1. Application for approval and real world data (3 lines)

• Application for approval:It is a procedure to collect evidence collected in clinical trials and ask the regulatory authority to determine whether to sell this medicine. In the United States, NDA (new drug approval application), BLA (biological formulation approval application), MAA (sales approval application) in the EU, and manufacturing approval application in Japan
• What is the speeding system:to quickly deliver the drugs of heavy illnessesGet advice during development、Shorten Examination Period、Approve with early-stage data and check laterThe system of the three directions is in Europe and Japan.
• Real World Data:FDA"Data on the provision of patient health and medical care collected from various sources on a daily basis"Examples of electronic medical records, medical remuneration claim data, product and disease registry, etc.Facts

2. New Drug Approval Flow - From Clinical Trial Notification to Approval

According to the FDA description, legal ex s are required to transport clinical trials across the state,"Ind is a means to obtain technically this application ex、 from the FDA"HomeFactsHome IND requires the following three areas:Facts。

AllFacts(FDA "Investiga。 New Drug (IND) Application" [Reference Material 1]) FDA after submission of IND30 daysFDA has the opportunity to examine in terms of safety, not beginning clinical trialsFacts。

There is a mechanism of the same way of thinking in Japan. According to PMDAA person who has received a clinical trial plan for the first time about a new active ingredient drug, etc., must not ask a medical institution or start a clinical trial if it is not after 30 days from the date of the notification.Contact UsFactsHome PMDA attaches to clinical trial reportEvaluation and management of DNA reactivity (mutatogenic) impuritiesThe following examples are also published.Facts。 Management of impurities is questioned from the entrance of clinical trialsThat is This article explains.

3. Type of application document – 5 modules of CTD

The application form for approval is supported by a common type in Japan and the United States. ICH“All information on quality, safety, and efficacy has revolutionized the regulatory review process.”Facts。

AllFacts(ICH "M4: The Common Technical Technical" [Reference Material 3]) CTD according to ICHNovember 2000andEU and Japan 2003、US FDA 2017It has become a mandatory form of new drug application. Part 2 - 5All Regionsis intendedFacts。

4. From the perspective of material ians 1: Who will enter the material information?

5. Japan-U.S. speeding system

The system for fast delivery of heavy disease drugs is located in each of Japan and the United States. NameWhat to do fasterIt is better to organize them (This article).

AllFacts(FDA [Reference Materials 4/5], FDA [Reference Materials 7/8/9], Ministry of Health, Labour and Welfare [Reference Materials 10/11/12])Three lines are divided by this article.Each system may have multiple personalities. Example: Japanese pioneering drugs include both consultation and examination.

(1) U.S.A. – 4 Speeding Programs

FDA2014dance in May 2014Fast Track、Breakthrough Therapy、Accelerated Approval、Priority ReviewCompare 4FactsHome Breakthrough Therapy2012 FDA Safety and Innovation Act (FDASIA) Article 902by FD&C law 506 (a) added specificationFactsHome Priority Review"The goal is to dispose of within 6 months from receipt" (10 months in standard examination)Contact UsFactsHome Footdance is a footnote for PDUFA V's "Program" (for NDA and new BLA of new active ingredients received from October 1, 2012 to September 30, 2017).The examination clock starts from the end of the 60 calendar day acceptance periodExplainFactsHome For the mechanism of accelerated approval (Ac rated Approval) and substitute endpoints, we will handle it in detail in this series "biomarkers and clinical evaluation indicators".

(2) EU – PRIME, Quick Review, and Conditions

The evaluation of sales approval application by the central examination method is"It can take up to 210 days without counting the clock stop for the applicant to exit additional information."HOME CHMP if there is enough ground150 daysto shortenFactsHome Conditional Sales Approval"The benefit of the drug to use immediately exceeds the risk of being within the fact that additional data is still necessary."In cases, it is usually recognized by less clinical data, 1 yearcan be switched to standard sales approval if you fulfill certain obligationsFactsHome In the case of a public health emergency (such as pandemic)"Less pharmacological and nonclinical data can also be accepted"Facts。

(3) Japan – pioneering drug and conditional approval

The Ministry of Health, Labour and Welfare is one of the requirements for pioneering pharmaceuticals.Using Innovative Drug Delivery SystemHomeFacts。 The DDS (drug delivery system) itself can be the basis of the system’s “experience”That is This article explains. 4thThe intention and system to apply in Japan ahead of the world (or simultaneously)from the first country of applicationApplication within 3 months shall be considered as concurrent applicationContact UsFacts。

