THE BILL ITSELF
HB 1381
Experimental Treatments for Terminal Conditions and Life-threatening Rare Diseases
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A bill to be entitled
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An act relating to experimental treatments for
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terminal conditions and life-threatening rare
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diseases; providing a short title; amending s.
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499.0295, F.S.; providing legislative findings and
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intent; defining terms; authorizing licensed
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physicians to prescribe and eligible facilities to
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administer experimental treatments if certain
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requirements are met; requiring the department to
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adopt certain rules; requiring a specified written
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informed consent from eligible patients; requiring
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approval of a specified platform or master protocol by
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an institutional review board; requiring the
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Department of Health to establish and maintain a
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patient registry for specified purposes; requiring
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eligible facilities to submit certain information to
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the registry; requiring the department to enter into
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specified contracts with eligible facilities;
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providing a penalty for breach of such contact;
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requiring the department to annually publish a
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specified report; encouraging health insurers and
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health maintenance organizations to provide specified
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insurance coverage for experimental treatments;
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prohibiting such insurers and organizations from
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denying insurance coverage for experimental
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treatments; providing construction; authorizing
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licensed physicians and eligible facilities to receive
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reimbursement for the prescription or administration
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of experimental treatments if certain requirements are
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met; establishing the reimbursement rate; requiring
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the department to adopt specified rules; providing for
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reimbursement rate expiration; requiring manufacturers
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of experimental treatments to provide for a specified
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temporary price concession set by the department;
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providing for expiration of such concession;
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authorizing the Department of Management Services to
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enter into a specified contract with an eligible
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facility or manufacturer; requiring the department to
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adopt a specified rule for such contract; requiring
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the Department of Health to adopt by rule certain
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procedures for licensing of experimental treatment
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centers; requiring the department to approve or deny a
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completed application within a specified timeframe;
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requiring the department to establish by rule certain
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procedural and operational standards; prohibiting
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certain actions by specified licensing boards and a
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specified state agency against a licensed physician in
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certain circumstances; providing construction and
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applicability; providing for limitation of liability;
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providing that certain participation is entirely
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voluntary at all times; prohibiting public officials,
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public employees, and public agents from denying or
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attempting to deny access to experimental treatment;
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providing construction; requiring the department to
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adopt rules; providing an effective date.
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Be It Enacted by the Legislature of the State of Florida:
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Section 1. "This act may be cited as the "Promising
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Pathways Act."
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Section 2. Section 499.0295, Florida Statutes, is amended
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to read:
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(Substantial rewording of section. See
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s. 499.0295, F.S., for present text.)
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499.0295 Experimental treatments for terminal conditions
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and life-threatening rare diseases.—
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(1) The Legislature finds that residents of this state
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with terminal conditions and life-threatening rare diseases may
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benefit from access to experimental treatments that have
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demonstrated initial human safety but are not yet approved by
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the United States Food and Drug Administration.
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(2) It is the intent of the Legislature to:
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(a) Create a safe, regulated, and transparent pathway to
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experimental treatments through licensed state institutions with
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board oversight.
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(b) Condition payment on evidence development.
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(c) Encourage coverage parity by state-regulated health
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plans.
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(d) Align temporary pricing during the conditional period
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with the maturity of clinical evidence.
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(3) As used in this section, the term:
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(a) "Board" means the Board of Medicine, created under
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chapter 458, or the Board of Osteopathic Medicine, created under
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chapter 459, as applicable.
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(b) "Department" means the Department of Health, created
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under s. 20.43.
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(c) "Eligible facility" means a hospital or health care
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facility licensed under chapter 395 that operates under the
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federal policy for the protection of human subjects under 45
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C.F.R. part 46 and maintains, or participates under,
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Institutional Review Board oversight for experimental
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treatments.
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(d) "Eligible patient" means a person who:
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1.a. Has a terminal condition that is attested to by the
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treating physician and confirmed by a board-certified physician
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in an appropriate specialty for that condition; or
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b. Has a life-threatening rare disease that is attested to
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by the treating physician and confirmed by a board-certified
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physician in an appropriate specialty.
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2. Has considered and exhausted all other treatment
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options for the terminal condition or life-threatening rare
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disease currently approved by the United States Food and Drug
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Administration.
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3. Has given written informed consent pursuant to
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subsection (4)(c).
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4. Has documentation from the treating physician that the
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patient meets the requirements of this paragraph.
