Appendix A to Item 11.03
Managed Access Programme framework following March 2015 PBAC.
Framework for the Managed Access
Programme for submissions to the PBAC
The Managed Access Programme (MAP) is a mechanism that enables the Pharmaceutical
Benefits Schedule (PBS) listing of products, under special circumstances of high unmet
clinical need, on terms that allow for the resolution of otherwise unacceptable clinical or
economic uncertainty for the Pharmaceutical Benefits Advisory Committee (PBAC). It seeks
to enhance the quality and strength of evidence provided to decision-makers in
reimbursement applications.
Listing is in conjunction with, and linked to, the subsequent provision of more convincing
evidence that is able to resolve specific existing areas of uncertainty as identified by the
PBAC. The PBAC provides advice in relation to the initial sources of uncertainty and whether
the evidence provided in the submission is sufficient to support initial listing under this
programme until a final review of the additional evidence is completed at a predefined point in
time.
In most cases where there are not sufficient high quality data to clarify the efficacy and safety
of a drug, randomisation in a Human Research Ethics Committee approved clinical trial is an
appropriate standard of care. Such trials are not subsidised via the PBS. The application of
the MAP mechanism is thus restricted to submissions where the PBAC agrees that, based on
the data available, the proposed drug would provide a significant net clinical benefit, but the
PBAC is uncertain about the magnitude of that benefit and therefore the price that should be
paid, and when:

there is a high and urgent unmet clinical need for the drug;

the PBAC would not otherwise recommend the listing of the drug at the proposed
price because the extent of the clinical effect is uncertain and/or there is a high cost
to the PBS overall and/or on a per patient basis; and

there is evidence that can reliably be reported and evaluated within a reasonable
timeframe, which the PBAC is satisfied would resolve the identified area of
uncertainty.
MAPs are complementary to risk sharing agreements (RSAs) which address financial or
budgetary uncertainty. MAPs, RSAs and special pricing arrangements (SPAs) are formalised
in deeds of agreement.
Managed Access Programme (MAP) parameters
A submission for a MAP should be considered as an exception and not the rule and should
meet the specified criteria below. A MAP may be initiated by a Sponsor as a proposal within
a major submission or by a Sponsor, the PBAC or the DoH during the assessment and
evaluation of a submission. MAP submissions may be appropriate where:

the PBAC considers or has agreed that there is a high and urgent unmet clinical
need for the proposed drug in the indication requested by the sponsor. The
consideration of unmet clinical need would include an assessment of the prevalence
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Appendix A to Item 11.03
Managed Access Programme framework following March 2015 PBAC.
and severity of the disease, whether alternative therapies are available, and the
extent to which the proposed drug is expected to meet the unmet clinical need;

the PBAC is satisfied that new evidence will become available within a reasonable
time frame that will resolve the issues of clinical and/or economic uncertainty that
would otherwise have prevented an initial positive recommendation. The time frame
should as far as possible be consistent with the standard term of Deeds of
Agreement and any additional follow up beyond the standard term should be agreed
at the time of initial listing;

implementation is via a deed of agreement, as an administrative tool to ensure clear
understanding by all parties of the obligations under the MAP framework and the
consequences of potential outcomes from the additional evidence, and to ensure that
the proposal, if adopted, is being used in conjunction with other existing tools
designed to manage the entry of a new drug, as appropriate; and

any subsequent review by the PBAC of the evidence specified by the deed of
agreement would also include a consideration of all other relevant evidence at that
time.
Sponsor
The major submission where the sponsor initiates consideration of a MAP would need to
include the following:

justification for the claim of high and urgent unmet clinical need;

all evidence available at the time of the initial PBAC consideration, including
evidence and rationale to support the validity of any surrogate outcomes used as the
basis of the PBAC assessment where relevant;

the areas of uncertainty (clinical and economic) as identified by the sponsor;

extensive sensitivity analyses to illustrate the potential impact of the identified
uncertainties on the initial incremental cost-effectiveness ratio (ICER);

details of the proposed additional evidence, with a discussion by the sponsor of how
such evidence would resolve the areas of uncertainty the sponsor or the PBAC had
identified, including those in relation to the extent of the clinical or economic effect (or
other circumstances in the future) which would be addressed in a subsequent
submission to the PBAC; and

anticipated timeframe for submission of the additional evidence.
Where a MAP is suggested by the PBAC or is proposed for consideration during the
evaluation of a product, the Sponsor must at a minimum provide:

details of the proposed additional evidence and a discussion by the sponsor of how
such evidence would resolve the identified areas of uncertainty, including those in
relation to the extent of the clinical or economic effect; and

the anticipated timeframe for submission of the additional evidence.
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Appendix A to Item 11.03
Managed Access Programme framework following March 2015 PBAC.
PBAC
As part of the MAP submission evaluation process, the PBAC would consider the usual
clinical and economic evidence available at the time of the initial consideration, and the
additional information relevant to the provision of further evidence in a subsequent
submission to the PBAC under the MAP framework.
The PBAC would determine if a MAP is appropriate and accordingly, may:

make a recommendation on listing and price based on the evidence available at the
time of its initial consideration;

identify its key areas of uncertainty for decision making (which may or may not be
identical to the uncertainties identified in the submission by the sponsor where
provided); and

identify the evidence it requires to resolve the areas of uncertainty, the timeframe for
submission of that evidence and the potential consequences of the evidence
outcomes.
Price
While a future price cannot be specified or guaranteed at the time of the initial listing, the
PBAC would consider the additional evidence in a subsequent submission and a price
change taking into account the extent to which the uncertainties are resolved by the additional
evidence.
A deed of agreement would be established in all cases to record the possible consequences
of the additional evidence on price.
Disclosure of information
At a minimum, the following information would be disclosed in the Public Summary
Documents (PSDs):

