Cancer Drug Reimbursement within the Context of Clinical Trials. (Draft for consultation purposes) Version 8.0

Transcription

1 Cancer Drug Reimbursement within the Context of Clinical Trials (Draft for consultation purposes) Version 8.0 May 17, 2013

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3 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. 3

4 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 4

5 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. Scope 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. 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. 5

6 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. 6

7 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. 7

8 Table 1: Use of a publicly funded drug within the conduct of a clinical trial Scenario Management Principle The drug will be funded. No special actions are required* Clinical trial use of drug is identical to the current funding policy (e.g., control arm therapy in a Phase III trial) Clinical trial use of drug is for same population as a drug that is funded; drug prescription may be altered (e.g., dose, schedule) or may be combined with another agent (e.g., experimental arm therapy in a Phase III trial) 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) Clinical trial involves re-treatment with a previously funded drug. The current funding criteria for that drug do not include retreatment. 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 The drug will be funded provided that the parameters for conduct of clinical trial must be satisfied**. Provision of drug cannot be associated with incremental cost to the publiclyfunded system.*** Drug will not be publicy funded Drug will not be publicly funded Decision process will be guided by content expertise and provision of the drug through the Evidence Building Program may be considered. research research and is consistent with evidence-based and fiscally responsible decision-making principle that coverage decisions are evidence-based and with policies of the Ontario Public Drug Programs. principle that coverage decisions are evidencebased. research and is consistent with evidence-based and fiscally responsible decision-making 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. the policies of the Ontario Public Drug Programs. 8

9 Use of a publicly funded drug subsequent to patient participation in a clinical trial Scenario Management Principle The clinical trial drug has the same pharmacologic class as the publicly funded drug The clinical trial drug has a different pharmacologic class as the publicly funded drug 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 Decision process is to be informed with content expertise. Anticipated outcomes are that drug will not be provided if cross-resistance is expected and a refractory state to the clinical trials agent exists. Drug may be provided if clinical trials drug is associated with beneficial effect and a period off of treatment has passed (i.e., retreatment with same class of agent). Decision process is to be informed with content expertise. Due consideration for cross-resistance and expected benefit will be applied. Drug will be funded. No special actions are required research and is consistent with evidence-based and fiscally responsible decision-making research and is consistent with evidence-based and fiscally responsible decision-making 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 ii 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 9

10 vi vii viii CCO correspondence with Finland KELA. ix CCO correspondence with New Zealand Pharmac. 10