The drug discovery pipeline is the process by which new drugs are discovered, developed,
and brought to market. It typically involves several stages, including:
● Disease hypothesis and early target identification: A drug discovery pipeline begins
with the identification of a disease hypothesis and early target, usually performed in
academia with public funding over the course of decades.
● Target identification and validation: In this stage, researchers identify the most
promising biological target that is thought to be involved in a disease or condition,
and then validate that target by demonstrating its involvement in the disease or
condition. The timeline for this stage depends on the involvement or not involvement
of academia and might be up to 2.5 years with an average cost of $353M;
● Lead discovery and optimization: In this stage, researchers search for compounds
that can bind to the validated target and have the desired therapeutic effect. These
compounds are then optimized to improve their potency, selectivity, and other
properties. It usually takes up to 2 years and cost approximately more than half of
billion dollars;
● Preclinical development: In this stage, the optimized compounds are tested in
laboratory and animal models to determine their safety and efficacy. There is an
average time of 1 year for it to be completed and it costs approximately $340M;
● Clinical development: In this stage, the compounds are tested in clinical trials to
determine their safety and efficacy in humans. Timelines and costs vary between
phases, but in general, phase I takes 1.5 years and phases II and III take 2.5 years,
with approximately half a billion dollars spent in total. In Phase I, the drug's safety
and dosage are assessed. In Phase II, drugs are evaluated for their effectiveness
and adverse effects. In Phase III trials, new treatments are compared with standard
protocols to determine whether they are more effective;
● Regulatory approval: In this stage, the compounds are submitted to regulatory
authorities for approval to market the drug. In general, it takes 1.5 years to achieve
FDA approval, which is the cheapest phase for the transition and costs approximately
$3M;
● Commercialization: In this stage, the drug is brought to market and made available to
patients.
Drug discovery is a very expensive and long process. Each step of the drug
discovery program is important. However, failures in Phase 2 and Phase 3 are the most
crucial in terms of time and cost spent on potential candidates. Phase 2 trials provide key
information to determine the feasibility and potential success of a drug candidate, helping to
ensure that resources are being focused on the most promising compounds. Successful or
unsuccessful completion of Phase 2 for an individual drug will cost the pharmaceutical
companies around 20% of the sum spent on the drug discovery pipeline recalculated for
each individual drug. At the same time, Phase 3 trials play a critical role in the drug
discovery process by providing the necessary data to determine the safety, efficacy and
overall profile of a drug candidate, and helping to ensure that it is suitable for widespread
use.
It is estimated that approximately only 30% of people involved in drug development
work on projects that eventually become successful and reach the market. The high failure
rate in drug development is due to a combination of factors, including:
● The complex and challenging nature of drug development, including the difficulty of
finding effective treatments for many diseases;
● The high costs and long timelines associated with clinical trials, which can lead to
projects being abandoned if they fail to show promise;
● The regulatory hurdles and the stringent requirements for approval, which can lead to
many drugs failing to gain approval despite promising results in the early stages of
development;
● The high failure rate of clinical trials, where many drugs fail to demonstrate efficacy
or safety in larger populations.
Despite these challenges, the success of even a single drug can have a significant
impact and drive significant benefits for patients and society, making the pursuit of drug
development an important and ongoing effort.
