In Chapter 2, we explored the lengthy and complex process of bringing a new drug to market. We described the high failure rate and significant investment required throughout various stages of drug development.
Key points:
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The entire drug development process for a successful drug typically takes about 10 years, with many hurdles and unforeseen challenges. The process starts with identifying a promising molecule or target through laboratory research (discovery). Preclinical studies assess the compound’s safety and efficacy in cells and animals. After submitting an Investigational New Drug Application (IND) application to the FDA, clinical trials are conducted in three phases:
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Phase I: Test safety and dosage in a small group of healthy volunteers (sometimes patients).
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Phase II: Evaluate effectiveness and safety in a larger group with the target disease.
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Phase III: Confirm efficacy, monitor side effects, and compare it to existing treatments in hundreds to thousands of patients.
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If successful, a New Drug Application (NDA) is submitted to the FDA for a thorough review process. Once approved, post-marketing surveillance monitors the drug’s safety and effectiveness in the real world. This whole process can be modified and shortened for orphan diseases and some cancers, for example starting Phase 1 trials in patients (often called Phase 1b studies) and potentially allowing for accelerated approval based on a Phase 2b study, with full approval granted after post-marketing studies are completed (if they support approval).
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We highlight the benefits of this rigorous system to those us of that live in the U.S., which include:
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Access to safe and effective new drugs.
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Confidence in drug safety due to rigorous testing.
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Removal of ineffective drugs from the market.
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Reduced healthcare costs due to preventative measures.
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Improved life expectancy and overall health.
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We also describe the positive global impact of the U.S. Biopharmaceutical industry:
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Leading research and development (R&D) efforts.
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Increasing global access to medicines and vaccines.
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Fostering technological innovation and knowledge sharing.
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Contributing to economic growth and job creation worldwide.
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Drug development has a low probability of success, takes a long time, and is very expensive! In the next chapter we’ll dive into the financial aspects of this process in more detail.