Global Information
회사소개 | 문의

세계의 희귀의약품 파이프라인 및 규제 인사이트

Global Orphan Drug Pipeline & Regulatory Insight 2025

리서치사 PNS Pharma
발행일 2018년 09월 상품 코드 693032
페이지 정보 영문 2200 Pages
가격
US $ 3,300 ₩ 3,725,000 PDF by E-mail (Single User License) help
1명만 이용할 수 있는 라이선스로 편집 및 인쇄가 불가능합니다.
US $ 4,500 ₩ 5,079,600 CD-ROM help
CD-ROM 의 형태로 우편 배송되며 편집 및 인쇄가 불가능합니다.
US $ 5,200 ₩ 5,869,700 Hard Copy help
Hard Copy의 형태로 우편 배송됩니다.
US $ 6,000 ₩ 6,772,800 PDF by E-mail (Multi-User License) help
동일 기업 내의 모든 직원들이 이용할 수 있는 라이선스입니다. 편집은 불가능하며 인쇄는 1회에 한해 가능합니다.

주의 : 본 보고서는 라이선스에 따라 자료 이용 및 인쇄 등에 제약이 있을 수 있습니다. 자세한 내용은 라이선스 설명문( )을 참고하여 주시기 바랍니다.



세계의 희귀의약품 파이프라인 및 규제 인사이트 Global Orphan Drug Pipeline & Regulatory Insight 2025
발행일 : 2018년 09월 페이지 정보 : 영문 2200 Pages

한글목차

세계의 희귀의약품(Orphan Drug) 시장, 임상시험 동향, 규제 환경에 대해 조사했으며, 시판화 촉진요인, 주요 지역의 임상시험 규제, 단계·적응증·기업별 인사이트, 임상시험중인 치료제에 관한 인사이트, 출시된 치료제의 임상 인사이트, 향후 전망 등의 정보를 전해드립니다.

제1장 희귀의약품 : 희귀질환의 투약 계획

제2장 희귀의약품 시판화 촉진요인

  • 비용 측면
  • 특허 의약품의 만기
  • 경제적 및 비경제적 유인
  • 특허 및 시장 독점의 이점
  • 연구개발(R&D)에 대한 고액의 투자

제3장 세계의 희귀의약품 시장 전망 : 현재 시장 평가

제4장 세계의 희귀의약품 시장 : 세분화

  • 시장 : 클래스별
  • 시장 : 치료 용도별
  • 시장 : 지역별

제5장 세계의 희귀의약품 임상 파이프라인 개요

제6장 세계의 희귀의약품 지정 기준

  • 미국
  • 유럽
  • 아시아 및 호주
    • 일본
    • 대만
    • 한국
    • 호주

제7장 세계의 희귀의약품 의료비 상환 정책

  • 미국
  • 유럽
  • 아시아

제8장 FDA의 희귀의약품 임상시험 규제

제9장 EMA의 희귀의약품 임상시험 규제

제10장 아시아의 희귀의약품 임상시험 규제

제11장 세계의 희귀의약품 임상 파이프라인 : 기업별, 적응증별, 단계별

  • 불명
  • 연구중
  • 전임상
  • 임상
  • 단계 I
  • 단계 I/II
  • 단계 II
  • 단계 II/III
  • 단계 III
  • 등록전
  • 등록

제12장 세계의 출시된 희귀의약품 임상 인사이트 : 기업별, 적응증별

제13장 경쟁 상황

  • AOP Orphan
  • Agenus
  • Alexion
  • Bristol Myers Squibb
  • Biogen Idec
  • Celgene
  • Eli Lilly
  • Genethon
  • Genzyme Corporation
  • Glaxosmithkline
  • Merck
  • Novartis Pharmaceuticals
  • Orphan Europe
  • Pfizer
  • Prosensa
  • Rare Disease Therapeutics
  • Roche
  • Sanofi
  • Shire
  • Teva Pharmaceutical
KSM 18.09.14

영문목차

" Global Orphan Drug Pipeline & Regulatory Insight 2025" Report Highlights:

