gsk u.s. public policy position paper communicating ... · pdf filea publication of gsk public...

A Publication of GSK Public Policy and Government Relations

Population health decision makersdevelopers, compendia publishers, hospital pharmacy & therapeutics committees,

GSK U.S. Public Policy Position Paper

Communicating Effectiveness Research and Evidence with PopulationMakers The Issue Many healthcare and research organizationsand use evidence beyond what is typically product approvaliii, including comparative effectiveness research (CER) and real world evidence (the growing electronic data infrastructure improves access to data, policymakers and healthcare purchasers continue to explore ways to achieve highvalue, high-quality healthcare to inform population health decision making. CER and RWE help to fill an “evidence gap” by providing decision makers with more comprehensive information to supplement Randomized Clinical Trial (RCT) data to determine which treatment options are best for patients and ensure innovative, high-quality care. The biopharmaceutical industry generatesamount of high-quality data that adheres to wellaccepted research standards. Through the medicine discovery and development process, the biopharmaceutical industry acquires deep expertise that can provide a greater understanding of a disease, its treatment patterns and affect on paas product performance, to meaningfully contribute to population health decision makingcurrent regulatory environment does not allow for effective

GSK Position We believe that proactive communication of biopharmaceutical industry, can significantly benefit public health bymakers with an expanded evidentiary basregulatory guidance is needed to define real world research that can be appropriately shared with population health decision makers


decision makers include government agencies, payers, healthcare systems, guideline developers, compendia publishers, hospital pharmacy & therapeutics committees, and group purchasing

organizations.

GSK U.S. Public Policy Position Paper

ing Comparative Effectiveness Research and Real World

ith Population Health Decision

organizations generate typically required for

comparative effectiveness world evidence (RWE). As

the growing electronic data infrastructure improves olicymakers and healthcare

purchasers continue to explore ways to achieve high-quality healthcare to inform population

CER and RWE help to fill an decision makers with more

rmation to supplement Randomized data to determine which treatment

options are best for patients and ensure access to

generates a significant hat adheres to well-

Through the medicine discovery and development process, the biopharmaceutical industry acquires deep expertise that can provide a greater understanding of a disease, its treatment patterns and affect on paas product performance, to meaningfully contribute to population health decision making.iv

regulatory environment does not allow for effective communication of this data.

communication of CER and RWE by healthcare stakeholders, including the significantly benefit public health by providing population health decision

with an expanded evidentiary base to match the right treatment with the right patient. define uniform methodology standards for comparative effectiveness and

that can be appropriately shared with population health decision makers

Research Type

Definition

CER

Comparative effectiveness research is comparative by design, and evaluates the benefits and harms of alternative methodsprevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care

RWE

Real world evidence from Real World Data via analytics, observational studies, and pragmatic trialsoutcomes such as clinical, economic, and patientIt seeks to include data that is collected outside of an RCT that is more generalizable to a “reaworld” setting; eregistries, administrative claims databases, surveys, and medical records.ii


include government agencies, payers, healthcare systems, guideline group purchasing

Real World Decision

that can provide a greater understanding of a disease, its treatment patterns and affect on patients, as well iv However, the

by healthcare stakeholders, including the population health decision

match the right treatment with the right patient. However, effectiveness and

that can be appropriately shared with population health decision makers.

Definition

effectiveness omparative by design,

and evaluates the benefits and ternative methods to

prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.i Real world evidence is derived from Real World Data via analytics, observational studies, and pragmatic trials, assesses outcomes such as clinical, economic, and patient-reported. It seeks to include data that is collected outside of an RCT that is more generalizable to a “real-

; e.g. patient registries, administrative claims databases, surveys, and medical


Key Points • Currently, there is a lack of clear regulatory standards for defining, generating and proactively

communicating CER and RWE to population health decision makers.Administration (FDA) to provide guidance on when, how, and to whom industry is permitted to proactivelshare CER and RWE information.

• Timely and accurate information from comparative, observational, and/or retrospective studies, in addition to traditional RCT data, can help providersand ultimately improve patient outcomes

• The development of consensus standardsCER and RWE data ensures all healthcare stakeholders ensure soundness of evidence.