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(e) "Experimental treatment" means:
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1. A drug, biological product, or medical device that has
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successfully completed phase 1 of a clinical trial but has not
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been approved for general use by the United States Food and Drug
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Administration (FDA) and remains under investigation in a
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clinical trial approved by the FDA; or
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2. Personalized treatment for use by a patient based on
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his or her genomic or molecular profile, including, but not
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limited to, gene-targeted therapies, antisense oligonucleotides
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(ASOs)-mediated therapies, and neoantigen-targeting personalized
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cancer vaccines (PCVs), which have not been approved for general
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use by the FDA and remain under investigation in a clinical
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trial approved by the FDA.
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(f) "Institutional Review Board" or "IRB" has the same
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meaning as in s. 381.86.
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(g) "Patient registry" or "registry" means a clinical data
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collections system from which health information is collected at
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specific points during the course of treatment and all personal
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identifiers have been removed or obscured to protect patient
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privacy.
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(h) "Rare life-threatening disease" means a progressive
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disease or medical or surgical condition that affects less than
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200,000 persons in the United States. The term includes any
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progressive disease or medical or surgical condition that meets
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the criteria of a rare disease under 21 U.S.C. s. 360bb.
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(i) "Terminal condition" means a progressive disease or
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medical or surgical condition that causes significant functional
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impairment, is not considered by a treating physician to be
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reversible even with the administration of available treatment
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options currently approved by the FDA, and, without the
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administration of life-sustaining procedures, will result in
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death within 1 year after diagnosis if the condition runs its
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normal course.
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(4)(a) A physician licensed under chapter 458 or chapter
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459 may prescribe to an eligible patient an experimental
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treatment pursuant to this section.
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(b)1. An eligible facility may administer to an eligible
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patient an experimental treatment if such treatment is
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administered pursuant to a platform or master protocol approved
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by an IRB pursuant to subsection (5) and complies with the
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registry requirements established in subsection (6).
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2.a. An eligible facility that does not maintain its own
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IRB may establish and maintain an affiliation with another
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eligible facility that maintains IRB oversight.
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b. The department shall adopt rules for affiliations
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pursuant to this subparagraph, to include, but not be limited
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to, the handling of drugs, adverse-event escalation, quality
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assurance, and health data submission.
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(c) Written informed consent, signed by an eligible
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patient, a court-appointed guardian for an eligible patient, or
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a health care surrogate designated by an eligible patient, is
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required, and must include:
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1. An explanation of the currently approved treatments for
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the patient's terminal condition or life-threatening rare
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disease and that such treatments have been considered and
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exhausted.
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2. Identification of the specific experimental treatment
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that the patient is seeking to use.
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3. A description of the potential risks and benefits of
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using the experimental treatment, including best-case outcome
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and worst-case outcome. The description must be based on the
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physician's knowledge of the experimental treatment for the
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patient's terminal condition or life-threatening rare disease.
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4. A statement that the patient's health plan or third-
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party administrator and physician are not obligated to pay for
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care or treatment consequent to the use of the experimental
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treatment unless required to do so by law or contract.
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5. A statement that the patient's eligibility for hospice
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care may be affected by the use of experimental treatment.
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6. A statement that the patient understands he or she is
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liable for all expenses consequent to the use of the
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experimental treatment but that liability does not extend to the
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patient's estate, unless a contract between the patient and the
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manufacturer states otherwise.
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7. An authorization from the patient to release his or her
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health information, collected at specific points during
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treatment, for submission into the registry established in
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subsection (6).
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(5) A platform or master protocol must be approved by an
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IRB under which treating physicians and eligible facilities may
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adapt dosing, sequencing, or combinations of authorized
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experimental treatments, within predefined limitations, without
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seeking approval of an IRB for each adaptation. The platform or
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master protocol must specify safety limits, stopping
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requirements, and patient registry information pursuant to
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subsection (6).
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(6)(a) The department shall establish and maintain a
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patient registry for the collection and analysis of experimental
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treatment outcomes under this section. Eligible facilities shall
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submit health information to the registry from which all
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personal identifiers have been removed or obscured to protect
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patient privacy.
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(b) The department shall enter into a written contract
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with eligible facilities which specifies the scope of services
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provided, the service level, the duration of the agreement, the
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responsible parties, and the service costs. Failure to meet the
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contract requirements may result in the filing of an action by
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the department and a temporary suspension of reimbursement for
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the prescription or administration of experimental treatment
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pursuant to subsection (8).
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(c) The department shall annually publish a public report
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of experimental treatment outcomes and safety signals in the
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aggregate.