the identification of the proposed listing as a listing under the MAP and the
consequences this may have for patients and any future competitors;

the uncertainties identified at the time of the initial consideration by the PBAC;

the PBAC-agreed description of the evidence required to address the area of
uncertainty; and

the anticipated timeframe for provision of the additional evidence to the PBAC.
Further, a future PSD would be required, arising from PBAC deliberations relating to the
additional evidence submitted according to the deed of agreement, and any other available
evidence considered at that time by the PBAC.
Patients who may be prescribed a drug subsidised under an MAP must, through their
relevant prescribers, receive appropriate disclosure as to the circumstances of the drug
being listed on the PBS. At a minimum, they must be informed why a drug is listed through
a MAP and of the possibility that the drug may be delisted at the end of the MAP in which
case they could elect to pay for the drug privately to continue treatment. If clinically
appropriate, or recommended by the PBAC, patients and their prescribers must receive full
disclosure (consistent with the informed consent requirements of a clinical trial) of the risks
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Managed Access Programme framework following March 2015 PBAC.
involved in taking the drug, including the risk that the drug may not be as effective as
anticipated, as well as the risk of unanticipated adverse events. Arrangements should be
made to ensure a record of written acknowledgement that this information has been
conveyed to and understood by each patient prescribed a drug via a MAP. If the MAP also
requires that data be collected about PBS-subsidised patients, then the information to be
collected must also be appropriately disclosed and written consent obtained from the
patient.
Not for disclosure
If the company requests, the effective price of the drug would not be disclosed.
Cost recovery
The PBAC and its subcommittees would need to evaluate the new evidence provided in the
further submission, and in this context, the subsequent submission would be treated as a
major submission and an appropriate cost recovery process undertaken.
Deed of agreement
As part of the MAP process, there would be a deed of agreement which would specify items
including:

the agreed initial price;

the areas of clinical and/or economic uncertainty as identified and specified by the
PBAC that would need to be resolved by provision of additional evidence;

the timeframe for submission of additional evidence and a statement of intent by the
PBAC to consider the evidence within the accepted time frame;

acknowledgment that there may be other areas of uncertainty yet to be identified
which could impact on the initial ICER;

agreement on how the additional evidence would be presented in updated clinical
and/or economic evaluations, and how these evaluations could influence subsequent
listing recommendations by the PBAC affecting restrictions, pricing and/or rebates;

the expected consequences for pricing following the submission of the additional
evidence via the MAP which acknowledges the need for a ‘confidence discount’
reflecting the reduced confidence in the initial evidence submitted to the PBAC, such
as:
o
if the PBAC has recommended listing at a higher price than the PBAC
considered was justified by the initial existing evidence, a mechanism needs to
be available for payment of a retrospective rebate with interest to the
Commonwealth should the submission of additional evidence show that the
drug is failing to deliver on its claimed benefits;
o
if the PBAC has recommended listing at a lower price, that the PBAC
considers is justified given the existing evidence, a mechanism needs to be
available for recommendation of a higher price to be paid subsequently should
the submission of additional evidence confirm that the drug is delivering on its
claimed benefits;
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Appendix A to Item 11.03
Managed Access Programme framework following March 2015 PBAC.

other consequences of potential outcomes from the additional evidence, which may
include amending or introducing restrictions on use of the drug or, if deemed
necessary, full disinvestment via de-listing;

specification of the rights and obligations for the Sponsor of the original drug in the
event that an subsequent competitor drug is recommended by the PBAC for listing
before the MAP is completed and where the subsequently recommended drug may
materially alter the basis of the MAP (for example, the subsequently recommended
drug has a lower price, is supported by more convincing evidence, or might be used
alongside or in sequence with the original drug, thus confounding any data collected
in the context of the original PBS listing for the MAP);

commitment by the sponsor and the Commonwealth to disclose any confidential
information to the Sponsor of the subsequently recommended drug that would or
could have a material impact on the extent or value of the clinical or economic effect
of the original drug listed under the MAP or its competitor.
Competitors
MAPs are not intended to discourage competition.
A competing drug subsequently submitted to PBAC that might replace a drug already listed
under a MAP could have the potential to alter the extent of future clinical need, however not
the QALY gained of the original drug over its original comparator. However, data being
collected to verify key aspects of the estimated QALY gained for the original drug under its
MAP might be confounded by the listing of the competing drug.
The pricing of the competing drug is a matter for the PBAC based on its usual criteria,
including, but not limited to, the comparability of the two products given the reduced degree of
confidence the PBAC had in the incremental QALY gain modelled for the original drug at the
time of its recommendation.
If the competing drug is recommended by the PBAC, then, under applicable criteria outlined
in the deed, the original sponsor would share the relevant terms of the deed with the
subsequent sponsor and the subsequent sponsor would be informed of these terms, including
on a confidential basis as necessary.
Summary
In summary, a submission that would not normally be recommended for listing by the PBAC
because of unacceptable clinical and economic uncertainty could be recommended under a
MAP provided the MAP parameters are met. This would mean:

earlier access to the drugs by patients;

earlier access to a subsidised market for the sponsor whilst acknowledging that some
form of confidence discount may be required in recognition that the initial evidence is
less convincing;

clear articulation of the evidence required to resolve the identified area of uncertainty
and the consequences of potential outcomes from the additional evidence;
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Appendix A to Item 11.03
Managed Access Programme framework following March 2015 PBAC.

agreement by the PBAC to review a submission once the additional evidence
becomes available and to reconsider the listing in light of the new evidence;

appropriate sharing of risk.
It is envisioned that relevant stand-alone process guidelines applicable to the MAP and/or an
update to existing guidelines may follow on from this framework.
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