Orphan Drug


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Pacific-Link Consulting is the agency for all of drug consultant and biotechnology is providing here. Contact: San Diego, CA 92127 Phone: 858-335-1300

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Orphan Drug

  1. 1. 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) ..
  2. 2. 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
  3. 3. 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
  4. 4. 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
  5. 5. 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
  6. 6. 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.
  7. 7. 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
  8. 8. 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
  9. 9. 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.
  10. 10. 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
  11. 11. 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.
  12. 12. Pacific-Link Consulting Services 8195 Run of the Knolls Court San Diego, CA 92127 Phone: 858-335-1300