  • Global Orphan Drug Market Overview: US$ 260 Billion Opportunity
  • Global Orphan Drug market by Segment
  • Regulatory Landscape: USA, Europe & Asia
  • Orphan Drug Designation & Reimbursement Policy: USA, Europe & Asia
  • Global Orphan Drug Clinical Pipeline Insight: 973 Drugs
  • Marketed Orphan Drug Clinical Insight: 366 Drugs

The global orphan drug market opportunity is expected to surpass US$ 260 Billion by 2025 as per recent research report "Global Orphan Drug Pipeline & Regulatory Insight 2025" published by PNS Pharma. The orphan drug segment has emerged as a new growth frontier for pharmaceutical companies faced with issues like patent expiry, competition from generics and biosimilars, exhausting pipelines and stringent regulatory and policy framework. Increasing investment in clinical research and rapid commercialization of orphan drugs has resulted in the paradigm shift in operational strategy of pharmaceutical companies. In past, pharmaceutical companies were more focused on the research and development of essential medicines leading to high competition and low financial margins.

The exponential growth in global orphan drug market is driven by favorable regulatory and policy framework, increasing research and developmental activities, high ROI rates , government incentives for drug developmental, market exclusivity along with strong support from FDA and EU Commission in special protocols. The research and developmental activities for orphan drugs have also received impetus thrust from the pharmaceutical companies efforts to overcome the impact of revenue loss due the patent expiry of blockbuster drugs.

In terms of therapeutic application, oncology has been predominant segment in the global orphan drug market landscape followed neurology, rheumatology, hematology, infectious diseases and others. Currently, more than 200 companies are partnering for research and development of more than 300 orphan designated drugs in oncology segment. Oncology, as a therapeutic area has also transformed from evolution to customized or precision medicine in the pharmaceutical industry. Utilizing biomarkers to develop targeted treatments and therapies is an approach which is being increasingly used in recent years, which also further explains the reasons behind this therapeutic area's majority share of orphan drug approvals.

“Global Orphan Drug Pipeline & Regulatory Insight 2025” report gives comprehensive insight on clinical and non-clinical parameters related to development and commercialization of orphan drug market. As per report findings, there are more than 350 orphan designated drugs commercially available in the market and more than 900 in the clinical pipeline. Most of the orphan drugs in clinical pipeline are in Phase - II trials followed by Phase - III trials. More than 30 new orphan designated drugs are further expected to enter the market in next five years.

Table of Contents

1. Orphan Drugs: Regimens For Rare Diseases

2. Drivers of Orphan Drug Commercialization

  • 2.1. Cost Aspects
  • 2.2. Expiration of Patented Drugs
  • 2.3. Economic & Non-Economic Incentives
  • 2.4. Advantage of Patent & Market Exclusivity
  • 2.5. High Investment in Research & Development

3. Global Orphan Drug Market Perspectives: Current Market Evaluations

4. Global Orphan Drug Market by Segmentation

  • 4.1. Market by Class Variation
  • 4.2. Market by Therapeutic Application
  • 4.3. Market by Regions

5. Global Orphan Drug Clinical Pipeline Overview

6. Global Orphan Drug Designation Criteria

  • 6.1. US
  • 6.2. Europe
  • 6.3. Asia & Australia
    • 6.3.1. Japan
    • 6.3.2. Taiwan
    • 6.3.3. South Korea
    • 6.3.4. Australia

7. Global Orphan Drug Reimbursement Policy

  • 7.1. US
  • 7.2. Europe
  • 7.3. Asia

8. FDA Regulation for Clinical Trials Orphan Designated Drugs

  • 8.1. Content & Format Of A Request For Written Recommendations
  • 8.2. Provision For Granting & Refusing Written Recommendations
  • 8.3. Content And Format Of A Request For Orphan Drug Designation
  • 8.4. Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor
  • 8.5. Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs
  • 8.6. Deficiency Letters And Granting Orphan Drug Designation
  • 8.7. Refusal To Grant Orphan Drug Designation
  • 8.8. Amendment & Change In Ownership To Orphan Drug Designation
  • 8.9. Publication & Revocation Of Orphan Drug Designations
  • 8.10. Annual Reports Of Holder Of Orphan Drug Designation
  • 8.11. Scope & FDA Recognition Of Orphan Drug Exclusive Approval
  • 8.12. Protocols for Investigations & Availability of Information

