Drug market speed-up opportunity: not meeting the scope of clinical needs

December 20, the CFDA issued the Technical Guidelines for conditional approval of clinically urgently needed drugs for market (draft for comments) and the Measures for the Management of Drugs for The Use of Clinical Trials for Extended Compassion (Draft for Comments). Both drafts are implementing the requirements of the General Office of the CPC Central Committee and the General Office of the State Council on deepening the reform of the review and approval system and encouraging the innovation of pharmaceutical medical devices ("Article 36") on speeding up the clinical urgent need for drug review and approval and supporting expanded clinical trials, which will be conducive to promoting the enthusiasm of China's new drug innovation.
, however, some of these details need to be carefully worked out and interpreted to find innovative projects that are truly suitable for domestic companies.The range of "unsealed clinical needs" is large:
the best breakthrough point?in accordance with China's previous regulations, domestic new drugs before the market needs to spend a long clinical time, low return on investment, resulting in China's domestic manufacturers to invest in new drugs lack of motivation. The publication of the Technical Guidelines for Conditional Approval of Clinically Urgent Medicines (Draft for Comments) means that clinically urgently needed medicines (i.e., drugs that prevent or treat serious diseases or reduce their progression to greater severity, including medicines for rare diseases) are expected to shorten their market life.
The need to be aware that not only are treatments that are not currently available "unsympathetic to clinical needs", but that improvements, supplements, combinations, safety improvements, and treatments to address emerging or anticipated public health needs are expected to shorten the time to market."
definition of "unsealed clinical needs" promotes diversity in drug innovation. According to the author, the relatively easy breakthroughs are "the efficacy of existing therapies, but can avoid the serious toxicity of existing therapies or reduce harmful drug interactions or improve patient compliance" and "replace drugs that are not compatible with other key drugs in the location of existing drugs."
, as shown in the Schedule, more than 50 drugs are included in the list to be included in the priority review list because of their clinical advantage or clinical value, and are mainly imported drugs.three categories of drugs have been "conditional approval for listing":
to speed up the listing also need rules
three categories of "conditional approval of listing" drugs in particular caused concern.
the first clinical study involving the application of alternative endpoint indicators or intermediate clinical endpoint indicators can predict products that are likely to have efficacy and clinical benefits. Intermediate clinical endpoint indicators are generally used for clinical benefit evaluation of chronic diseases, and short-term clinical benefits are generally considered likely to predict long-term clinical benefits. As with FDA regulations, the draft notes that clinical research alternative endpoints are generally biomarker indicators. However, the FDA's biomarker qualification has a set of identification procedures, the draft for comments has not yet seen the relevant identification procedures.
the second is based on early or interim clinical trial data that reasonably predicts or determines its clinical benefits and has a clear advantage over existing treatments, allowing conditional approval to be made before the completion of a corrorogical clinical trial. Early or interim clinical trial data are clinical trial data submitted before the clinical trial program has been completed. Corroroative clinical trials generally refer to the clinical phase III. Complete phase III. Preclinical conditional approval listing, that is, complete Phase II clinical conditional listing can be listed, it is expected that the future completion of Phase II clinical communication declaration of the listing of new pharmaceutical products will be more and more.
Previously, the CFDA has issued the General Administration Office of public consultation on the adjustment of drug clinical trial review and approval of the announcement (draft for comments), the process of clinical application of new drugs accelerated, if the completion of Phase II clinical conditional listing, the corresponding new drugs are expected to declare clinical to declare the listing only 5 years (and before the review and approval system reform, the time of new drug market is expected to be 8 to 10 years), the time is greatly shortened.
third is a rare disease treatment drug that has been approved for sale abroad. For rare disease drugs that have been approved for listing abroad, applicants may apply data supporting their overseas approval for listing to directly declare conditional approval for listing. Foreign rare disease drugs often also take the priority review and approval of the green channel, complete phase II clinical declaration and listing, then support its overseas approval of listing data can accept clinical relief?
noteworthy is that innovative drugs can be qualified for early listing, but it is not clear whether rare disease generics approved for listing abroad can also use this rule to obtain early listing. In addition, rare disease generic drugs may even be exempt from clinical listing abroad, domestic rare disease generic drugs can refer to foreign generic rare drug system as appropriate clinical exemption?
in addition to chemicals and biological products, Chinese medicine can also shorten the development time of clinical trials, early market. Xanda data V3.5 found that only 4 Chinese medicine products entered the list of priority review, of which 3 are children's medicine, respectively, children's malong cough flat wheezing particles, children's halal oral solution and soil clear heart particles. Only myocardial particles were included in the proposed priority review list on the grounds of rare diseases and clinically urgent needs.expanding compassion for the use of clinical trials:
public welfare projects?Sym developmental compassionive use of clinical trials is to allow the use of drugs that have not yet been approved for market in institutions conducting clinical trials to patients in urgent need when patients are unable to obtain drugs for clinical trials by participating in clinical trials. This is primarily aimed at patients who are unable to participate in a new drug registration clinical trial due to non-compliance with the trial group/exclusion criteria, or who are unable to participate in a new drug registration clinical trial due to geographical or time constraints, etc.
A drug that allows for expanded sympathetic use clinical trials means that a registered clinical trial has ended but the drug has not yet been approved for sale in China, and available research data initially indicate the possible effectiveness and safety of the drug among the chinese population intended to be registered with indications.
Given that research data for extended clinical trials are generally not used as the subject of registration applications, they can only be included as supporting security data in registration filings, which means that extended clinical trials are not "free vehicles" for use as hyper-adaptives, and companies are more likely to apply validity data to marketing, observing literature releases, and driving changes in clinical guidelines.
addition, the expanded use of experimental drugs is limited to the use of institutions conducting Phase II, III. clinical trials, the number of users shall not exceed the number of subjects prescribed in clinical trials. In principle, registered applicants are not allowed to charge for drugs used in clinical trials during extended clinical trials. This means that expanded clinical trials are more of a public good, giving sympathetic drugs to diseases that currently have no effective means of treatment. It is expected that pharmaceutical enterprises will be more motivated to carry out expanding clinical trials.
outlook >>>The Technical Guidelines for conditional approval of clinically urgently needed medicines for market (draft for comments) are good for the innovative drugs needed for clinical use and increase the enthusiasm of pharmaceutical companies to invest in innovative drugs.
, however, pharmaceutical companies need professionals who can really assess whether a project can be listed on a conditional basis. After all, for a new drug to be eligible for market, the research and development team must be familiar with the overall planning of clinical trials, especially the end-of-clinical design of clinical treatments, the pros and cons of clinical guidelines, and have expert resources in the field of adaptation within the company.
are not many teams in the country that can complete the entire process, the high risk of investing in new drugs will still be the root cause of most drug companies' ability to rush to invest in the field.