New drug development is the biggest gamble in the world

Source: Sohu Health 2017-02-14 according to Chen Bo, global deputy director partner of McKinsey, "R & D", internationally known as R & D (Research and development), namely, two sets of steps of research and development When it comes to new drug research and development, people in the industry often mention two numbers The first one is "ten" years One new drug research and development takes about ten years, which is divided into two stages One is drug discovery, which takes 2-5 years The other is drug development, which takes 6-10 years, which is very long The second number is "10" Billion dollars Every new drug from the laboratory to the official market costs an average of 1 billion dollars, which is a very expensive thing Of course, if a company calculates the fund for drug experiment failure, the investment may be far more than one billion dollars, and it may cost more than two billion dollars From the mechanism research of biology itself to the start of making drugs, it involves biochemistry, pharmaceutical chemistry, pharmacology, pharmaceutics Drug testing in human body involves clinical medicine, drug law and so on It involves the cooperation of multiple scientific research teams and requires a large number of highly educated talents Screening drug molecules For example, to treat a disease, scientists should first know how the disease is caused and find the target of causing the disease This requires a lot of biological experiments to confirm that the target is indeed related to the disease itself When the target is confirmed, it needs to be handed to the team of pharmaceutical chemistry to design a compound around the target, from a large number of chemicals Or biological compounds for screening, after finding this compound, scientists then modify the chemical structure, select the molecules with cell activity, then make a reasonable preparation with pharmaceutics, through a series of pharmacological experiments for the next step of screening As a screening library, there may be hundreds of thousands of compound molecules, from which we need to find such a series of small molecules that are more suitable for this target After transformation, a large number of compounds will be screened out, which itself will go through a very long process; all compounds are divided into biological products and chemicals, which is also very different Because biological products are macromolecules, polypeptides, glycoproteins, etc., there are many kinds of biological activities, so it has a large development space, but the cost is very high; small molecule chemicals are now screened by large-scale computer, also known as "computer-aided drug design", screened many times at home and abroad, and its development space is very limited The drug itself is not purely synthesized in the laboratory, but also eaten in the body, so a large part of the work still needs to be carried out in the animal body, to see whether the drug can really act on this target and whether there are any side effects That is to say, a drug molecule originally assumed that the drug should have such a role, and the result was the opposite effect, that is, it was constantly eliminated The next step is clinical research, which is also divided into many small links For example, in the above preclinical process, large animals are used to test the effect and toxicity of the drug If the large animals are finished with the expected effect, then human body experiments can be started, and the regulatory department can be reported for filing, saying that clinical experiments will be done Clinical trials are divided into three phases, each with a different purpose The first phase of clinical medicine is to give medicine to healthy people It is just to test whether the safety of people after taking the medicine is good, whether there is any problem, and how the distribution of the body is, that is, to verify it on the human body, not necessarily on the patients, and then the next phase is reached after the safety; In the second phase of clinical trials, it is the first time to do such experiments on real patients Since all the previous results are from animals, whether the drug works or not depends on this step If in a small group of people, such as 50 patients, it can be seen that the effect of their medication is better than that of placebo, they will carry out drug experiments on a larger scale In the third phase of clinical practice, scientists will consider different races, ages, and stages of disease development, as well as the dosage of different drugs, etc to detect what the effective and safe dosage of this drug is on a larger scale, and how to use it better If it turns out to be safe and effective at last, then apply with the regulatory authorities, saying that it can be listed, then you can start to apply for registration There is also a special case, such as an infectious virus outbreak Due to the scale and strictness of clinical experiments, it often takes many years And for regulators, especially in the United States, to adopt more and more flexible ways, some diseases are very urgent, we can see whether we can skip some of these steps, or combine phase I and phase II, and directly test it on patients - both to see its safety and effectiveness Other drugs can even directly skip the third phase of clinical trials In the second phase, it is found that 50 people who do experiments have good results They can also apply for listing first, and then do the fourth phase of clinical trials later On the one hand, patients can take it, on the other hand, pharmaceutical companies and scientists need to make close observation and recall any problems as soon as they occur There is such a mechanism to prevent the safety situation after the listing, and at the same time, it can continuously collect clinical data So in the aspect of supervision, because of the higher and higher demand, more and more flexible supervision methods should be adopted, and there are many innovations in the supervision methods In some aspects, it is also because of the innovation of regulatory methods that promotes the development of new drug industry For example, a large glass of water can be used to select such a drop or two drops of water The probability of success of new drug research and development is very small, so in essence, this is a very difficult work - capital intensive, talent intensive, and risk is very huge The countries that really do new drug research and development in the world can be counted with two hands Most countries do not do this because it is too expensive In addition, even rich countries do not have so many top scientific talents In China, a large number of overseas talents have been introduced in recent years, and the investment in drug research and development is also very high, but compared with the United States and other drug developed countries and enterprises, the investment is still very limited