lecture 4 - drug discovery and development process Flashcards
describe the discovery phase and the development phase.
discovery phase - target idea for a disease
identify active compounds. continue to validate target
development phase
check safety and scale up, formulation
clinic rials and onto market. life cycle management
who are some of the stakeholders in healthcare and what are their needs ?
pharmaceutical industry,
- Is there a Market?
provider eg NHS and
- Is there sufficient benefit
to the patient vs our
ability to render care?
payers eg government and insurance companies
- Is the benefit worth the cost?
What is the long-term view?
patients - will this cure us ?
what is TPP?
target product profiles is a summary of a drug development program. It includes factors such as labelling concepts used within the regulatory body eg FDA.
indications and usage - specific disease and population of patients
dosage and administration - regimen, duration and route
dosage from and strengths
contraindications
warnings and precautions
adverse effects
what are other product aspirations that TPP can include?
Efficacy Profile – setting the bar for compound activity
Standard of Care (competitors)
Differentiation claims
Why this drug is better than existing therapy
Anticipated Launch Date
Territory
Pricing /Market Access
what are the characteristics of disease when assessing the disease burden ?
What is the prevalence of the disease?
What is the clinical presentation?
How is it diagnosed?
Is detection easy – are there accepted biomarkers?
How is it currently treated?
describe burden of COPD
short and long term outcome of disease if untreated are SOB, wheezing, chest tightness, increased risk of developing heart disease, lung cancer and a variety of other conditions
inhaled bronchodilators, corticosteroids, PDE4I, theophylline, antibiotics and antivirals
burden of ADKPD?
short and lan term outcomes; high bp, back or side pain, headache
earl detection and prevention of hon, renal comaplicationadn pain tx, other cardiovascular risk factors, management fo renal associations, rrt for esrd tolvaptan recently approved
compare the strategies for treating COPD and ADPKD.
COPD
symptomatic relief through bronchodilators which is a crowded market
Reduction of exacerbations with steroids and PDE4 inhibitors, noting that trials for these are very long and difficult, requiring a high number of statistics to show effectiveness.
Disease-modifying treatments aimed at longer-term outcomes, potentially leading to remission, but these involve long and costly trials with a lack of predictive models.
ADPKD
Symptomatic relief using anti-inflammatory medications and narcotics to control severe abdominal pain.
Slowing of progression with V2 antagonists like Tolvaptan, which also require lengthy and challenging trials with a high number for statistics to demonstrate their effect.
Disease-modifying treatments that are geared towards longer-term treatment and aim for remission, but are associated with long and costly trials
outline how to treat COPD patient according to the GOLD criteria
GOLD defines COPD severity based on the ratio of FEV1 (forced expiratory volume in one second) to FVC (forced vital capacity) after the administration of a bronchodilator. A ratio of less than 0.70 is used to diagnose COPD
GOLD 1 (Mild): FEV1 is greater than or equal to 80% of the predicted value. Patients might not notice their lung function is impaired.
GOLD 2 (Moderate): FEV1 is between 50% and 80% of the predicted value. Patients often seek medical attention at this stage due to chronic symptoms or flare-ups.
GOLD 3 (Severe): FEV1 is between 30% and 50% of the predicted value. Patients experience repeated exacerbations that negatively affect their quality of life.
GOLD 4 (Very Severe): FEV1 is less than 30% of the predicted value. At this advanced stage, patients’ quality of life is considerably impaired, and exacerbations can be life-threatening.
discusses the importance of risk mitigation in drug discovery through the lens of stratified medicine and translational science.
Right Disease: There needs to be a confirmed need for new treatments, which is assessed from various perspectives including regulatory, patient care, and the pharmaceutical supplier’s viewpoint.
Right Target: Drug targets should be chosen based on a thorough understanding of the disease’s clinical and biological aspects, as well as the overall burden of the disease.
Right Patient: It is crucial to identify the correct patient population for the new treatment. A patient population that is too broad may mask the drug’s efficacy due to variability among patients. The emphasis is on personalized healthcare, considering the genetic components and specificities of disease presentation in individuals.
