drug approval process Flashcards
Milestone 1
Submission of a Pre Submission Planning forum
- Medicine type and data that will be provided to the authority: CMC, Pre-clinical and Clinical
- Used by the TGA to determine resource timelines and required to deliver resources to review the dossier
- Can be used to determine if there is sufficient quantity and quality of information for a dossier review
- Processed on the first day of every month
Milestone 3
Phase: first round assesment
Milestone: Internal review by TGA regulators/ Outcome of first round assessment is sent
- Informal requests of information
- Major issues go through the section 31 request letter
- 4 months for new medicine; 3 months for generic medicine
Milestone 2
Phase: submission
Milestone 2: Outcome of submission consideration sent
- Receipt of dossier- must be complete, outline of type of medicine or extension (repurposing)
- Delivered by the 7th of month; aprox 3 weeks following receiving of submission letter acceptance; reviewed and deemed effective or ineffective within 14 days
- Must abide by all regulatory requirements ( ICH guidelines etc
Milestone 4
Phase: Consolidated section 31 request response
Milestone 4: Applicant reviews the section 31 request letter
- Provides responses or clarifies factual errors
- Must be provided in 30 or 60 days (determined by the sponsor)
Milestone 5
Phase: Second round assessment
Milestone 5:
- Internal evaluators will review and consider the responses
- Completes final evaluation report
- If all ok recommended to registration
- If not okay, rejected, recommended with conditions (i.e. reporting) or recommended to expert advisory review
Milestone 6
Phase: Expert advisory review
Milestone 6: Expert advisory review
- Occurs after 2nd round of assessment phase
- Key advisory group is the advisory committee of medicines. Pharmaceutical subcommittee and pharmacometrics working group are sub groups of this
- These advisory committees can only be made to the TGA which can be adopted or ignored by the TGA. Accepted recommendation are then returned to the sponsor via a section 31 letter
Expert advisory groups
Advisory Committee on medicines (ACM): Final overview big clinical review of everything (main group)
Pharmaceutical subcommittee (PSC) (part of the ASM): requested to provide advice after first or second round of comments back from the sponsor. Formulation, bioequivalence issues etc.
Pharmaceutics working group (part of the PSC): evaluates all the PK/PD data the sponsor provides
- only make recommendations to the TGA. Recommendations are provided back to the sponsor via a section 31 request letter
Time difference between generic and NCE
- Generic medicine = 8.5 months NCE = 10.5 months
- Generic medicine does not require expert review as all tox, safety, SAD, MAD, Phase 1, Phase 2 and Phase 3 has already been completed.
- Only need to conduct BE, PK study (across different doses and formulations)
Priority reviews
Priority reviews are designated for drugs which treat chronic diseases which are highly life threatening and predicted to save a lot of lives.
Same: Clinical, preclinical CMC data
Differences:
- Accelerated reviews use a surrogate end-point (biomarker), which is an objective endpoint that the drug is working. For example shrinkage of a tumor.
- If missing a piece of safety data: black triangle on the PI
- Once given conditional approval: Onus on the sponsor, must continue to monitor the drug in the market via phase IV studies… Any. Evidence of lack of efficacy or severe toxicity, then reviewed by regulatory body.
- If lack of evidence that it works. Then can be removed from the market!!
Orphan drug designation
- Rare disease (5 in 10 000)
- Less than 250 000 patients
- Same regulatory processes
- Protection against competitors (extended patent period)
- needs to improve quality of life (cant be on a drip 20 hours a day)
- Clinical trials are hard (use lots of computer stimulation )