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(7)(a) A health insurer or a health maintenance
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organization regulated under the Florida Insurance Code are
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encouraged to provide insurance coverage for experimental
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treatments in the same manner as any other covered treatment or
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therapy.
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(b) A health insurer or a health maintenance organization
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may not deny insurance coverage based solely on a treatment
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being experimental or denied approval for general use by the
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FDA.
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(c) This subsection does not require insurance coverage
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and does not expand the coverage an insurer or health
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maintenance organization must provide under the Florida
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Insurance Code.
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(8)(a) A licensed physician or an eligible facility may
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receive reimbursement for the prescription or administration of
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an experimental treatment under this section. However, in order
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to receive such reimbursement, an eligible facility must
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actively participate in the patient registry established under
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subsection (6). Failure to participate in the patient registry
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may result in temporary suspension of reimbursement until
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compliance is restored.
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(b)1. Reimbursement for a prescribed or administered
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experimental treatment under this subsection may not exceed a
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discounted rate set by the department as a percentage of a
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recognized pricing benchmark, including wholesale acquisition
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cost or average sales price.
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2. The department shall adopt rules that:
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a. Establish an allowable discount band to reflect
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evidentiary uncertainty.
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b. Apply a registry noncompliance adjustment which
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increases the discounted rate set by the department.
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c. Provide for renewal and potential adjustment of the
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discounted rate every 2 years based on registry compliance.
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(c) Upon approval by the FDA of an experimental treatment,
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the discounted rate set by the department for reimbursement
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expires and the standard reimbursement rate applies.
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(9) A manufacturer of an experimental treatment shall
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provide for a temporary price concession, which price must be
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set by the department by rule as a percentage of a recognized
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pricing benchmark. The temporary price concession expires upon
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approval by the FDA of the experimental treatment.
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(10) An eligible facility or manufacturer and the
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Department of Management Services pursuant to s. 110.123 may
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enter into a healthcare contract under which a portion of
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payment is linked to experimental treatment outcomes. The
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department shall adopt a rule for such contract, which rule must
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provide for standard contract terms and authorize reconciliation
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intervals not to exceed 2 years.
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(11)(a) The Department of Health shall adopt by rule
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procedures for licensing experimental treatment centers and
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shall approve or deny a completed application within 90 days.
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(b) The department shall establish by rule the following
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minimum procedural and operational standards:
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1. Written policies and procedures.
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2. Oversight and governance standards.
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3. Inspection and safety standards.
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4. Staff training provisions.
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5. Recordkeeping provisions.
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6. Data quality assurance.
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(12)(a) A licensing board may not revoke, deny renewal,
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suspend, or take any action against a physician's license issued
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under chapter 458 or chapter 459 based solely on the physician's
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recommendations to an eligible patient regarding access to or
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treatment with experimental treatment authorized under this
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section. A state agency responsible for Medicare certification
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may not take action against a physician's Medicare certification
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based solely on the physician's recommendation that an eligible
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patient have access to experimental treatment.
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(b)1. This section does not create a private cause of
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action:
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a. Against a manufacturer of an experimental treatment;
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b. Against a person or an entity involved in the care of
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an eligible patient who is using an experimental treatment; or
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c. For any harm to the eligible patient that is a result
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of the use of an experimental treatment,
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if the manufacturer or other person or entity complies in good
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faith with the terms of this section and exercises reasonable
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care.
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2. This paragraph does not apply to judicial action
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brought for gross negligence or intentional, willful, or wanton
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misconduct.
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(c) If an eligible patient dies while using experimental
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treatment pursuant to this section, the patient's heirs are not
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liable for any outstanding debt related to the patient's use of
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such treatment.
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(d) A hospital or licensed health care facility is not
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required to provide new or additional health care services or
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treatments under this section without prior consent of the
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hospital or licensed health care facility. Participation
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pursuant to this section is entirely voluntary at all times.
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(e) A public official, a public employee, or an agent of
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this state, or any political subdivision thereof, may not deny
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or attempt to deny access to experimental treatment under this
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section. Counseling, advice, or recommendations by a licensed
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physician consistent with the standard of care do not constitute
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the denial of access or the attempted denial of access to
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experimental treatment under this paragraph.
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(13) This section does not authorize the possession, use,
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dispensing, or administration of a controlled substance in
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violation of chapter 893, except to the extent expressly
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permitted by state or federal law.
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(14) The department shall adopt rules pursuant to ss.
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120.536(1) and 120.54 to implement this section.
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This act shall take effect upon becoming a law. Section 3.