US Orphan Drug Market Outlook 2018
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In the largest market for orphan drugs, USA, there was a shortage of adequate therapies for treating many rare diseases. These therapies were not developed as companies did not expect these drugs to be highly profitable. Hence there was a lack of interest and thus investment on the part of pharma companies in the USA. Therefore, the FDA introduced incentives for developing such drugs. This step taken by the FDA was successful in creating a thriving market for orphan drugs.
It was in the USA first that a special law exclusively for governing orphan drugs was framed in the form of the Orphan Drug Act of 1983. This led to an increase in the popularity of orphan drugs. The FDA also has been continuously increasing its efforts to support this market by providing significant financial and non-financial incentives to the pharmaceutical companies to attract them. This has been one of the major drivers of growth for the US orphan drugs market.
The orphan drugs enjoy significant competitive advantages in the market owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.
A scenario where orphan drugs would be denied coverage is highly difficult to visualize. However it is most likely that the payer scrutiny would increase with new products entering the market and budgets contracting in a weakening economic environment. The plans would be based on costs and would also include payer resources, philosophies, and available benefit design options, as all these factors could affect patient access. A deep and clear understanding of the clinical and economic value of the drugs will play an increasingly important role in decision-making.
“US Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to US orphan drug market:
In the largest market for orphan drugs, USA, there was a shortage of adequate therapies for treating many rare diseases. These therapies were not developed as companies did not expect these drugs to be highly profitable. Hence there was a lack of interest and thus investment on the part of pharma companies in the USA. Therefore, the FDA introduced incentives for developing such drugs. This step taken by the FDA was successful in creating a thriving market for orphan drugs.
It was in the USA first that a special law exclusively for governing orphan drugs was framed in the form of the Orphan Drug Act of 1983. This led to an increase in the popularity of orphan drugs. The FDA also has been continuously increasing its efforts to support this market by providing significant financial and non-financial incentives to the pharmaceutical companies to attract them. This has been one of the major drivers of growth for the US orphan drugs market.
The orphan drugs enjoy significant competitive advantages in the market owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.
A scenario where orphan drugs would be denied coverage is highly difficult to visualize. However it is most likely that the payer scrutiny would increase with new products entering the market and budgets contracting in a weakening economic environment. The plans would be based on costs and would also include payer resources, philosophies, and available benefit design options, as all these factors could affect patient access. A deep and clear understanding of the clinical and economic value of the drugs will play an increasingly important role in decision-making.
“US Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to US orphan drug market:
