RetiredCareSourcePolicy N/A

Experimental or Investigational Item or Service

Defines CareSource Arkansas PASSE policy for determining when a health care item or service is considered experimental or investigational and therefore not covered; affects CareSource members and providers under the Arkansas PASSE program.

Policy Summary

PayerCareSource

PolicyExperimental or Investigational Item or Service

Policy CodePolicy N/A

Change TypeAdded Phase 4 drug exception

Effective Date07/01/2023

Next Review DateN/A

Key ActionDo not authorize items or services determined to be experimental or investigational; provide supporting literature, FDA/IRB documents, consent/protocols, medical records, or expert opinions when requesting review.

SourceLink

POLICY UPDATE CHANGES

Added Phase 4 drug exception to Definitions and Clinical Trial Coverage policy to sec. F; updated references.

07/01/2023 - 10/31/2023Effective range

May be coveredPhase 4 exception

9 conditionsDefinition criteria count

ArchivedPolicy status

Coverage Criteria

Not Covered - Experimental/Investigational

Not covered when any of the following apply:

Primary exclusion: Any health care item or service CareSource determines in its sole discretion to be experimental or investigational is not covered.

See Definitions (Section C) for enumerated conditions

Definition criteria: Item meets one or more Definition conditions: not approved by the U.S. Food and Drug Administration for the proposed use; not identified in the American Hospital Formulary Service or the U.S. Pharmacopeia Dispensing Information as appropriate for the proposed use; determined by the FDA to be contraindicated for the specific use; subject to institutional review board review/approval for the proposed use and final approval has not been granted; the subject of an ongoing Phase 1, 2, or 3 clinical trial; provided as part of a clinical research protocol or clinical trial intended to evaluate safety, toxicity, or efficacy; or described as experimental or investigational in informed consent documents.

Devices FDA-approved under the Humanitarian Use Device exemption are not considered experimental or investigational.

Policy Summary

PayerCareSource

PolicyExperimental or Investigational Item or Service

Policy CodePolicy N/A

Change TypeAdded Phase 4 drug exception

Effective Date07/01/2023

Next Review DateN/A

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Provider Actions and Review Considerations

Denial Risk

Coverage exclusion for experimental/investigational items — highlight that such items/services are not covered; reference Definitions and evidence review.

CareSource does not cover items or services it determines to be experimental or investigational. Experimental or investigational items or services include medical, surgical, diagnostic, psychiatric, substance use disorder treatments, technologies, equipment, supplies, procedures, therapies, biologics, drugs, or devices that at the time CareSource makes a coverage determination: are not FDA‑approved for the proposed use; are not identified in the American Hospital Formulary Service or the United States Pharmacopoeia Dispensing Information as appropriate for the proposed use; are determined by the FDA to be contraindicated for the specific use; are subject to institutional review board review for the proposed use and such final approval has not been granted; are the subject of an ongoing Phase 1, 2, or 3 clinical trial; are provided as part of a clinical research protocol or trial or otherwise intended to evaluate safety, toxicity, or efficacy; or are provided pursuant to informed consent documents that describe the item or service as experimental or investigational. Devices approved under the FDA Humanitarian Use Device exemption are not considered experimental or investigational. Drugs used in Phase 4 trials may be covered if they are part of the formulary.

CareSource will not cover any health care item or service it determines in its sole discretion to be experimental or investigational.
Experimental or investigational is defined by criteria including FDA approval status, formulary/dispensing identification, IRB approval status, clinical trial phase, inclusion in research protocols, and informed consent language.
Humanitarian Use Device (HUD) approvals are excluded from being considered experimental/investigational.
Phase 4 drugs may be covered if they are included on the formulary.

Prior Authorization

PROVIDER ACTIONS — CareSource assesses benefit compared to established alternatives and may require supporting evidence/documentation.

When reviewing requests for items or services that may be experimental or investigational, CareSource assesses whether the item or service is supported by credible research demonstrating measurable and beneficial health outcomes compared to established alternatives. CareSource may require providers to supply supporting documentation and will evaluate whether evidence demonstrates improved net health outcomes for the population for whom the service is proposed, that benefits outweigh harms, and that outcomes are achievable under usual conditions of medical practice outside investigational settings.

CareSource assesses benefit compared to established alternatives and may require supporting evidence and documentation.
Determination includes whether evidence shows improved net health outcomes and applicability outside clinical research settings.

Documentation Required

PROVIDER ACTIONS — When reviewing requests, CareSource may consider published literature, FDA/IRB/consent/protocol documents and other clinical evidence.

CareSource may consider published peer‑reviewed literature, evaluations from national medical associations and technology assessment bodies, FDA and other regulatory documents, institutional review board documents, consent forms and study protocols, medical records, and opinions from consulting providers or experts when making coverage determinations.

Published authoritative, peer‑reviewed medical or scientific literature (or absence thereof).
Evaluations from national medical associations, consensus panels, and technology evaluation bodies.
FDA and other regulatory agency documents and filings.
Institutional review board documents, consent documents, and study protocols.
Medical records and opinions of consulting providers and other experts.

Denial Risk

PROVIDER ACTIONS — CareSource will not cover any item/service it determines in its sole discretion to be experimental or investigational.

CareSource will not cover any item or service it determines in its sole discretion to be experimental or investigational.

Background

Experimental or investigational items or services encompass a broad range of health care interventions, including medical, surgical, diagnostic, psychiatric, and substance use disorder treatments, as well as technologies, equipment, supplies, biologics, drugs, devices, procedures, and therapies. Items meet this definition when, at the time CareSource makes a coverage determination, they are not FDA‑approved for the proposed use; not identified in AHFS or USP Dispensing Information as appropriate for the use; determined by the FDA to be contraindicated for the use; subject to IRB review without final approval; the subject of ongoing Phase 1, 2, or 3 clinical trials; provided as part of a research protocol or trial to evaluate safety/toxicity/efficacy; or described as experimental in informed consent documents. Note that drugs in Phase 4 trials may be covered if they are part of the formulary, and devices FDA‑approved under the Humanitarian Use Device exemption are not considered experimental.

Definitions

Experimental or Investigational — definition details

FDA approval statusHealth care items/services not approved by the FDA for the proposed use are considered experimental or investigational.

Authoritative reference absenceItems not identified in the AHFS or USP Dispensing Information as appropriate for the proposed use meet investigational criteria.

FDA contraindicationItems determined by the FDA to be contraindicated for the specific use are considered investigational for that use.

IRB review statusItems subject to review/approval by an institutional review board (or similar body) where final approval has not been granted are investigational.

Research/consent designationItems described as experimental/investigational in informed consent or provided to evaluate safety, toxicity, or efficacy are investigational.

Humanitarian Use Device exemptionDevices FDA‑approved under the Humanitarian Use Device exemption are not considered experimental or investigational.

OpenPayer

Experimental or Investigational Item or Service

Coverage Criteria

Coding and Trial Phases

Provider Actions and Review Considerations

Background

Definitions