关键词：FDA；孤儿药计划；药品；生物制品；安全有效；治疗；罕见疾病/障碍
摘 要：According to the FDA, the orphan drug designation program provides orphan designation to drugs and biologics that are used for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect less than 200,000 people in the US or that affect above 200,000 people but are not estimated to recover the costs of developing and marketing a treatment drug. Also, orphan drugs are developed by the pharmaceutical industry to serve the public health need. They are granted orphan drug status by the regulatory agencies to accelerate the approval process.
目 录：

PART 01: Executive summary

Highlights

PART 02: Scope of the report

Top-vendor offerings

PART 03: Market research methodology

Research methodology

Economic indicators

PART 04: Introduction

Key market highlights

PART 05: Overview

Orphan medicines legislation

Orphan medicines centralized procedure in US/EU

Product registration process for orphan drugs in Japan

Orphan drug designations and approvals

Annual cost of orphan drugs

R&D costs for orphan and non-orphan drugs

PART 06: Pipeline snapshot

PART 07: Market landscape

Market overview

Market size and forecast

Five forces analysis

PART 08: Market segmentation by product

Global biologic orphan drugs market

Global non-biologic orphan drugs market

PART 09: Geographical segmentation

Orphan drugs market in Americas

Orphan drugs market in EMEA

Orphan drugs market in APAC

PART 10: Market drivers

Shorter development timelines

Assistance from regulatory bodies

Incentives for developing orphan drugs

High return on investment

Exemption from pricing constraints

PART 11: Impact of drivers

PART 12: Market challenges

High cost of drug development

Decreased investment in marketing

Complications in conducting clinical trials

Unknown pathophysiology

Adverse policies of FDA

PART 13: Impact of drivers and challenges

PART 14: Market trends

Increased focus on biological drugs

Growing awareness of orphan drugs

Channel marketing strategy

Expansion of business

Entry into untapped market

PART 15: Vendor landscape

Competitive scenario

Major vendors 2014

Worldwide orphan drug sales revenue: Top

companies 2013

Top 10 orphan drugs in US 2014

Other prominent vendors

PART 16: Key vendor analysis

BMS

Celgene

Novartis

Pfizer

PART 17: Appendix

List of abbreviations

PART 18: Explore Technavio