Notification of conditional approval (August 31, 2020, October 23, 2024 amended) is a serious disease and has superior medical clarity than existing methods, It is difficult to conduct a clinical trial, or it takes a considerable period of time to conduct such as few patients.PharmaceuticalsFactsHome not necessarily clinical trials,Research using medical information database such as MID-NET and patient registryAlso availableFactsHome Connect with real world data in Chapter 8

6. The review of this paper: How much is the review period?

How much is it actually used? According to the annual report of FDA (CDER),46 new drugs approved in 2025FactsHome The speeding system was used as follows:Facts。

46 items32 items (70%) approved in the United States first from other countriesComment39 items (85%) approved in the first review cycleComment 44 items (96oz)Also noteFactsHome Examination period1-2 months from 10-12 monthsThe first disposal of the new "Commissioner's National Priority Voucher(CNPV)" aiming to shrink toFactsHome How to use this new system in the futureUndetermined / FutureComment

7. Material Technician Perspective 2: The faster the screening, the more the manufacturing is done

8. Real World Data (RWD) and Real World Evidence (RWE)

(1) Definition and Legal Position

FDA RWE"Clinical evidence of use and potential benefits or risks of medical products obtained from RWD analysis"definedFactsHome In other wordsRWD material (data), conclusions RWE led from there (evidence)It is a relationship This article explains.

FDA meets thisRWE in 2018 FrameworkWe make and evaluate the approval of new efficacy of approved drugs and the use of them for test requirements after approval.FactsHome FDA RWD and RWELong-term safety monitoring and evaluationbut use for backing of efficacyMoreIt is writtenFacts。

(2) Examples that were actually used for approval

On July 16, 2021, the FDA will prevent the refusal reaction after transplantation.Lung transplantationNew efficacy has been approved. FDA"The study of non-intervention (observation) that provides efficacy real world evidence"Approve and explainFacts。

• Data Source:U.S. Department of Health and WelfareSRTR Registry of Transplant RecipeRWD. USAAll Lung TransplantsData collected about the death record of the Social Security AgencyFacts
• Why is it recognized?FDAA well-designed non-intervention study that reflects the appropriate contrast based on the RWD (fit-for-purpose) for the right purposeFDA Regulation“Appropriate and Fully Managed”It indicates that it is considered as above. Comparison contrast is not immune suppression therapy (or minimal)Well recorded natural courseCommentFacts
• Other evidence:Randomized comparison tests with other solid organ transplantsVerified ProofJoin asFacts

RWD has not decided to approve it alone, but the condition “return is objective, and the natural course of contrast is well known” and the evidence of other tests have been obtained.Points are important This article explains. This article does not evaluate the effectiveness and safety of this drug.

(3) EU and Japan RWD Foundation

AllFacts(Reference Material 16, PMDA, Ministry of Health, Labour and Welfare [Reference Material 12])

9. Lag and Loss

Ministry of Health, Labour and WelfareRossHome"The drug approved by pharmaceutical has not been developed in Japan"ExplainFactsHome In general, the condition that the approval is delayed although it is developed in JapanLagIt is called (explanation by this article).

As one of the measures, the Ministry of Health, Labour and Welfare noticed on December 25, 2023.Pharmaceuticals Precedented by Clinical Development OverseasBefore Japan participates in international joint studiesIn principle, there is no need to perform additional trials in Japanese unless necessary.FactsHome as a backgroundInnovative pharmaceuticals for early development of biopharmaceutical companies overseasThe number of cases in which Japanese participation is considered after early clinical development in overseasFactsHome However,Small safety medicinesIn such cases, we judge the importance of Japanese Phase I test more carefully.Facts。

10. Challenges and things that have not yet been determined

(1) Range to show "Efficacy" in RWE

The FDA uses RWE for its effectivenessMore than everHomeFactsHome An example of 2021 was a condition with objective outcomes, clear natural passages, and evidence of other tests.Facts。 The general rule of what conditions can indicate the effectiveness of RWE is established at the time of the investigation of this article (September 2026)Undetermined / Future。

