How long does it usually take to develop a new drug to market?

It takes about fifteen years for a new drug to be developed from clinical phase III to market launch. New drug development from clinical phase III to market launch needs to go through the following processes:

I. Preclinical research

1. Research and development (generally 2-3 years)

Laboratory research to find new compounds with potential for treating a specific disease

A. Drug target Discovery and confirmation of drug target

This is the starting point of all the work, only after the target is identified, all the subsequent work will have a basis to start.

B. Screening and synthesis of compounds

Based on the spatial structure of the target, a series of matching molecular structures are screened from the virtual compound library, and these compounds are synthesized, which are called lead compounds.

C. Validation and optimization of active compounds

Not all the lead compounds can meet the requirements, and in this stage, it is necessary to validate the compounds through in vitro cellular tests, initially screen out the compounds with high activity and low toxicity, and structurally optimize them according to the conformational relationship, which are known as drug candidates.

At the same time, there is also the case that a compound has no effect on target A, but may have very good activity on other B and C targets, which will be left aside for the time being.

2, preclinical experiments (generally 2-4 years)

The purpose of this stage is to assess the pharmacology and toxicology of the drug, drug absorption, distribution, metabolism and excretion (ADME). The second is to carry out studies on production process, quality control and stability (CMC).

The first part of the experiment needs to be carried out at the animal level, and the results of cellular experiments and those of live animal experiments can sometimes be very different. The purpose of this step is to determine the effectiveness and safety of the drug. The second part needs to be done in a GMP-compliant workshop.

A, pharmacological studies

including: pharmacodynamics, pharmacokinetics

B, toxicological studies

acute toxicity, long toxicity, reproductive toxicity, carcinogenicity, teratogenicity, mutagenicity

C, the development of preparations

Can't just get a few compounds and dump them in your mouth, the development of the preparations is an important part of the application of drugs. For example, some drugs are poorly absorbed orally, they need to be developed as injections. Some drugs will lose their activity in stomach acid, so they need to be developed into enteric formulations.

There are compounds that don't dissolve well, which can also be partially solved by formulations. Others need to be administered locally, and they need to be developed into nebulizers, creams, etc. through formulations.

Two, clinical trial approval InvestigationalNewDrug (IND)

Three, clinical trials (generally 3-7 years)

Human trials **** divided into three phases:

Phase I clinic 20-100 cases of normal people, mainly for safety evaluation.

Phase II clinical 100-300 cases, patients, mainly for effectiveness evaluation.

Phase III clinical 300-5000 cases, patients, expanding the sample size, further evaluation.

Because phase I-III clinical is very important in the whole drug development process, we focus on this part.

Traditionally, the clinical study of new drugs was divided into phase I/II/III, then phase II was divided into IIa and IIb (largely because of oncology drug research), and then the concept of phase 0 study appeared (largely because of oncology drug research and development as well). Then it was proposed that Phase 0/I would be early clinical studies, IIa would be mid-stage clinical studies, and IIb and III would be late-stage clinical studies.

This categorization is actually, I think, important in two ways for those of us who do clinical study design:

1. Staging is related to the purpose of the study, which can be divided into different periods and stages for different purposes.

2. Different periods and phases of clinical research often correspond to certain study framework designs.

Expanded information:

Phase III clinical trials of drugs:

Clinical trials of drugs are divided into phases I, II, III, and IV. Phase III clinical trials are the confirmatory phase of therapeutic effect. Its purpose is to further verify the therapeutic effect and safety of the drug for patients with the target indications, evaluate the relationship between benefits and risks, and ultimately provide a sufficient basis for the review of drug registration applications.

Trials should generally be randomized blinded controlled trials with adequate sample size. Only when the trial obtains statistically significant results, the drug regulatory authority will approve the marketing of the product.

The number of cases in the phase III clinical trial should meet the statistical requirements and the minimum number of cases.

The minimum number of cases (test group) in the phase III clinical trial is 300 cases.

Phase I clinical trials are preliminary clinical pharmacology and human safety evaluation tests. It is to observe the degree of tolerance and pharmacokinetics of the new drug in human beings, and to provide a basis for the development of the dosing regimen.

Because it is the first time to carry out drug experiments on human beings, the main purpose is twofold: one is to study the safety and tolerance of the drug in human beings, and to examine the relationship between drug side effects and drug dose increment, and the other is to examine the pharmacokinetic properties of the drug in human beings, including the identification of metabolite products and metabolism pathways of the drug in human beings.

Phase II clinical trials are the stage of preliminary evaluation of therapeutic effects. Its purpose is to initially evaluate the therapeutic effect and safety of the drug in patients with the target indication, and also to provide a basis for the study design of the phase III clinical trial and the determination of the dosage regimen. The study design for this phase can take several forms, including randomized blinded controlled clinical trials, depending on the specific study objectives.

This phase of the clinical study focuses on the safety and efficacy of the drug. The efficacy of the new drug is evaluated by applying a placebo or a marketed drug as a control drug, and in the process the influence of the disease process on the efficacy of the drug is investigated; the dosage and regimen of the drug for the phase III trial are determined; and more information on the safety of the drug is obtained.

Phase III clinical trials are the phase of confirming the therapeutic effect. Its purpose is to further validate the therapeutic effect and safety of the drug in patients with the target indication, to evaluate the relationship between benefit and risk, and ultimately to provide a sufficient basis for the review of the drug registration application. The trial should generally be a randomized blinded controlled trial with sufficient sample size.

The sample size of this trial is much larger than that of the previous two trials, and the larger sample size will help to obtain more information about the safety and efficacy of the drug, and to evaluate the benefits/risks of the drug, which will provide support for the approval and marketing of the product.

Baidu Encyclopedia - Phase III Clinical Trial