Challenges 1 Flashcards
How are priorities chosen
Population - incidence & prevalence, demographics, sex, age
Disease burden - mortality/morbidity, impact on quality of life
Treatment options - size & type of benefit, adverse effects/risks, accessibility
Describe mortality
Top causes of global death - cardiovascular, respiratory & neonatal conditions
7/10 noncommunicable (chronic) - 74% of all deaths globally
Describe morbidity
Top global causes of DALYs neonatal, cardiovascular & neonatal infections
- due to communicable disease dropped 50%
- From diabetes increased 80%
- Alzheimer’s more than doubled
Describe rare or orphan diseases
Huge number of chronic, disabling, multi-system, complex diseases
Do not feature on global death, DALY totals or pipeline priority list
What is the pipeline
Discovery
Refine lead
Pre-clinical
Clinical
Drug
Why were the pipeline priorities chosen
- Target therapies (easier to treat)
- Marketable/severe (more likely to do trials)
- Know more about
- High risk to benefit ratio
- Long term treatment super safe
- Subjective measures
- Modelling chronic and neonatal disease difficult
Describe the current pipeline approach
One treatment fits all - but all unique and respond differently to both
What 3 groups do patients fall into
Responders
Non-responders
Adverse-responders
What are the problems of the current pipeline approach
Most drugs ineffective for more people than they help (chemical and biological)
lack efficacy relates to disease parameters or drug kinetics
Why are studies biased
Variation increases risks, complexity & costs simpler to exclude in research & drug trials
Look through differences between females and males
Differences in drug processing
Describe overcoming all bias
treatments tailored to patient-specific characteristic
Must be classified based on disease susceptibility and treatment responsiveness
Matching of patient genetics and biomarkers with their response to drugs
