Anavex Life Sciences Corp (AVXL)

[sokol]

Dranesthesia:

The author of this article, Paul Kubler, does not agree with your statement that "Until they have a large appropriately powered study that confirms their thesis than they have nothing." https://www.nps.org.au/australian-prescriber/articles/fast-tracking-of-new-drugs-getting-the-balance-right

Paul Kubler that wrote the article has received sponsorship from Bristol-Myers Squibb to attend the 2017 EULAR Annual European Congress of Rheumatology and has acted as a consultant to Abbvie, Eli Lilly and Reckitt Benckhiser.

Read the article, but I summarize the article below:

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.