NICE Flashcards
What is a conditional approval?
Orphan drugs or life threatening disease.
1) The risk benefit balance of the drug is positive
2) It’s likely that comprehensive data can be provided over time.
3) Unmet clinical needs will be fulfilled
4) Benefits of immediate availability outweigh the risks due to additional data to be provided.
When is PFS accepted as a clear evidence of benefit?
When there is also a trend to overall survival benefit.
What are the advantages of using PFS?
When further lines of therapy will modify OS
When it is more clinically relevant (not a surrogate for OS)
What are the problems associated with PFS?
Potential bias or problems with the subjective nature of determining progression.
Improvements in PFS may not correlate with an improvement in OS. (problems with using it as a surrogate)
How is potential bias in defining progression overcome>
Using a blinded independent central review for analysis of results.
What is a benefit of using PFS when cross-over or other post-progression treatments are implemented in a trial?
If a patient is receiving the experimental therapy after progression they may experience a survival benefit similar to the originally treated group. This negates the benefit seen in PFS by producing a similar OS.
Also, further lines of treatment after progression will have a similar dilutional effect on OS.
What do long post progression survival trends mean for the detection of OS benefit (especially if there has been cross-over/post-progression therapy)
It is harder to detect differences in OS and requires a much larger sample size.
What can be done to improve the phase II/phase III link
Phase II trials - Increase the sample size, randomise, use validated surrogates, better defined clinical controls
OR optimise phase 2-3
Need to identify correct target population. By transitioning into phase III, the phase II patients aren’t lost so it is easier to achieve the sample size required to form a highly powered study.
What are intermediate clinical endpoints?
Time to second progression/ time to the second subsequent treatment. If a PFS benefit is shown is carried over to the second progression.
What is NICE?
An independent organisation for providing national guidance on the promotion of good health and the prevention and treatment of ill health.
What does NICE look at when making a decision?
The cost effectiveness as well as the clinical benefit.
What is the cost effective assessment NICE implements?
They base cost effectiveness on the amount of quality life years gained (at £30,000 per quality of life year gained)
