Global Orphan Drug Pipeline & Regulatory Insight 2025

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" 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 levels 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.

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

Figure 2-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time

Figure 2-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs

Figure 3-1: Global - Orphan Drugs Market Value (US$ Billion), 2017-2025

Figure 3-2: US - Orphan Drugs Market Value (US$ Billion), 2017-2025

Figure 3-3: Europe - Orphan Drugs Market Value (US$ Billion), 2017-2025

Figure 3-4: Asia* - Orphan Drugs Market Value (US$ Billion), 2017-2025

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


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