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.

Orphan Drug

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.

Orphan Drug

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

Pre-IND Meeting

Multiple informal discussions, consultations and negotiation

Early FDA Interactions and PreIND Meeting

File NIH RAC

File IND

Clear IND

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.

OrphanDrug

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

Phone: 858-335-1300