Understanding the Drug Development Pipeline

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Introduction

The drug development pipeline (DDP) is, at its core, the fundamental process that biopharma companies undergo in the quest to bring their therapeutic to market. It is composed of multiple stages that can be lumped into two groups: research and development. The research stages include everything from discovery of a potential product to phase 2 clinical trials, whereas the development stages range from phase 1 clinical trials to post-clinical studies relating side effects of the therapeutic that were not highlighted in clinical testing. The overlap between the two groups of stages highlights the balance required for companies in obtaining funding, and the promise of eventual commercialisation that must be shown to investors to support these endeavours.

Explaining the stages within the DDP

• Discovery
  • “Pure” research that aims to find an innovative compound/family of compounds that has potential to impact disease. This stage is mainly undertaken by scientists at research institutions.

• Target validation
  • Locating a disease, molecule or biological pathway that your newly discovered drug can have an effect on. The components that cause illness through your target must be understood to highlight how your drug may have an impact.

• Lead identification
  • Identification of the drug from your family of compounds that is most likely to see success through the later stages in the DDP, which is achieved through testing on cells and tissues associated with the target you have already identified, as well as computational and machine-based models.

• Lead optimisation
  • Gathering the necessary resources to make commitment to developing your prospective therapeutic as streamlined as possible. Achieving a clear pipeline is vital in attracting investors to fund drug development.

• Preclinical studies
  • These trials are conducted on animal models to test the safety and verify the indication (prove that it does what it is designed to do) of the therapeutic. The most common animal model is mice due to their small size and the fact that they are mammals, like humans.

• Phase 1 clinical trials
  • After you have proven low toxicity in your animal models and gathered preliminary information on your drug’s interactions with the body (a.k.a. pharmacokinetics), you’re ready to enter the clinical trial stage of the DDP. Phase 1 clinical trials deal with a small group of (often imbursed) participants to further test the drug’s safety and determine the best dose to give to patients.

• Phase 2 clinical trials
  • Once the phase 1 clinical trials are completed, trials that are designed to determine the efficacy and side effects of the drug must be completed. These are the phase 2 clinical trials. The participants of these clinical trials suffer from the disease that your drug is trying to target. These trials weigh up the risks and benefits of using the new therapeutic before being able to traverse to the next and final clinical trials.

• Phase 3 clinical trials
  • These clinical trials are the final ones that must be completed before regulatory approval is granted. They test for effectiveness and safety on a global scale, often in comparison to what is already on the market for the target disease. In the USA, at least three phase 3 clinical trials need to be completed before being approved for commercial use. These trials are by far the most expensive to conduct, so lots of external funding is required.

• Post-clinical studies
  • Congrats! You have successfully beaten the VERY slim odds and reached regulatory approval for your drug. Although you may now be raking in the big bucks, your work is not done yet. Those using the drug for its intended purpose in the general populace must be monitored for side effects that were not picked up during the clinical trials.

Key considerations

It is vital that, at every stage of the DDP, adequate funding is acquired, which is achieved through a number of different ways. One source of funding to consider is that of venture capital firms, who provide an investment into the company on the basis that they will be able to make a profit. It is a risky proposition for any investor, so the company involved with creation of the new drug must provide a successful, detailed pitch to the investor to convince them that their product is profitable.

Intellectual property is an important aspect in the road to commercialisation. It is what protects your product from other companies being able to replicate your hard work without spending nearly as much money. The way protection over intellectual property is through application of patents, which, although expensive to obtain, protect your product for ~20 years.

It is incredibly difficult to get a product off the lab bench into pharmacies, costing lots of time and lots more money. Patience, scientific knowhow and business acumen must be employed in equal measures to have even a sliver of a chance to create a product that is safe and effective.