Anavex Life Sciences Corp (AVXL)

[sokol]

AVXL 2-73 is unquestionably a candidate for fast track provisional approval for one or all of the CNS indications involved in its existing multiple clinal trials.

Those that argue against AVXL 2-73 obtaining a fast or provisional approval in Australia are using traditional drug regulatory processes for approval of a drug in support of their arguments. Those arguments do not fit here, and are clearly outdated. In March 2018, Australia adopted a new provisional pathway as discussed below, and it definitely encompasses faster non-traditional approval for drugs for serious diseases with an unmet need, including dementia.

The eligibility criteria are (a) a new prescription medicine or new indications medicine, (b) treating a serious condition with a favorable comparison against existing therapeutic goods, (d) a major therapeutic advance and (f) evidence of a plan to submit comprehensive clinical data. https://www.tga.gov.au/provisional-approval-pathway-prescription-medicines#what

Provisional approval in Australia recognizes that:

I. Patients and their doctors have a growing desire to access new drugs as soon as possible.
2. A traditional drug regulatory framework may no longer be the most appropriate assessment process for dealing with quickly evolving scientific advances.
3. In the last 20 years, several regulatory bodies have tried to develop and test fast-track approval processes for drugs to treat severe diseases for which the options are limited.
4. That, as advocates maintain, targeted treatments such as modern immunotherapies do not fit current regulatory processes.
5. Novel immuno-oncology drugs are clinically tested in trials with small patient numbers and often in the setting of knowing the patient’s genetic profile.
6. These attributes may allow for better prediction of response with fewer significant adverse events.
7. Advances in digital technology, remote monitoring, patient sensors and data analytics are allowing for improved recording of reliable and validated patient-related outcomes in studies with smaller sample sizes.
8. Making a drug available early with temporary authorisation is not a new concept, particularly for patients with life-threatening or seriously disabling conditions for which there is a clear unmet therapeutic need.

''In Australia, like the rest of the world, patients and their doctors have a growing desire to access new drugs as soon as possible. They hope to make an impact on conditions with limited pharmacotherapeutic options, such as cystic fibrosis and rare cancers like mesothelioma. New approaches to more common diseases, such as lung cancer and dementia, may offer greater efficacy or less toxicity than current therapies. The pharmaceutical industry is also hungry for expedited drug approvals as a vehicle to reward and encourage innovation. Faster approvals may increase company profits as products get to the market more rapidly....

...a traditional drug regulatory framework may no longer be the most appropriate assessment process for dealing with quickly evolving scientific advances.

The traditional approach in the assessment of a new drug involves a sequence of clinical trials (phase I–III). Accumulated evidence of dose justification, efficacy and safety in specified treatment indications and target populations then enables the drug’s sponsor to apply for registration of the drug. However, in the last 20 years, several regulatory bodies have tried to develop and test fast-track approval processes for drugs to treat severe diseases for which the options are limited.

Following a review1 the TGA consulted about expedited approvals and has introduced a priority review pathway. This aims to assess new drugs within 150 days....

In these programs drugs for serious illnesses are rapidly approved on the basis of limited clinical trial data or data reliant on surrogate outcome measures, some of which are biochemical, for example glycated haemoglobin (HbA1c), rather than clinical. Anticancer drugs may be approved on response rates, often measured over relatively short time frames, rather than on improved survival.....

Advocates of rapid access to new therapies claim that targeted treatments such as modern immunotherapies do not fit current regulatory processes. With an enhanced contemporary understanding of disease pathogenesis pre-study, novel immuno-oncology drugs are clinically tested in trials with small patient numbers and often in the setting of knowing the patient’s genetic profile. It is claimed that these attributes allow for better prediction of response with fewer significant adverse events. Furthermore, advances in digital technology, remote monitoring, patient sensors and data analytics are allowing for improved recording of reliable and validated patient-related outcomes in studies with smaller sample sizes.

In March 2018, the TGA announced a provisional approval pathway. This will allow drugs to be available for up to six years based on preliminary data.9 The anticancer drug olaratumab is the first drug to be considered for provisional approval in Australia.

Access to new therapies is a balance between evidence (determining the risk of acceptable adverse effects versus efficacy) and the speed of availability, intersected by the issue of affordability. Making a drug available early with temporary authorisation is not a new concept, particularly for patients with life-threatening or seriously disabling conditions for which there is a clear unmet therapeutic need. Temporary access is akin to a learner driver receiving their provisional licence – a full licence is only granted after more experience. Rapidly approved drugs should receive provisional registration for a period of three years and the drug company should be required to provide annual data on the postmarketing experience.''

https://www.nps.org.au/australian-prescriber/articles/fast-tracking-of-new-drugs-getting-the-balance-right