L38 Clinical Trials LA Flashcards
Outline the 3 main drivers for change in clinical drug development
- Rising cost of research
- Late phase failures and market withdrawals
- Low return of investment
expound on how rising cost of research a driver for change in clinical drug development
- $2.5-3 Billion from discovery to marketing improvals
- Larger trials and increasing complexity.
- Need for tested drug vs comparators data
- High failure rate in human testing
- Increasingly stringent regulatory environment.
expound on how late phase failures and market withdrawals are a driver for change in clinical drug development
Poorly desinged studies -> should not have advanced for further testing.
Market withdrawals due to unexpected adverse events -> e.g Viox
expound on how low return of investment is a driver for change in clinical drug development
There is a lack of justification of high prices. So prices arent kept up
Need to prove value before price is paid -> pharmacoeconomics
Outline the Clinical Trial solutions
Translational Medicine
Biomarkets
Precision Medicine
Give a detailed overview of translational medicine
- Involves increasing the speed at which newly discovered medicine reaches patients.
The Process has three phases:
Phase 1: Acess the type of disease to be treated. Develop potential treatments for it.
Test the safety and efficacy of the developed therapy using RCT
Phase 2: Examine how treatments developed earlier fuction when applied in routine practice later.
Phase 3: Transforming the effective therapy into a cost-effective sustainable solution.
what are biomarkers
A Biomarker is a characteristic that is objectively measured and evaluated as an indicator of normal, biological processes or responses to a therapeutic intervention
How are biomarkers useful
1 They can help provide further information in regards to the efficacy of the drug.
2 They can also help indicate signs of possible toxicity.
3 They can be used to provide a more precise choice of dose selection in clinical trials to help reduce potential sub therapuetic doses.
Biomarkes will be used as a surrogate endpoint. (must state this) To subsitute the current ‘clinical endpoint’ currently used.
Precision medicine aims
Aims of precision medicine is to treat the disease at the molecular level. The target is clearly defined.
Right medicines -> right patient -> right disease
The use of pharmacogenomics and pharmacogenetics -> employed to give individual genetic profiles.
involves gradual transition away from “one size fits all”
Gradual change in disease classifications based on genomic differences.
More clinical trials will be condicted -> will have fewer subjects
e.g Herceptin for HER2 positive breast cancer.
Define pharmacogenomics (not genomics)
defined as the study of variations of DNA and RNA as related to drug response.
