

• Project management

• Site management, CRF, data collection

• Data management

• Biostatistics

• Medical writing: Clinical study Report

• Value messages related to different stakeholders




TECHNIQUES: RWD ANALYSISExamples:

• Retrospective data review

» Medical schemes claims data

» Expanded access programs

• Retrospective chart review

Types of questions answered:• Total cost of illness

• Patient flow modeling

• Adherence

• Data inputs for PE modeling

• Epidemiology

• Medicine initiation and switching rates

• Competitor analysis

• Local patient outcomes




TECHNIQUES: RWD ANALYSIS

HOW?:• Research question

• Primary and secondary objectives

• Data specification

• SAP

• Data collection and analysis

• Results

• Impact and value messages




TECHNIQUES: OBSERVATIONAL STUDIES

Involves the direct observation of phenomena in their natural setting

Examples:

• Registries

• Epidemiological studies




TECHNIQUES: OBSERVATIONAL STUDIES

Registries:



FRAMING OR

A PATIENT REGISTRY is an organized system that uses observational study methods to collect uniform

data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease,

condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy Purposes.

A REGISTRY DATABASE is a file (or files) derived from the registry.

FOCUSED on the outcomes, effectiveness, comparative clinical effectiveness, and appropriateness of

pharmaceuticals, devices, and health care services.

DEFINITIONS

TECHNIQUES



FRAMING OR

PROCESS

Creating registries:

• Planning

• Design

• Data elements

• Use

• Data sources

Source:

https://effectivehealthcare.ahrq.gov/ehc/products/420/1897/

registries-guide-3rd-edition-vol-1-140430.pdf

TECHNIQUES



FRAMING OR

PROCESS

The key points to consider in designing a registry include:

• formulating a research question;

• choosing a study design;

• translating questions of clinical interest into measurable exposures and outcomes;

• choosing patients for study, including deciding whether a comparison group is needed;

• determining where data can be found; and

• deciding how many patients need to be studied and for how long.

TECHNIQUES



FRAMING OR

PROCESS

TECHNIQUES

Ethical considerations:

• Ethics, data ownership, privacy

• Patient informed consent

• Data protections (FDA CRF Part 11 compliance)

http://www.fda.gov/RegulatoryInformation/Guidances/ucm125067.htm



FRAMING OR

PROCESS

TECHNIQUES

Operating registries:

• Recruiting and retaining patients

• Data collection and QA

• AE’s recording and reporting

• Data analysis

• Medical writing




SCIENTIFIC RIGOR

https://www.graceprinciples.org/doc/GRACE-Checklist-031114-v5.pdf




Static Registries to Dynamic eRegistriesTM




Static vs. Dynamic Registries




• Paper based CRF

• Transcription into e-CRF

• Significant monitoring

• Data management cumbersome

• Takes time: high touch, low tech

• Reporting is laborious

Static Registries




E-REGISTRY:




Computational Model + E-Registry = Dynamic e-Registry

DYNAMIC e-REGISTRIES




DYNAMIC e-REGISTRIES

• Automated descriptive analyses: Simple descriptive statistics

• Automated predictive analyses: Predict heterogeneity of treatment effect; predict primary/secondary outcomes

• Automated prescriptive analyses: Machine learning - Prescribe what patient characteristics will provide best outcomes

• Dashboards: Create bespoke dashboards as required by stakeholders

• Automated exposure to stakeholders: All technology platforms




TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)

“While the implementation of CER has

implications across global healthcare

systems, the US focus on CER points to the

growing influence of payers and their desire

to control spending.”1

1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0




TECHNIQUES: COMPARATIVE EFFECTIVENESS TRIALS (CER)

“In an era marked by increasing prominence

of CER evidence, successful pharmaceutical,

biotechnology and medical device manufacturers

will need to develop products with unambiguous

evidence of economic as well as clinical value

relative to alternatives.”1

1. Nellesen et al. Pharmacoeconomics 2010; 28 (10): 789-798, 1170-7690/10/0010-0789/$49.95/0



FRAMING OR

“CER is defined as research comparing interventions in the real world settings”1

1. http://www.med.upenn.edu/sleepctr/documents/FederalCoordinatingCoucilforCER_2009.pdf

CER DEFINITION

TECHNIQUES




• Non-inferiority trials • Superiority trials • Observational studies

CER EXAMPLES




TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)

“PRO is a health outcome directly reported by the patient

who experienced it.”




TECHNIQUES: PATIENT REPORTED OUTCOME STUDIES (PRO)

Examples:

• Symptoms (impairment)

• Functioning (disability)

• General health perceptions

• Quality of life

• Rating health care



FRAMING OR TECHNIQUES CASE STUDIES

3. CASE STUDIES

Question:

• Can you think of a specific clinical/competitive issue that might be addressed by using OR techniques?

• Would you like to use your specific problem as a case study for this session?




SETTING THE CASE STUDY

CONTEXT:

• As product manager, you are informed that, in 2018, casestudymab, your company’s MAB in the treatment of prostate cancer, will be launched in SA

• You are told it will cost approximately US$40,000 per treatment

• There are at least 3 competitors in this setting in SA

• Their comparative cost is about US$25,000 per treatment

Using the knowledge gained in this session, how will you proceed?




FRAMING THE RESPONSE

Activity Activity plan

Pre-launch activities

Launch activities

Post-registration activities



FRAMING OR TECHNIQUES CASE STUDIES APPLICATION

4. APPLICATION




4. APPLICATION

CONTEXT:

• Clinical affairs

• Medical affairs

• Regulatory affairs

• Market access

• Commercial




4. APPLICATION

THE BIG OR OBJECTIVE:

• It is part of your competitive strategy …

• It is about building a comprehensive portfolio of real world evidence …

• That can be used by all departments …

• To reach commercial objectives of market access and reimbursement ...

• Using S.M.A.R.T. Goals.



“In times of change the learners shall inherit the earth … while the learned will find

themselves perfectly equipped to manage a world that no longer exist” – Eric Hoffer

[email protected]

www.tcd-global.com

Dynamic Solutions to Dynamic QuestionsTM

introduction to outcomes research

Health & Medicine