• An environment that promotes collaborationinformation enhances access to data and evolving value-based healthcare syst

Evidence Communication Policy Opportunities • Advocate through the House Energy & Commerce Committee’s

to data to drive innovation (FDA Sentinel, PCORNet, CMS data, NIH Collaboratory Network, etc.), well as legislation and regulation that encourages adoption of hinteroperability of data sources to increase the capacity and

• Collaborate with the Pharmaceutical Research and ManufacturersBiotechnology Industry (BIO) to support and RWE to payers, formulary commit

• Advance the work of the Medical Imanufacturers, to consider issues relating to the federal government’s regulation of truthful, nonmisleading, scientifically substantiated manufacturer communications about new uses of approved drugs and approved/cleared medical devices

i Institute of Medicine. (2009). Initial National Priorities for Comparative Effectiveness ResearchEffectiveness Prioritization. National Academies Press.http://www.iom.edu/Reports/2009/ComparativeEffectivenessResearchPriorities.aspii Garrison, L.P., Neumann, P.J., Erickson, J., Marshall, D., Mullins, C.D. (2007). Decisions: The ISPOR Real-World Data Task Force ReportAvailable at: http://www.ispor.org/workpaper/RWD_TF/ISPORRealWorldDataTaskForceReport.pdfiii Drug approval requires safety and effectiveness din Section 505(d) of the Federal Food, Drug and Cosmetic ActGenerally at least two adequate and well-controlled studies are required to establish effectiveness. Congress amended the Act to clarify that FDA may consider data from one study and confirmatory evidence to constitute substantial evidence if FDA determisuch evidence is sufficient to establish effectivenessiv Medical Information Working Group (MIWG) to FDA’s Regulation of Manufacturer Speech. Ahttp://www.regulations.gov/contentStreamer?objectId=090000648192b34f&disposition=attachment&contentType=pdf


regulatory standards for defining, generating and proactively communicating CER and RWE to population health decision makers. GSK urges the Food and Drug

to provide guidance on when, how, and to whom industry is permitted to proactivelshare CER and RWE information. Timely and accurate information from comparative, observational, and/or retrospective studies, in addition

data, can help providers, payers, and patients make more informed treatment ultimately improve patient outcomes.

consensus standards by the FDA (using a multi-stakeholder bodyll healthcare stakeholders are held to equally rigorous

promotes collaboration through the appropriate sharing of accurate, science

information enhances access to data and better informs conversations to ensure quality outcomes in an based healthcare system.

Evidence Communication Policy Opportunities House Energy & Commerce Committee’s 21st Century Cures initiative

Sentinel, PCORNet, CMS data, NIH Collaboratory Network, etc.), on that encourages adoption of health information technology

to increase the capacity and use of real world data. Pharmaceutical Research and Manufacturers of America (PhRMA

Biotechnology Industry (BIO) to support increased regulatory clarity concerning communication of to payers, formulary committees, or other similar entities.

Information Working Group (MIWG), a coalition of medical product consider issues relating to the federal government’s regulation of truthful, non

misleading, scientifically substantiated manufacturer communications about new uses of approved approved/cleared medical devices.

Updated

Initial National Priorities for Comparative Effectiveness Research. Committee on National Academies Press. Available at:

http://www.iom.edu/Reports/2009/ComparativeEffectivenessResearchPriorities.aspx Garrison, L.P., Neumann, P.J., Erickson, J., Marshall, D., Mullins, C.D. (2007). Using Real-World Data for Coverage and Payment

World Data Task Force Report. International Society for Pharmacoeconomics and Outcomes Research. http://www.ispor.org/workpaper/RWD_TF/ISPORRealWorldDataTaskForceReport.pdf

Drug approval requires safety and effectiveness data, where a drug’s effectiveness is established by “substantial evidence” defined in Section 505(d) of the Federal Food, Drug and Cosmetic Act (FFDCA) as “consisting of adequate and well-controlled investigations”.

controlled studies are required to establish effectiveness. Congress amended the Act to clarify that FDA may consider data from one study and confirmatory evidence to constitute substantial evidence if FDA determi

stablish effectiveness. White Paper. (2014). Systemic, Societal, and Legal Developments Require Changes

. Available at: http://www.regulations.gov/contentStreamer?objectId=090000648192b34f&disposition=attachment&contentType=pdf


regulatory standards for defining, generating and proactively Food and Drug

to provide guidance on when, how, and to whom industry is permitted to proactively

Timely and accurate information from comparative, observational, and/or retrospective studies, in addition more informed treatment decisions

stakeholder body) for generating equally rigorous measures to help

appropriate sharing of accurate, science-based ensure quality outcomes in an

initiative for access Sentinel, PCORNet, CMS data, NIH Collaboratory Network, etc.), as

echnology (HIT) and

of America (PhRMA) and the increased regulatory clarity concerning communication of CER

alition of medical product consider issues relating to the federal government’s regulation of truthful, non-

misleading, scientifically substantiated manufacturer communications about new uses of approved

Updated February 2015

Committee on Comparative

World Data for Coverage and Payment and Outcomes Research.

ata, where a drug’s effectiveness is established by “substantial evidence” defined controlled investigations”.

controlled studies are required to establish effectiveness. Congress amended the Act to clarify that FDA may consider data from one study and confirmatory evidence to constitute substantial evidence if FDA determines that

Systemic, Societal, and Legal Developments Require Changes

http://www.regulations.gov/contentStreamer?objectId=090000648192b34f&disposition=attachment&contentType=pdf.

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