Provisional Approval and Priority Review are two separate pathways for expedited approval. These are my thoughts after reading the consultation “Provisional Approval pathway for prescription medicines – Proposed registration process and post-market requirements”. The consultation paper can be found at:

It is in the best interest of pharmaceutical companies to bring their new products on the market sooner to get a fair chunk of market share and financial benefits. The consultation paper clearly states the kind of medicine that the provisional pathway will be available to – those with promising evidence that earlier availability has the potential to provide a significant benefit to patients with inadequate treatment options for serious and life-threatening conditions. It is not intended to provide a second-tier regulatory pathway for any medicine with insufficient evidence. The clarity provided in the consultation paper is satisfactory. But what about those medicines that have a significant public health benefit?

The paper states that it is available only to certain medicines that meet the transparent eligibility criteria – how does one establish this?

I think it is a good idea to have a patient registry. Since by definition, the medicines will be used for serious and life-threatening conditions, the registry will be helpful in the surveillance of the effects of the medicine on patients.

I am not too sure about the timeframe stated for a pre-designation meeting of ~6-7 months. Would this mean that sponsors will have to go through this meeting before they start clinical trials etc? How feasible is this timeframe for serious and life-threatening conditions? Will sponsors be paying any kind of fee during this meeting process and what it may involve? It may be a double-edged sword – if pre-designation meeting/some kind of submission involved in this type of meeting is free – regulators may need to bear more burden and if not, industry trying to bring in critical medicines to the market loses. While this may not have a huge impact on well-established pharmaceutical companies, it will negatively impact small/start-up companies trying to bring in the next ‘block-buster’ drug to the market.

I agree to the automatic lapsing of provisional registration (provisionally and then fully) and withdrawal from the market not being subject to appeal as long as sponsors are given opportunities to extend the registration, they get sufficient time and are covered by provisions to convert it to full registration.

As mentioned in the paper, medicines may simultaneously qualify for orphan drug designation and provisional approval. In this case, there will not be much financial burden on industry of paying two separate fees for provisional registration and then a separate full registration (e.g. current CAT 1). However, if they don’t qualify for a waiver of application and evaluation fees, this can get expensive. Will TGA consider reduced fees for full registration of medicines that were previously provisionally registered?

After being provisionally approved,

–                Data can be made available within 2 years. Any possibility of using the same initial application with additional data for full registration? This could save resources on both the regulator’s as well as industry’s end.

–                How will extension of indication of provisionally registered medicines be handled?

Rolling submission of clinical or other relevant data during assessment period – may be done on a case-by-case basis – it is a good provision but should be specified a bit better – in what cases are sponsors likely to get this benefit? Otherwise it may place undue burden on the evaluator to make a decision whether or not they’d allow submission of additional data. If this is not clear, it may negate the whole process of provisional approval by making it too time-consuming. Isn’t that why TGA has moved away from this approach in the past?

I agree with TGA providing standard wording to describe what provisional registration means for CMIs and PIs.

A mix of fully and provisionally registered indications should, in my opinion, be considered under the umbrella of provisionally registered medicine and a more rigorous process that is applicable to provisionally registered medicine should be applied to the combination.

How will TGA prioritise the approval of provisional approval? Will it also be given priority approval?