Cancer Drug Reimbursement within the Context of Clinical Trials

Cancer Drug Reimbursement within the Context of Clinical Trials
(Draft – for consultation purposes)
Version 8.0
May 17, 2013
Introduction
Clinical and cost-effectiveness factors have led most public payers to align cancer drug funding
criteria with clinical trials supporting its use. Thus, funding criteria for most drugs tend to be
highly specific, requiring use of the drug as part of a certain regimen, for a certain line of
therapy, or for use after disease progression from a certain sequence of therapies or
“pathway”.
Increasingly, public payers are being asked to reimburse publicly funded drugs when used as a
component of clinical trials research, or after the patient has participated in a clinical trial, even
when the patient is no longer eligible under the existing funding criteria. This raises questions
about how to balance the competing demands of fiscal responsibility and evidence-based
coverage versus support for clinical research activity and patient access to what are expected to
be beneficial treatments.
The Ministry of Health and Long-Term Care (MOHLTC) and Cancer Care Ontario (CCO) recognize
the central role that clinical trials research has in improving patient care and health system
performance: They provide the evidence to support better care. This has led to the
development of a draft policy document that outlines the principles, policy, and processes for
public funding consideration of cancer drugs when used within the context of clinical trials
research.
Background
Clinical trials research offer many health system and societal benefits. Trial participation allows
for early access to new therapies and promotes adoption of new standards of care.i Clinical
trials may be initiated and sponsored by either the academic community including clinical trial
groups (e.g., cooperative groups), or the pharmaceutical industry.
Four types of clinical trials exist: phase I trials evaluate the safety and dose of a drug; phase II
trials are directed towards a larger group of people to determine effectiveness and toxicities;
phase III trials are randomized controlled trials in which alternative interventions are compared
in randomly assigned populations; and phase IV trials are typically post-marketing/ postadoption surveys that provide additional information on a drug’s safety and clinical benefit.ii In
general, the evidence needed to obtain new drug licensing approval by Health Canada and
funding approval by the Ontario Ministry of Health and Long-Term Care requires the
demonstration of efficacy and/or effectiveness from testing within a phase III trial.
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Other Jurisdictions
A survey of Canadian provinces conducted by the Ministry and by CCO in 2012 indicates that
with the exception of two provinces, few policies exist on the public funding of a cancer drug
within the context of clinical trials.iiiiv Data in other countries is also limited, based on written
correspondence and a review completed by OPDP and CCO. In the United States, some federal
programs (e.g., Medicare, Veterans Affairs) and privately managed care organizations (e.g.,
Kaizer Permanente) have public policies in place.v,vi,vii These programs fund routine care costs in
cancer clinical trials provided that certain trial criteria are met.vvi,vii However, the funding of
drugs subsequent to clinical trial participation does not appear to have been addressed.
In the United Kingdom, the National Health Service (NHS) covers both treatment and support
costs (treatment costs are patient care costs that would continue to be incurred if patient care
is continued after the trial has stopped).v Finland requires that public funding criteria be met;
while New Zealand’s PHARMAC allows its District Health Boards to make discretionary rulings
on the use of publicly funded drugs within the context of trials, even if the drug is not used
within the established funding criteria, provided the drug is being used as part of a “bona fide
clinical trial which has Ethics Committee approval.”viiiix The same rule may also be applied on
occasion for patients who require access to publicly funded drugs subsequent to trial
participation.ix
Activities to Date
A policy that addresses the public funding of drugs when used within the context of clinical
trials has been under consideration by CCO and the MOHLTC for some time. An interprovincial
survey across Canada and internationally on the management of publicly funded drugs in this
setting has been completed. An environmental scan has also been conducted, in addition to
interviews with stakeholders from the medical and research community. This work is ongoing
and has supported the development of the draft policy document.
The Consultation Process
The draft clinical trial policy framework has been developed for consultation with trial
investigators, members of the research community (Ontario Institute of Cancer Research, NCIC
Clinical Trials Group, Ontario Cancer Research Ethics Board), clinicians, and pharmacists. In
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addition, the draft policy will also be shared with administrators, patient representatives, and
the pharmaceutical industry for comment
Principles
The Overriding Principles of this policy are:
1.