- US Orphan Drug Market Overview
- Orphan Drug Designation Criteria
- Market Specific Reimbursement Policy & Regulatory Framework
- US Orphan Drug Pipeline by Phase, Indication & Originator
- Marketed Orphan drug List by Indication & Brand Name
- Key Issue to be Resolved
- Competitive Landscape
1. INTRODUCTION TO ORPHAN DRUGS
1.1 Orphan Drug Defined
1.2 Rising Popularity of Orphan Drugs
2. WHY SHIFT FROM NON-ORPHAN TO ORPHAN DRUGS?
2.1 Exhausting Product Pipelines
2.2 Profitability of Orphan Drugs
2.3 Increasing R&D Investment
2.4 Role of Economic Incentives
2.5 Patent Protection & Market Exclusivity
3. US ORPHAN DRUG MARKET OUTLOOK
3.1 Orphan Drug Designation Criteria
3.2 Market Overview
3.3 Reimbursement Policy for Orphan Drugs
4. US ORPHAN DRUG REGULATORY FRAMEWORK
4.1 Content & Format of a Request for Written Recommendations
4.2 Provision for Granting & Refusing Written Recommendations
4.3 Content & Format of a Request for Orphan Drug Designation
4.4 Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
4.5 Timing of Requests for Orphan Drug Designation & Designation Of Already Approved Drugs
4.6 Deficiency Letters &Granting Orphan Drug Designation
4.7 Refusal to Grant Orphan Drug Designation
4.8 Amendment & Change in Ownership to Orphan Drug Designation
4.9 Publication & Revocation of Orphan Drug Designations
4.10 Annual Reports of Holder of Orphan Drug Designation
4.11 Scope & FDA Recognition of Orphan Drug Exclusive Approval
4.12 Protocols for Investigations & Availability of Information
5. US ORPHAN DRUG PIPELINE INSIGHT BY PHASE & INDICATION
5.1 Research
5.2 Preclinical
5.3 Phase I
5.4 Phase I/II
5.5 Phase II
5.6 Phase II/III
5.7 Phase III
5.8 Preregistration
5.9 Registered
6. MARKETED ORPHAN DRUGS IN US: BRAND NAME & INDICATION
7. KEY ISSUE TO BE RESOLVED
7.1 High Initial Investment
7.2 Clinical Study Hurdles
7.3 Regulatory Hurdles
7.4 Different Reimbursement Policy
8. COMPETITIVE LANDSCAPE
8.1 Pfizer
8.2 Roche (Genentech)
8.3 Sanofi
8.4 Rare Disease Therapeutics
8.5 Glaxosmithkline
8.6 Merck
8.7 Novartis Pharmaceuticals
8.8 Alexion
8.9 Celgene
8.10 Biogen Idec
8.11 Eli Lilly
8.12 Bristol Myers Squibb
1.1 Orphan Drug Defined
1.2 Rising Popularity of Orphan Drugs
2. WHY SHIFT FROM NON-ORPHAN TO ORPHAN DRUGS?
2.1 Exhausting Product Pipelines
2.2 Profitability of Orphan Drugs
2.3 Increasing R&D Investment
2.4 Role of Economic Incentives
2.5 Patent Protection & Market Exclusivity
3. US ORPHAN DRUG MARKET OUTLOOK
3.1 Orphan Drug Designation Criteria
3.2 Market Overview
3.3 Reimbursement Policy for Orphan Drugs
4. US ORPHAN DRUG REGULATORY FRAMEWORK
4.1 Content & Format of a Request for Written Recommendations
4.2 Provision for Granting & Refusing Written Recommendations
4.3 Content & Format of a Request for Orphan Drug Designation
4.4 Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
4.5 Timing of Requests for Orphan Drug Designation & Designation Of Already Approved Drugs
4.6 Deficiency Letters &Granting Orphan Drug Designation
4.7 Refusal to Grant Orphan Drug Designation
4.8 Amendment & Change in Ownership to Orphan Drug Designation
4.9 Publication & Revocation of Orphan Drug Designations
4.10 Annual Reports of Holder of Orphan Drug Designation
4.11 Scope & FDA Recognition of Orphan Drug Exclusive Approval
4.12 Protocols for Investigations & Availability of Information
5. US ORPHAN DRUG PIPELINE INSIGHT BY PHASE & INDICATION
5.1 Research
5.2 Preclinical
5.3 Phase I
5.4 Phase I/II
5.5 Phase II
5.6 Phase II/III
5.7 Phase III
5.8 Preregistration
5.9 Registered
6. MARKETED ORPHAN DRUGS IN US: BRAND NAME & INDICATION
7. KEY ISSUE TO BE RESOLVED
7.1 High Initial Investment
7.2 Clinical Study Hurdles
7.3 Regulatory Hurdles
7.4 Different Reimbursement Policy
