NEW DELHI | 4th February 2021: “Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026” Report Highlights:

  • Global Orphan Drug Market Opportunity:  US$ 300 Billion
  • US Dominates Global Orphan Drug Market: 50% Market Share
  • US Orphan Drug Opportunity To Surpass: US$ 150 Billion
  • Global Orphan Drug Clinical Insight: More Than 900 Drugs
  • Clinical Insight on Marketed Orphan Drugs: More Than 400 Drugs
  • Oncology To Dominate Orphan Drug Development: 35% Share
  • FDA & EMA Regulations For Orphan Drugs
  • Orphan Drug Designation Criteria & Reimbursement Policy by Country

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Orphan drug market is dedicated to treat the disease conditions that are rare as well as life-threatening to small percentage of patient population at global level. As per witnessed for the global orphan drug market, it is analyzed that the overall cost of the development of orphan drugs is much higher than the drugs that are considered to be working against some of the common diseases such as cardiovascular diseases due to the less availability of the participants for the clinical trials and less drug numbers in the market. Such challenges observed in the orphan drug market is estimated to be taken into severe consideration by the government of different economically strong countries and with respect to this, to overcome supply chain issues hundreds of strategies and investment plans are encouraging the market to boost and meet the needs of the patient population.

In the past few years, it can be observed that there has been an increase in the number of opportunities that are associated with the orphan drug makers as the drug makers involved in the market are focusing on expanding the drug policies and specifically product range for each and every rare disease conditions. The forthcoming years of the orphan drug market is estimated to observe increase in the research and development as well as overall business operations and outreach, increase in the patient rights, increase in orphan drug competition, healthcare solutions getting delivered by orphan drugs and increase in the number of investors at a splendid rate. In addition to all the above-mentioned parameters, increase in the number of drugs developed for rare blood diseases and uncured diseases are estimated to explore the global market growth towards bright future.

There has been an increase in the number of research and development risk, which is aligning the market to hold true value towards the unstructured treatment paradigm for the patients who are suffering from rare disease. The future fragmentation of the market with respect to increase in competitive landscape is believed to be providing tough competition to the other traditional therapies. Specific changes being performed in the market with respect to policies and collaborations for exchanging expertise is also leading the market to adopt great dynamics in less period of time.

Global orphan drug market has led to the emergence of a transformation that is important when witnessed from public-health measures. The respective trifecta settled by the market is estimated to set up a market size in the future that will be robust and strong. The current state of the global orphan drugs in the pharmaceutical sector is estimated to determine and unravel the hidden parameters of the rare diseases that will make the researchers and the patients inclined towards the intense competition that the market is about to deliver. The analysis of the upcoming trends and opportunities associated with global orphan drug market is estimated to deliver significant development across the therapeutics research and development across industry.

As per report findings, it is estimated that the emergence of the market at global level has led to a progressive shift in the current healthcare sector. Within a short period of time, the market is on the urge of providing an aligned structure to the therapeutics market as well as a broad perspective of potential and promising applications that is held by the market. The global research report with respect to orphan drug market provides a deep comprehensive view about the dynamic forces that are driving the market as well as combination of opportunities, trends and challenges. In addition to all this, the report also provides a complete outlook of the market, the current profiles of the drugs and the timeline of the investigational drugs that are boosting the overall market.