9. EMA Regulations for Clinical Trials of Orphan Designated Drugs

  • 9.1. Committee for Orphan Medicinal Products
  • 9.2. How to Apply for Orphan Designation in Europe
  • 9.3. Marketing Authorization & Market Exclusivity
  • 9.4. Transferring An Orphan Designation To Another Sponsor
  • 9.5. Mandatory Submission Of Annual Report On Development
  • 9.6. Incentives For Micro, Small And Medium-Sized Enterprises
  • 9.7. Fee Reductions For Designated Orphan Medicinal Products
  • 9.8. Procedure for Orphan Designation & Incentives for R&D ( Regulation (EC) No 141/2000)

10. Asian Regulations for Clinical Trials of Orphan Designated Drugs

  • 10.1. Taiwan Rare Disease and Orphan Drug Act
  • 10.2. Japan Orphan Drug Regulation

11. Global Orphan Drug Clinical Pipeline by Company, Indication & Phase

  • 11.1. Unknown
  • 11.2. Research
  • 11.3. Preclinical
  • 11.4. Clinical
  • 11.5. Phase-I
  • 11.6. Phase-I/II
  • 11.7. Phase-II
  • 11.8. Phase-II/III
  • 11.9. Phase-III
  • 11.10. Preregistration
  • 11.11. Registered

    12. Marketed Global Orphan Drug Clinical Insight by Company & Indication

13. Competitive Landscape

  • 13.1. AOP Orphan
  • 13.2. Agenus
  • 13.3. Alexion
  • 13.4. Bristol Myers Squibb
  • 13.5. Biogen Idec
  • 13.6. Celgene
  • 13.7. Eli Lilly
  • 13.8. Genethon
  • 13.9. Genzyme Corporation
  • 13.10. Glaxosmithkline
  • 13.11. Merck
  • 13.12. Novartis Pharmaceuticals
  • 13.13. Orphan Europe
  • 13.14. Pfizer
  • 13.15. Prosensa
  • 13.16. Rare Disease Therapeutics
  • 13.17. Roche
  • 13.18. Sanofi
  • 13.19. Shire
  • 13.20. Teva Pharmaceutical

List of Figures

  • Figure 4-1: Biological & Non Biological Orphan Drug Segment (%), 2017 & 2025
  • Figure 4-2: Biological & Non Biological Orphan Drug Market (US$ Billion), 2017-2025
  • Figure 4-3: Global - Orphan Drugs Market by Therapeutic Area, 2017 & 2025
  • Figure 4-4: Regional Markets for Orphan Drugs, 2017 & 2025
  • Figure 5-1: Global - Orphan Drug Clinical Pipeline by Phase (%), 2018 till 2025
  • Figure 5-2: Global - Orphan Drug Clinical Pipeline by Phase (Number), 2018 till 2025
  • Figure 5-3: US - Orphan Drugs Clinical Pipeline by Phase (%), 2018 till 2025
  • Figure 5-4: US - Orphan Drugs Clinical Pipeline by Phase (Number), 2018 till 2025
  • Figure 5-5: Europe - Orphan Drug Clinical Pipeline by Phase (%), 2018 till 2025
  • Figure 5-6: Europe - Orphan Drug Clinical Pipeline by Phase (Number), 2018 till 2025
  • Figure 10-1: Japan - Orphan Drug/Medical Device Designation System
  • Figure 10-2: Japan - Orphan Drug/Device Designation Process

List of Tables

  • Table 2-1: Orphan Drugs in the US and their Costs
  • Table 2-2: Orphan Drugs in the EU and their Costs
  • Table 2-3: Drugs Patent Expiry in 2014
  • Table 2-4: Drugs Patent Expiry in 2015
  • Table 2-5: Drugs Patent Expiry in 2016
  • Table 2-6: Drugs Patent Expiry, 2017-2022
  • Table 2-7: Orphan Drug Incentives by Country
  • Table 2-8: Incentives for the Development of Orphan Drugs
Back to Top
아시아 최대 시장정보 제공
전세계에서 발행되고 있는 모든 시장조사보고서를
다루고 있습니다.
사이트에서 검색되지 않는 보고서도 문의 바랍니다.
 
BCC Research