W O W!!!!
Thanks Boffin, these were very interesting links!- After watching the conference video in your first link in Breakthrough Desigantion Criteria, I went down the rabbit hole, and the information I gleaned resonated with a lot of the drug efficacy comments that
@mason had emphasised in his presentation. At the risk of repeating, I am posting some of the key points I found useful and uplifting- maybe others will too.
What is It?
Created through the US FDA Safety and Innovation Act of 2012, Breakthrough Therapy Designation (BTD) was introduced to
shorten the development and review time of promising new drugs intended to treat serious or life-threatening diseases for which there is an unmet medical need
Breakthrough Therapy drugs will also spend a total of two to three years less in pre-market development compared with non-breakthrough therapy drugs. Furthermore, receiving this designation provides some credibility to the clinical promise of a given product and, as a result, can add significant value to a company.
The criteria for this designation is also very much in line with what we are hearing from Masons research, and indeed from the RAC teams results...
"Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
And lastly, Some key points on what the type of endpoint results are expected in order to gain this designation - again, some big ticks from the results we have with Bisantrene so far.
For purposes of Breakthrough Therapy designation, clinically significant endpoint generally refers to an endpoint that measures an effect on irreversible morbidity or mortality (IMM) or on symptoms that represent serious consequences of the disease.
A clinically significant endpoint can also refer to findings that suggest an effect on IMM or serious symptoms, including:
An effect on an established surrogate endpoint
An effect on a surrogate endpoint or intermediate clinical endpoint considered reasonably likely to predict a clinical benefit (i.e., the accelerated approval standard)
An effect on a pharmacodynamic biomarker(s) that does not meet criteria for an acceptable surrogate endpoint, but strongly suggests the potential for a clinically meaningful effect on the underlying disease
A significantly improved safety profile compared to available therapy (e.g., less dose-limiting toxicity for an oncology agent), with evidence of similar efficacy
A drug that receives Breakthrough Therapy designation is eligible for the following:All Fast Track designation featuresIntensive guidance on an efficient drug development program, beginning as early as Phase 1 Organizational commitment involving senior managers
Sorry for the copy and paste but its interesting stuff!
