Orphan Medication Status in Japan (Orphan Item Approvals)
Key offerings: Create Technique and Certification to Acquire Orphan Medication Status in Japan
Identify regulating path
Literature Look for the Industry (the variety of individual and healthcare practice)
Identify Unmet Medical Needs for Orphan Products
Consultation with KOLs in Japan
Negotiate with MHLW (Ministry of Wellness, Work and Welfare)
Manage Medical Trials
File NDA for Orphan Products
Support Integrating Initiatives for Orphan Products
Apr 14, 2010
PLC Appointed as JETRO US Medical Business Advisor
Pacific-Link Consulting (PLC) ..
Our consultants have particular expertise with obtaining Orphan Drug Designations in Japan and the United
States for novel biologics and drugs with a successful track record of approves through the PMDA and FDA. We
also specialize in Japanese Bridging Strategies and clinical trial applications in Japan forOrphan Drugproducts.
Pacific-Link is committed to support clients in meeting their business objectives, project milestones and
customizes our approach to the needs of the client company.
Pacific-Link Consulting can help the clinical improvement needs for our customer's starting clinical trials.
Key offerings: Regulatory Strategy Development and Consultation
JapaneseOrphan DrugDesignation and Bridging Strategies
Medical and Technical writing for reg. submissions (e.g. CSRs, CMC Sections, Module 2 Summaries)
Preparation of electronic Common Technical Dossier (eCTD) applications for submission to Health Authorities
Investigational New Drug Applications (INDs) and Investigational Medicinal Product Dossiers (IMPDs) in the Unites States, Japan and Europe
Our expert Regulatory staff can develop strategic regulatory plans, facilitate Agency in the United States and Japan.
Key offerings:Orphan Drug& Designation prepare documents
Request and hold meetings
Dr. Tanimoto is a pharmaceutical professional with over 15 years of experience in clinical research and development of biotechnology products. She is the
Managing Director for Pacific-Link Consulting providing a range of development services to biotechnology and pharmaceutical companies.
Ms. Joyce Reyes is an executive with over 15 years of Regulatory Affairs and Quality Assurance experience in various roles at both small and large
biotechnology or pharmaceutical companies. Ms. Reyes has extensive experience in cell and viral production processes and has been in both
manufacturing and quality assurance roles prior to joining regulatory groups at various companies.
Orphan Drug Designation in Japan (Orphan Product Approvals)
·Develop Strategy and Documentation to ObtainOrphan DrugDesignation in Japan
·Identify regulatory path
·Literature Search for the Market (the number of patient and medical practice)
·Identify Unmet Medical Needs for Orphan Products
·Consultation with KOLs in Japan
·Negotiate with MHLW (Ministry of Health, Labor and Welfare)
·Manage Clinical Trials
·File NDA for Orphan Products
Full Project Management
Manage Manufacturing site and prepare study drug for clinical trial
Manage GLP Toxicology Studies
Work with Academia on Preclinical Program
FDA Interactions and Consultation
Pre-Pre IND Consultation
Multiple informal discussions, consultations and negotiation
Early FDA Interactions and PreIND Meeting
File NIH RAC
Prepare and conduct Phase I/II Stud
An overview on Japan Orphan Designation
In Japan, drugs and medical devices could be designated asorphan drugs
or medical devices based on the Article 77-2 of the Pharmaceutical
Affairs Law. This was in the event that they are expected for use
in fewer than 50,000 patients in Japan. For which there is a high
medical necessity. They are designated by the Minister of Health,
Labor and Welfare based on the idea of the Pharmaceutical Affairs
and Food Sanitation Council (PAFSC).
•Japan Orphan Designationdoes not immediately prompt promoting approbation.
•The objectives and diagram of the system are described beneath.
The basis of the designation system
TheMHLWOrphandrug/medical apparatus system had established
drugs and medical devices to be used for the treatment of troublesome
to-treat diseases and (AIDS) had not been sufficiently created.
This was despite the high medical needs because the amount of
patients was small. With the diversification of open healthcare needs,
safe and quality medical products were obliged to be supplied to
patients as soon as possible. As needs be, there is a huge increase in rising
open expectations and the changing circumstances of drug and medical-
mechanism research and improvement. It had been chosen to take special
measures to support and push research activities for the advancement
of orphan drugs/medical devices.
Criteria for designation
The Minister of Health, Labor and Welfare may designate drugs
and medical devices satisfying the accompanying criteria as orphan drugs
medical devices. This was in the wake of getting applications for
OrphanDrugfrom the applicants. The amount of
patients who may use the drug or medical mechanism should be less
than 50,000 in Japan. The amount of patients could be estimated
based on the report of Health and Labor Science Research or the
information published by solid scientific societies. The amount of patients
with a challenging to-treat disease is sometimes troublesome to estimate
correctly because of absence of research on the patient populace.
Pacific-Link Consulting Services
8195 Run of the Knolls Court
San Diego, CA 92127