(2) How to make new speeds

U.S. CNPV (Aiming to shrink the examination in one to two months)First phase of disposalHomeFactsHome As of December 2024, FDA guidance embodying the new rules of rapid approval (the 2023 law amendment)DraftHomeFactsHome How to determine the operationUndetermined / FutureComment

(3) Effect of anti-drug loss

Review of the concept of the Japanese Phase I trial, requests for development to undelivered items, public offerings, etc.How many undelivered items are reducedHome There is no track record at the time of research.Undetermined / Future。

11. Glossary

12. Reference materials (primary information)

1. FDA“Investiga) New Drug (IND) Application” (updated on September 16, 2026) — fda.gov
2. Pharmaceutical Medical Devices (PMDA)Clinical Trial Plan Notification System for Drugs — pmda.go.jp
3. ICH「CTD — M4 : The Common Technical Document」 — ich.org
4. FDA“Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics” (PDF) — fda.gov
5. FDA「Breakthrough Therapy」 — fda.gov
6. FDA“Expedited Program for Serious Conditions — Accelerated Approval of Drugs and Biologics —ancedance for Industry (Draft)” December 2024 (PDF) — fda.gov
7. European Medicines Agency「PRIME: priority medicines」 — ema.europa.eu
8. European Medicines Agency「Accelerated assessment」 — ema.europa.eu
9. European Medicines Agency「Conditional marketing authorisation」 — ema.europa.eu
10. Ministry of Health, Labour"About pioneering drug designation system" — mhlw.go.jp
11. Ministry of Health, Labour and Welfare, Department of Health, Labour and Welfare9th Administrative Evaluation and Monitoring Committee on Drug Designation System (Pharmaceutical) Preliminary Examination Designation System) — Material 2, September 4, 2022 (PDF) — mhlw.go.jp
12. Director, Department of Pharmaceutical Examination, Ministry of Health, Labour and WelfarePartial Amendments to the Handling of Conditions-based Approvals for Pharmaceuticals, No.1023, No.2, No.6, No.2024, Oct.23 — mhlw.go.jp
13. U.S. Congress21st Century Cures Act, Public Law 114-255, December 13, 2016 — govinfo.gov
14. FDA"Real-World Evidence" (updated on June 3, 2026) — fda.gov
15. FDAFDA approves new use of transplant drug based on real-world evidence fda.gov
16. European Medicines Agency「Data Analysis and Real World Interrogation Network (DARWIN EU)」 — ema.europa.eu
17. Pharmaceutical Medical Devices (PMDA)"MID-NET" — pmda.go.jp
18. Ministry of Health, Labour"Research on Drug Loss (FY2019-)" Material 2-3, 65th Unapproved Drugs and Non-ap) Drug Review Conference, September 29, 2019 (PDF) — mhlw.go.jp
19. Director, Department of Pharmaceutical Examination, Ministry of Health, Labour and Welfare"Basic Approach to the implementation of phase I trials in Japanese before the start of international joint clinical trials of drugs prior to clinical development in overseas", Pharmaceutical Trial No. 1225 No. 2, Dec. 25, 2023 — mhlw.go.jp
20. FDA「Drug Master Files (DMFs)」 — fda.gov
21. eCFR「21 CFR 314.420 Drug master files」 — ecfr.gov
22. Pharmaceutical Medical Devices (PMDA)Original Drug Registration (MF) — pmda.go.jp
23. FDA（CDER）「Advancing Health Through Innovation: New Drug Therapy Approvals 2025」（PDF） — fda.gov

13. Response Table of Claim and Source (Audit)

Last updated: September 26, 2026 / Sources are limited to primary information (FDA, FDA, Ministry of Health, Labour and Welfare, PMDA publications and notifications, ICH publications, U.S. law and federal regulations). They are distinguished from the facts that have been sourced as "explanation" because they contain system arrangements, materials and manufacturing reads. Since the examination period differs from each country, the monthly conversion of Chapter 6 is to see the difference between standard and speed in the same country, and it is not a comparison of the country. Operation of new rules of CNPV and quick approval, conditions that show validity in RWE, and the effects of anti-drug loss are "undetermined / future" because there is no primary information confirmed. This article is an explanation of the approval system,It does not evaluate the effectiveness and safety of individual medicines, but it is not medical advice. All diagrams are illustrations for explanation. Fig. 1 - Fig. 6 is a vector drawing, and the image is an AI-generated image.We do not show the actual application documents or screening records.