Costly resub pathways? You asked for it

25 March 2021

The Department of Health explained the four new pathways designed to expedite PBAC resubmissions in an industry webinar on Wednesday, revealing that unless the reimbursement committee classifies a rejected submission as only needing minor changes to progress, then don’t expect it to appear again in under eight months – and quite likely 12.

Health officials could not say whether the outcome statement for the March 2021 PBAC meeting, to be published in April, would reveal which pathway the rejected submissions have been offered. Sponsors know and have already made the call as to whether their particular pathway offer was worth the asking price.

The changes, arising from Medicines Australia Strategic Agreement processes which commenced over three years ago, were supposedly what industry asked for. The PBAC will now direct rejected submissions to one of three new pathways, based on unresolved issues and whether the vaccine or therapy offers High Added Therapeutic Value (HATV).

The default is a Standard Re-entry Pathway, which comes with a somewhat eye-watering price of $166,650. This is where all rejects will end up if the sponsor doesn’t accept their pathway offer, wants to include new information, or wants to lodge later.

Either way, this route leads to an eight-month delay before the committee will reconsider the medicine for funding – and an even longer wait for patients.

The Golden Pathway

The Facilitated Resolution Pathway is the new gold class option – and comes with a price tag to match. At $238,660 (including a hefty $72,000 deposit), this path back to PBAC will be offered to medicines or vaccines deemed to address high unmet need or to offer a significant improvement over existing therapies, and where the PBAC believes the outstanding issues can be resolved through a workshop.

Naturally, the company will pay for the privilege of the workshop and, for that price, will have the pleasure of the PBAC chair and deputy chair at their chat fest, along with potentially other influential players such as the chair of the Economic Sub-Committee and/or the Drug Utilisation Sub-Committee.

But don’t expect to get a fast turnaround for the big bucks invested on The Golden Pathway. The best outcome for this track is appearing on the PBAC agenda eight months later and, if you miss that one, the following year.

Of course, the promise is that, given all issues are resolved, a Golden Pathway resubmission will get a green light the second time around. Given it currently takes on average 2.5 submissions to get a PBAC recommendation, the conclusion is that sponsors will in fact save time using this option.

For a faster turnaround, rejections have to be offered an Early Re-Entry or Early Resolution Pathway, both priced at just under $42,000. These may get sponsors up at the following PBAC major meeting, if they move quickly enough, or the one after that if they don’t but don’t hold your breath for this option. And could these issues not be better resolved through deferral?

Is this it?

The resubmission pathways is the final piece in the PBS Process Improvements jigsaw to fall into place.

A company can now hope that a therapy deemed to meet high unmet need or to offer a significant improvement over existing therapies has a best-case scenario of getting through the PBAC first time at a cost of $365,000.

As this situation is rare, then the more realistic situation is an offer of a facilitated pathway, boosting the chances of getting recommended after two PBAC appearances at a cost of $600,000. If it is a complex vaccine, then that cost is potentially $800,000.

Merck Healthcare’s Leah Goodman recently told the Zimmerman parliamentary inquiry the cost of getting a positive recommendation often cost $1 million, plus substantial consulting and inhouse fees.

She also told the inquiry she didn’t know of other countries which charged to assess medicines for funding.

So will it work?

Let’s take a look at four rejects from the November 2020 meeting which may have met requirements for fast-tracking.

The Medical Oncologists Group of Australia (MOGA) categorised four cancer submissions as being therapies of “highest priority for PBS listing” due to them meeting high and unmet needs or a significant improvement over existing care.

Janssen’s ERYLAND (apalutamide) was already onto its third submission, so arguably had the new pathways been up and running earlier could have saved the cost and effort of three rejections over two years and still being no closer to funding.

The prostate cancer drug is for patients who don’t have another option but the outcome? Rejected. Major resubmission required.

Sanofi’s LIBTAYO (cemiplimab) appeared for the first-time appearance in cutaneous squamous cell carcinoma – patients with a high unmet need. The outcome again was rejected. Major resubmission required.

Another first-timer, Bayer’s pan-tumour VITRAKVI (larotrectinib), was looking at NTRK fusion solid tumour – patients who have no treatment options other than “disfiguring surgery or limb amputation”. Despite TGA provisional approval indicating the importance of the therapy, the PBAC marked it ‘Rejected. Major resubmission required’.

MSD’s KEYTRUDA (pembrolizumab) was looking for expansion in head and neck squamous cell carcinoma patients – a currently uncurable cancer that impacts almost 4,500 Australians with a five-year survival rate of five per cent. The PBAC noted the high need and labelled the therapy as “clinically superior” to current therapy but the outcome? Rejected. Major resubmission required.

All four cancer drugs ticked the box for addressing high unmet need.

All four offer patients with few options the only chance of some treatment, making them qualifiers for the Facilitated Resolution Pathway. Yet even had this option been available, these patients would not have had access to funded therapy until later this year, and most likely next year.

Patients with a one in 20 chance of being alive in five years.

If the PBS process improvements that supposedly deliver what industry asked for still result in years of delayed access to promising new therapies which medical specialists say are of urgent need, then they are still not fast enough.

Industry asked for it but did the department deliver? I think not.