Table of Contents

1. What Are Orphan Drugs?

2. Global Orphan Drug Designation Criteria

  2.1 US

  2.2 Europe

  2.3 Asia & Australia

  2.3.1 Japan

  2.3.2 Taiwan

  2.3.3 South Korea

  2.3.4 Australia

3. Market Exclusivity & Patent Protection for Orphan Drugs

4. Global Orphan Drug Reimbursement Policy

  4.1 US

  4.2 Europe

  4.3 Asia

5. Global Orphan Drug Market Opportunity Insight 2026

  5.1 Global Orphan Drug Sales Opportunity

  5.2 Market by Class Variation

  5.3 Market by Therapeutic Application

  5.4 Market by Regions

6. FDA Regulation for Clinical Trials Orphan Designated Drugs

  6.1 Content & Format Of A Request For Written Recommendations

  6.2 Provision For Granting & Refusing Written Recommendations

  6.3 Content And Format Of A Request For Orphan Drug Designation

  6.4 Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor

  6.5 Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs

  6.6 Deficiency Letters And Granting Orphan Drug Designation

  6.7 Refusal To Grant Orphan Drug Designation

  6.8 Amendment & Change In Ownership  To Orphan Drug Designation

  6.9 Publication & Revocation Of Orphan Drug Designations

  6.10 Annual Reports Of Holder Of Orphan Drug Designation

  6.11 Scope & FDA Recognition Of Orphan Drug Exclusive Approval

  6.12 Protocols for Investigations & Availability of Information

7. EMA Regulations for Clinical Trials of  Orphan Designated Drugs

  7.1 Committee for Orphan Medicinal Products

  7.2 How to Apply for Orphan Designation in Europe

  7.3 Marketing Authorization & Market Exclusivity

  7.4 Transferring An Orphan Designation To Another Sponsor

  7.5 Mandatory Submission Of Annual Report On Development

  7.6 Incentives For Micro, Small And Medium-Sized Enterprises

  7.7 Fee Reductions For Designated Orphan Medicinal Products

  7.8 Procedure for Orphan Designation & Incentives for R&D ( Regulation (EC) No 141/2000 )

8. Asian Regulations for Clinical Trials of Orphan Designated Drugs

  8.1 Taiwan Rare Disease and Orphan Drug Act

  8.2 Japan Orphan Drug Regulation

9. Global Orphan Clinical Pipeline Overview

  9.1 By Company

  9.2 Drug Class

  9.3 Formulation

  9.4 Indication

  9.5 Region

  9.6 Priority Status

  9.7 Patient Segment

  9.8 By Phase

10. Unknown Phase – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  10.1 Overview

  10.2 Clinical Pipeline Insight

11. Research Phase – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  11.1 Overview

  11.2 Clinical Pipeline Insight

12. Preclinical Phase – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  12.1 Overview

  12.2 Clinical Pipeline Insight

13. Clinical Phase – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  13.1 Overview

  13.2 Clinical Pipeline Insight

14. Phase-I – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  14.1 Overview

  14.2 Clinical Pipeline Insight

15. Phase-I/II – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  15.1 Overview

  15.2 Clinical Pipeline Insight

16. Phase-II – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  16.1 Overview

  16.2 Clinical Pipeline Insight

17. Phase-II/III – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  17.1 Overview

  17.2 Clinical Pipeline Insight

18. Phase-III – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  18.1  Overview

  18.2  Clinical Pipeline Insight

19. Preregistration – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  19.1 Overview

  19.2 Clinical Pipeline Insight

20. Registered – Orphan Drugs Clinical Pipeline By Company, Country & Indication

  20.1 Overview

  20.2 Clinical Pipeline Insight

21. Marketed Orphan Drugs Clinical Insight By Company, Country & Indication

  21.1 Overview

  21.2 Clinical Pipeline Insight

22. Competitive Landscape

  22.1 AOP Orphan

  22.2 Agenus

  22.3 Alexion

  22.4 Bristol Myers Squibb

  22.5 Biogen Idec

  22.6 Celgene

  22.7 Eli Lilly

  22.8 Genethon

  22.9 Genzyme Corporation

  22.10 Glaxosmithkline

  22.11 Merck

  22.12 Novartis Pharmaceuticals

  22.13 Orphan Europe

  22.14 Pfizer

  22.15 Prosensa

  22.16 Rare Disease Therapeutics

  22.17 Roche

  22.18 Sanofi

  22.19 Shire

  22.20 Teva Pharmaceutical