New Drug Discovery and Development (Overview) - Part 1 | Dr. Shikha Parmar

Summary

This video offers an insightful overview of the complex process of new drug discovery and development. It highlights the multi-phase journey from screening thousands of compounds to identifying a lead drug candidate. The process includes target identification, lead generation, preclinical studies in animals, and clinical trials in humans, which can take up to 15 years and cost around $1.5 billion. Post-market surveillance ensures long-term drug safety even after FDA approval. The video emphasizes the importance of monitoring adverse drug reactions, particularly in larger populations after a drug is released.

Takeaways

• 💊 Drug discovery and development is a complex, lengthy process that takes 10-15 years and costs around $1.5 billion.
• 🧪 The drug discovery phase involves screening 5,000 to 10,000 compounds to identify potential leads or drug candidates.
• 🔍 Drug discovery has two main steps: identifying a target in the disease and validating it, followed by screening active compounds to find hits and leads.
• 📊 Hits are the initial 100-200 compounds with potential, and from these, dozens of leads are optimized for effectiveness and selectivity.
• 🧬 Lead optimization involves modifying chemical structures to improve selectivity, efficacy, and the ADME (absorption, distribution, metabolism, and excretion) profile.
• 🐁 In drug development, 250 optimized compounds are tested in animals during preclinical trials, with only around five moving forward for human testing.
• 👩‍🔬 Clinical trials are conducted in three phases (Phase 1, 2, 3), and they typically last six to seven years.
• ✅ If a drug passes clinical trials, a New Drug Application (NDA) is submitted to the FDA for approval, which takes one to two years.
• 🛡️ After approval, the drug undergoes post-marketing surveillance (Phase 4) to monitor its safety in the general population.
• ⚠️ Drugs may be withdrawn from the market during Phase 4 if serious adverse effects are discovered, as was the case with the drug Sibutramine.

Q & A

What are the two main stages in the process of new drug discovery and development?

The two main stages are the new drug discovery stage and the new drug development stage.

How many compounds are typically screened during the drug discovery process?

During the drug discovery process, around 5,000 to 10,000 potentially active compounds are screened.

What are 'leads' in the drug discovery process?

'Leads' are potential drug candidates identified during the drug discovery process after screening thousands of compounds.

What is the role of high-throughput screening (HTS) in drug discovery?

High-throughput screening (HTS) is used to screen thousands of compounds to identify around 100 to 200 hits that interact with a specific target in drug discovery.

What is lead optimization, and why is it important?

Lead optimization involves modifying lead compounds chemically to produce candidates with higher affinity, selectivity, and a good ADME (absorption, distribution, metabolism, and excretion) profile, which is crucial for developing effective drugs.

What happens during the pre-clinical studies in the drug development process?

During pre-clinical studies, around 250 lead compounds are tested in animals for safety, efficacy, and toxicity. By the end of this phase, typically only 5 compounds remain for further testing.

What are the three phases of clinical trials, and how long do they take?

The three phases of clinical trials are Phase 1, Phase 2, and Phase 3, and they take around 6 to 7 years to complete.

What is the role of the FDA in the drug development process?

The FDA reviews the Investigational New Drug (IND) and New Drug Application (NDA) submissions, monitors safety and efficacy data, and grants approval for a drug to be marketed if it meets safety and efficacy requirements.

What is post-marketing surveillance, and why is it important?

Post-marketing surveillance, also known as Phase 4 trials, involves monitoring the drug's safety once it is available to the public. It helps detect rare or long-term adverse effects that may not have been observed during clinical trials.

Why was the drug 'Sibutramine' withdrawn from the market?

Sibutramine was withdrawn from the market because it was found to increase the risk of heart disease and stroke during post-marketing surveillance.