Note
TREATMENT OF PAEDIATRIC PATIENTS WITH REFRACTORY CROHN DISEASE
The following information applies to the prescribing under the Pharmaceutical Benefits
Scheme (PBS) for paediatric patients with adalimumab for severe refractory Crohn disease
and infliximab for moderate to severe refractory Crohn disease. Where the term "biological
medicines" appears in the following notes and restrictions, it refers to adalimumab
and infliximab only.
A patient is eligible for PBS-subsidised treatment with only one PBS-subsidised biological
medicine at any one time.
From 1 August 2015, under the PBS, patients will be able to commence a treatment cycle
where they may trial a PBS-subsidised biological medicine without having to experience
a disease flare when swapping to the alternate agent. Under these arrangements, within
a single treatment cycle, a patient may continue to receive long-term treatment with
a biological medicine while they continue to show a response to therapy.
A patient who received PBS-subsidised biological medicine treatment prior to 1 August
2015 is considered to have started their treatment cycle as of 1 August 2015.
Within the same treatment cycle, a paediatric patient cannot trial and fail, or cease
to respond to, the same PBS-subsidised biological medicine more than twice.
Once a patient has either failed, or ceased to respond to treatment for this condition
3 times, they are deemed to have completed a treatment cycle and they must have, at
a minimum, a 5-year break in PBS-subsidised biological medicine therapy for this condition
before they are eligible to commence the next cycle. The 5-year break is measured
from the date of the last approval for PBS-subsidised biological medicine treatment
in the most recent cycle to the date of the first application for initial treatment
with a biological medicine under the new treatment cycle.
A patient who has failed fewer than 3 trials of biological medicine therapy in a treatment
cycle and who has a break in therapy of less than 5 years may commence a further course
of treatment within the same treatment cycle.
A patient who has failed 3 trials or fewer of biological medicine therapy in a treatment
cycle and who has a break in therapy of more than 5 years may commence a new treatment
cycle.
There is no limit to the number of treatment cycles a patient may undertake in their
lifetime.
How to prescribe PBS-subsidised biological medicine therapy after 1 August 2015.
(1) Initial treatment.
Applications for initial treatment should be made where:
(i) a patient has received no prior PBS-subsidised biological medicine therapy in
this treatment cycle and wishes to commence such therapy - Initial 1 (new patient);
or
(ii) a patient has received prior PBS-subsidised (initial or continuing) biological
medicine therapy and wishes to trial an alternate agent - Initial 2 (Change or Re-commencement
of treatment after a break in therapy of less than 5 years ) [further details are
under 'Swapping therapy' below]; or
(iii) a patient wishes to re-commence treatment with a specific biological medicine
following a break in PBS-subsidised therapy with that agent - Initial 2 (Change or
Re-commencement of treatment after a break in therapy of less than 5 years ); or
(iv) a patient wishes to recommence treatment with a biological medicine following
a break in PBS-subsidised therapy of more than 5 years - Initial 3 (Recommencement
of treatment after a break in biological medicine of more than 5 years).
Initial treatment authorisations will be limited to provide for a maximum of 16 weeks
of therapy for adalimumab and 14 weeks of therapy for infliximab.
From 1 August 2015, a patient must be assessed for response to any course of initial
PBS-subsidised biological therapy following a minimum of 12 weeks of therapy for adalimumab
and up to 12 weeks after the first dose (6 weeks following the third dose) for infliximab,
and this assessment must be conducted no later than 4 weeks from the date that course
was ceased.
Where a response assessment is not conducted within these timeframes, the patient
will be deemed to have failed to respond to treatment with that biological medicine
unless the patient has experienced a serious adverse reaction of a severity resulting
in the necessity for permanent withdrawal of treatment.
(2) Continuing treatment.
Following the completion of an initial treatment course with a specific biological
medicine, a patient remains eligible to receive up to 24 weeks per course of continuing
treatment under the First continuing treatment and Subsequent continuing treatment
restrictions with that drug providing they continue to sustain the response.
It is recommended that a patient is reviewed for response following a minimum of 12
weeks of therapy and no later than 4 weeks from the completion of the most recent
course of treatment.
(3) Swapping therapy.
Once initial treatment with the first PBS-subsidised biological medicine therapy is
approved, a patient with severe disease may swap if eligible to the alternate biological
medicine within the same treatment cycle without having to requalify with respect
to the indices of disease severity (i.e. Paediatric Crohn Disease Activity Index (PCDAI)
Score, confirmation of Crohn disease), or the prior conventional therapies of corticosteroid
therapy, immunosuppressive therapy or enteral nutrition.
A patient cannot swap to a biological medicine if they have failed to respond to prior
treatment with that drug twice within the same treatment cycle.
To ensure a patient receives the maximum treatment opportunities allowed under these
arrangements, it is important that they are assessed for response to every course
of treatment within the timeframes specified in the relevant restriction.
A patient who is not able to complete an initial treatment course for a biological
medicine will be deemed to have failed treatment with that biological medicine unless
the patient has experienced a serious adverse reaction of a severity resulting in
the necessity for permanent withdrawal of treatment.
(4) Baseline measurements to determine response.
A response to treatment is to be determined by comparison of current disease activity
measurements relative to the baseline measurements submitted with the first authority
application for a biological medicine. However, prescribers may provide new baseline
measurements any time that an initial treatment authority application is submitted
within a treatment cycle and eligibility for continuing treatment must be assessed
according to these revised baseline measurements.
(5) Recommencement of treatment after a 5-year break in PBS-subsidised therapy.
A patient who wishes to recommence treatment following a break in PBS-subsidised biological
medicine therapy of at least 5 years, must requalify for initial treatment with respect
to the indices of disease severity under the Initial 3 restriction. A retrial of conventional
therapies is not required.