Ensure that patients have equitable and timely access to treatments that are safe,
offer maximum clinical benefits, and align with best practices;
2.
Ensure that coverage decisions are evidence-based, are fiscally responsible, and are
consistent with the policies of the Ontario Public Drug Programs;
3.
Support clinical trial research provided that it is consistent with the above two
principles; and
4.
Have a fair, transparent, and accountable process.
The Specific Principles associated with this policy are:
1.
Ensure Ontario citizens participating in a clinical trial have access to publicly funded
cancer drugs provided that:
a. Existing criteria for access to the funded drug are satisfied;
b. The cancer drug is to be prescribed in a manner that aligns with the funding
intent and will not be associated with incremental cancer drug costs to the
public programs; and
c. The clinical trial complies with standard regulatory and ethical requirements.
2.
Ensure Ontario citizens participating in a clinical trial, when applicable, have
subsequent access to publicly funded cancer drugs provided that:
a. This treatment aligns with the principles associated with the funding for the
drug; and
b. The subsequent use of the drug will not be associated with any incremental costs
to the public programs.
Scope
This policy applies to oral and injectable drugs currently funded by both CCO and OPDP for the
active treatment of cancer, and for supportive care drugs (e.g., colony stimulating factors) that
are part of cancer care regimens.
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The Policy for Cancer Drugs within the Context of Clinical Trials
Ontario Public Drug Programs and Cancer Care Ontario will consider requests for the
reimbursement of publicly funded cancer drugs within the context of clinical trials where the
drug is being used within the approved funding indication. No consideration will be given for a
drug or indication that is currently under review, or where the Executive Officer has decided
not to fund.
Policy criteria
All of the following criteria must be met for funding consideration:
1.
Technical Requirements:
a. The trial must be approved by a Research Ethics Board;
b. The trial must be registered in a recognized and accessible clinical trials registry,
such as clinicaltrials.gov, and any evolving Health Canada standards for clinical
trials registration;
c. Where the drug is combined with an experimental agent, the trial is associated
with a “No Objections” letter from Health Canada in response to a Clinical Trials
Application.
2.
Cost of Requested Drug
a. There are no incremental costs, or otherwise anticipated costs to the public
program have been deferred as a result of a patient’s participation in a clinical
trial that has met the technical requirements listed above.
Process
1.
Application and assessment
a. Prior approval must be obtained. An application must be filed to Cancer Care
Ontario’s Provincial Drug Reimbursement Programs that includes the link to the
clinical trials registration site, along with evidence that the trial requirements
have been met and an assessment of the public funding implications for patients
participating in the trial or subsequent to trial participation.
b. CCO will accept, track, and assess each request for its congruence to this policy
and will evaluate the funding policy implications as a result of trial participation,
both with respect to the trial in question and the subsequent patient treatment.
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c. Where necessary, clinician experts including the Provincial Disease Site Team
Heads, may be consulted for advice on safety and clinical effectiveness, and to
ensure that all funding issues have been addressed.
2. Funding Decision and Follow-up
a. CCO, in consultation with OPDP will review all requests made under this policy.
b. The Executive Officer will make all final funding decisions.
c. When a decision is made to approve a request, CCO will inform the Applicant, in
writing, of the final funding decision. CCO will also disseminate the funding
decision to the hospitals and regional cancer centres. Sites who participate in
the clinical trial may be required to submit data for adjudication purposes,
consistent with existing drug funding policies.
d. When a decision is made not to approve a request, CCO will inform the
Applicant, in writing, of the rationale for rejection.
e. Where applicable, CCO will provide trial site investigators with information on
the potential funding implications so that there may be appropriate
communication with patients about the implications of trial participation and
options for current and potential future therapy.
Expected Application of the Policy
Table 1 addresses possible funding situations that may occur during, or subsequent, to a
patient’s participation in a clinical trial along with the recommendations for management. The
management outlined describes the expected decision, based on an interpretation of the draft
policy.