8. COMPETITIVE LANDSCAPE
8.1 Pfizer
8.2 Roche (Genentech)
8.3 Sanofi
8.4 Rare Disease Therapeutics
8.5 Glaxosmithkline
8.6 Merck
8.7 Novartis Pharmaceuticals
8.8 Alexion
8.9 Celgene
8.10 Biogen Idec
8.11 Eli Lilly
8.12 Bristol Myers Squibb
LIST OF FIGURES
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: US Orphan Drug Market (US$ Billion), 2012-2018
Figure 3-2: US Share in Global Orphan Drug Market, 2012 & 2018
Figure 3-3: Biological & Non Biological Orphan Drug Segment (%), 2012 & 2018
Figure 3-4: Biological & Non Biological Orphan Drug Market (US$ Billion), 2012-2018
Figure 5-1: US- Orphan Drug Development by Clinical Phase (%), 2014
Figure 5-2: US - Number of Orphan Drug by Clinical Phase, 2014
Figure 5-3: US – Number of Suspended & Discontinued Number of Orphan Drug, 2014
Figure 5-4: US - Number of Discontinued Orphan Drug by Clinical Phase, 2014
Figure 5-5: US - Number of Suspended Orphan Drug by Clinical Phase, 2014
Figure 5-6: US - Number of No Development Reported Orphan Drug by Clinical Phase, 2014
Figure 7-1: Orphan Drug Regulatory Hurdles
Figure 8-1: Genzyme Drug Pipeline Chart
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: US Orphan Drug Market (US$ Billion), 2012-2018
Figure 3-2: US Share in Global Orphan Drug Market, 2012 & 2018
Figure 3-3: Biological & Non Biological Orphan Drug Segment (%), 2012 & 2018
Figure 3-4: Biological & Non Biological Orphan Drug Market (US$ Billion), 2012-2018
Figure 5-1: US- Orphan Drug Development by Clinical Phase (%), 2014
Figure 5-2: US - Number of Orphan Drug by Clinical Phase, 2014
Figure 5-3: US – Number of Suspended & Discontinued Number of Orphan Drug, 2014
Figure 5-4: US - Number of Discontinued Orphan Drug by Clinical Phase, 2014
Figure 5-5: US - Number of Suspended Orphan Drug by Clinical Phase, 2014
Figure 5-6: US - Number of No Development Reported Orphan Drug by Clinical Phase, 2014
Figure 7-1: Orphan Drug Regulatory Hurdles
Figure 8-1: Genzyme Drug Pipeline Chart
LIST OF TABLES
Table 2-1: Drugs Patent Expiry in 2014
Table 2-2: Drugs Patent Expiry in 2015
Table 2-3: Drugs Patent Expiry in 2016
Table 2-4: Orphan Drugs in the US and their Cost
Table 2-5: Orphan Drugs in the EU and their Cost
Table 2-6: Incentives for the Development of Orphan Drugs
Table 2-7: Orphan Drug Incentives by Country
Table 5-1: US - Orphan Drugs in Research Phase
Table 5-2: US - Orphan Drugs in Preclinical Phase
Table 5-3: US - Orphan Drugs in Clinical Phase I
Table 5-4: US - Orphan Drugs in Clinical Phase I/II
Table 5-5: US - Orphan Drugs in Clinical Phase II
Table 5-6: US - Orphan Drugs in Clinical Phase II/III
Table 5-7: US - Orphan Drugs in Clinical Phase III
Table 5-8: US - Orphan Drugs in Preregistration Phase
Table 5-9: US – Registered Orphan Drugs
Table 6-1: US – Marketed Orphan Drugs by Indication & Brand Name
Table 7-1: Orphan Drug Clinical Study Hurdles
Table 2-1: Drugs Patent Expiry in 2014
Table 2-2: Drugs Patent Expiry in 2015
Table 2-3: Drugs Patent Expiry in 2016
Table 2-4: Orphan Drugs in the US and their Cost
Table 2-5: Orphan Drugs in the EU and their Cost
Table 2-6: Incentives for the Development of Orphan Drugs
Table 2-7: Orphan Drug Incentives by Country
Table 5-1: US - Orphan Drugs in Research Phase
Table 5-2: US - Orphan Drugs in Preclinical Phase
Table 5-3: US - Orphan Drugs in Clinical Phase I
Table 5-4: US - Orphan Drugs in Clinical Phase I/II
Table 5-5: US - Orphan Drugs in Clinical Phase II
Table 5-6: US - Orphan Drugs in Clinical Phase II/III
Table 5-7: US - Orphan Drugs in Clinical Phase III
Table 5-8: US - Orphan Drugs in Preregistration Phase
Table 5-9: US – Registered Orphan Drugs
Table 6-1: US – Marketed Orphan Drugs by Indication & Brand Name
Table 7-1: Orphan Drug Clinical Study Hurdles
