Description
Clinical trials are research studies, conducted with patients, that are designed to answer scientific questions and to achieve multiple ends. Clinical trials may or may not involve investigational treatments. Trials may:

• Evaluate the safety and efficacy of new investigational treatments in comparison to standard or conventional treatments.
• Compare differing doses of FDA-approved drugs.
• Compare different combinations of approved drugs.
• Compare two conventional treatments (e.g., tissue plasminogen activator (TPA) and streptokinase).
• Compare the timing of two standard interventions (e.g., surgery and radiation therapy).

Types of clinical trials include:

• Treatment trials, which test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
• Prevention trials, which look for better ways to prevent disease in people who have never had the disease or to prevent the disease from returning. These approaches may include medicines, vaccines, vitamins, minerals or lifestyle changes.
• Diagnostic trials, which are conducted to find better tests or procedures for diagnosing a particular disease or condition.
• Screening trials, which test the best way to detect certain diseases or health conditions.
• Quality of life trials (or supportive care trials), which explore ways to improve comfort and the quality of life for individuals with a chronic illness.

In addition to various types of clinical trials, there are several phases of clinical trials:

• In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20 – 80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
• In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100 – 300) to see if it is effective and to further evaluate its safety.
• In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000 – 3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.
• In Phase IV trials, post-marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

All research studies are not of comparable quality. While many studies are well-designed, using proper scientific methodology and avoiding bias, others are not. Data from poorly designed, poor quality studies are not meaningful and cannot be used to resolve scientific questions.

The Affordable Care Act (ACA) requires the Health Plan to provide coverage for members covered under insured, non-grandfathered health plans, for the standard of care costs associated with participation in clinical trials, for plan years beginning on or after January 1, 2014. Under the ACA, routine costs include all items and services that the Health Plan would cover for a patient not enrolled in a clinical trial. For purposes of clinical trials, the ACA defines “life-threatening” as “any disease or condition from which the likelihood of death is probable unless the course of the disease or condition is interrupted.” 

Policy

• A member requesting coverage for participation in an approved clinical trial must be eligible, under the trial’s protocol, to participate in the clinical trial, which must be related to the treatment of cancer or other life-threatening disease or condition, and either:
  • The member’s referring provider has concluded that the member’s participation in the trial is appropriate to treat the member’s life-threatening disease or condition.
  • The member provides medical and scientific information establishing that the member’s participation in the trial would be appropriate to treat the member’s life-threatening disease or condition.
• An approved clinical trial means a phase I, phase II, phase III, or phase IV clinical trial that is conducted in relation to the prevention, detection, or treatment of cancer or other life-threatening disease or condition and meets the criteria set for in A, B, or C below:
  • The clinical trial is approved or funded by any of the following:
    1. The National Institutes of Health
    2. The Centers for Disease Control and Prevention
    3. The Agency for Health Care Research and Quality
    4. The Centers for Medicare & Medicaid Services
    5. A cooperative group or center of any of the entities described in 1 through 4 above or of the Department of Defense, or the Department of Veterans Affairs
    6. A qualified non-governmental research entity identified in the guidelines issued by the National Institutes of Health for center support grants
    7. The Department of Veterans Affairs, the Department of Defense, or the Department of Energy, if the study or investigation has been reviewed and approved through a system of peer review that the Secretary of the agency determines:
       • To be comparable to the system of peer review of studies and investigations used by the National Institutes of Health.
       • Assures unbiased review of the highest scientific standards by qualified individuals who have no interest in the outcome of the review.
  • The clinical trial is conducted under an investigational new drug (IND) application reviewed by the U.S. Food and Drug Administration (FDA).
  • The clinical trial is a drug trial that is exempt from the FDA’s IND application process.
• A copy of the clinical trial protocol, including the institutional review board (IRB) approval, must be provided to the Health Plan. 

Policy Guidelines

• The routine costs of approved clinical trials that qualify for coverage are eligible for coverage. Routine costs include:
  • Items and/or services that would be provided if there were no clinical trial (e.g., conventional care such as hospital services, room and board, physician services, office visits, laboratory and diagnostic tests).
  • Items and/or services required to administer the item or service being investigated (e.g., administration of a chemotherapy drug being tested).
  • Clinical monitoring of the effects of the item and/or service being investigated.
  • Items and/or services for the prevention of complications.
  • Other MEDICALLY NECESSARY items and/or services arising from the trial (e.g., the diagnosis or treatment of complications).
• Non-routine costs include, but are not limited to, the following and are ineligible for coverage:
  • The item and/or service being investigated.
  • Items and/or services provided only to satisfy the collection and analysis of data for the trial, which are not used in the direct clinical management of the patient (e.g., monthly CT scans when only a single scan would be medically necessary, transportation to and from the study site).
  • Items and/or services inconsistent with the established standard of care for the patient’s diagnosis (e.g., the study of a new combination of drugs in order to determine the safety and efficacy of those drugs when used in combination).
  • Items and/or services provided by the research sponsors free of charge.
  • Duplicative items and/or services.

References:

1. National Institutes of Health, Department of Health and Human Services. Clinical Trials Registration and Results Information Submission. Final rule. Fed Regist. 2016 Sep 21;81(183):64981-5157.
2. US Dept of Labor Employee Benefits Security Administration. FAQs about the Affordable Care Act implementation part 40. 2019 Aug 26 [https://www.dol.gov/agencies/ebsa/about-ebsa/our-activities/resource-center/faqs/aca-part-40] accessed 11/17/20.
3. US National Institutes of Health. Clinicaltrials.gov. Clinical research trials and you. [https://www.nih.gov/healthinformation/nih-clinical-research-trials-you] accessed 11/17/20.
4. Zon R, et al. American Society of Clinical Oncology Statement on minimum standards and exemplary attributes of clinical trial sites. J Clin Oncol 2008 May 20;26(15):2562-7 [http://jco.ascopubs.org/cgi/reprint/26/15/2562] accessed 11/17/20.
5. *Key Article

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, Blue Cross Blue Shield Association technology assessment program (TEC) and other nonaffiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines. 

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