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Table 1:
Use of a publicly funded drug within the conduct of a clinical trial
Scenario
Management
Clinical trial use of drug is identical
The drug will be funded. No
to the current funding policy (e.g.,
special actions are required*
control arm therapy in a Phase III
trial)
Clinical trial use of drug is for same
The drug will be funded
population as a drug that is funded; provided that the parameters
drug prescription may be altered
for conduct of clinical trial must
(e.g., dose, schedule) or may be
be satisfied**. Provision of drug
combined with another agent (e.g., cannot be associated with
experimental arm therapy in a Phase incremental cost to the publiclyIII trial)
funded system.***
Clinical trial use of drug is for an
indication that is not currently
funded (e.g., drug is tested in clinical
trial for line of therapy that does not
meet funding criteria)
Drug will not be publicy funded
Clinical trial involves re-treatment
with a previously funded drug. The
current funding criteria for that drug
do not include retreatment.
Drug will not be publicly funded
Clinical trial includes use of drug
within a Health Canada licensed
indication but funding for the drug
has not been implemented because
additional “real-world” evidence is
required
Decision process will be guided
by content expertise and
provision of the drug through
the Evidence Building Program
may be considered.
The drug is in the process of being
considered for an indication
represented by an arm of the trial.
Drug will not be funded, as a
formal funding decision
regarding that drug has not
been made.
Principle
Aligns with
principles of access
and support of
research
Aligns with
principles of access
and support of
research and is
consistent with
evidence-based
and fiscally
responsible
decision-making
Aligns with
principle that
coverage decisions
are evidence-based
and with policies of
the Ontario Public
Drug Programs.
Aligns with
principle that
coverage decisions
are evidencebased.
Aligns with
principles of access
and support of
research and is
consistent with
evidence-based
and fiscally
responsible
decision-making
Aligns with the
policies of the
Ontario Public Drug
Programs.
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Use of a publicly funded drug subsequent to patient participation in a clinical trial
Scenario
Management
Principle
The clinical trial drug has the same
Decision process is to be
Aligns with
pharmacologic class as the publicly
informed with content
principles of access
funded drug
expertise. Anticipated outcomes and support of
are that drug will not be
research and is
provided if cross-resistance is
consistent with
expected and a refractory state evidence-based
to the clinical trials agent exists. and fiscally
Drug may be provided if clinical responsible
trials drug is associated with
decision-making
beneficial effect and a period off
of treatment has passed (i.e.,
retreatment with same class of
agent).
The clinical trial drug has a different Decision process is to be
Aligns with
pharmacologic class as the publicly
informed with content
principles of access
funded drug
expertise. Due consideration for and support of
cross-resistance and expected
research and is
benefit will be applied.
consistent with
evidence-based
and fiscally
responsible
decision-making
Drug tested in clinical trial is
associated with funding criteria that
permit retreatment; circumstance
associated with subsequent use of
drug meets criteria for provision for
retreatment
*
Drug will be funded. No special
actions are required
Aligns with
principles of access
and support of
research
While control arm therapy would not require registration with CCO, the experimental arm may have
“line of therapy” or other implications on subsequent access to a publicly funded drug. Thus, trial
registration with CCO’s Provincial Drug Reimbursement Programs may thus be appropriate.
Requires Research Ethics Board approval and clinical trial registration (e.g., ClinicalTrials.gov); trial may
require Health Canada No Objections Letter associated with Clinical Trials Application
A negative funding recommendation does not exist for the combination.
**
***
i
CCRA Report Card on Clinical Trials. 2011.
http://www.cancer.gov/cancertopics/factsheet/Information/clinical-trials
iii
Roll Up: Public Funding and Clinical Trials (summer 2012 survey conducted by the Ministry’s Intergovernmental
Unit)
iv
Excel survey conducted July/August 2012 by CCO PDRP.
v
271. A preliminary literature review on policies on public funding of clinical trials v1.0
ii
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vi
http://www.kaiserpermanentejobs.org/our-business-structure.aspx
http://www.cms.gov/Medicare/Coverage/ClinicalTrialPolicies/downloads/finalnationalcoverage.pdf
viii
CCO correspondence with Finland – KELA.
ix
CCO correspondence with New Zealand – Pharmac.
vii
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