US Orphan Drug Market Outlook 2018

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.

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 US Orphan Drug Market Outlook 2018

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

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Table of Contents

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

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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)

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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

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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 Costs

Table 2-5: Orphan Drugs in the EU and their Costs

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

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