Executive Summary

According to Azoth Analytics research report, the Global Rare Disease Drugs was valued at USD 147.12 Billion in the year 2021. The increasing focus on research activities and the development of novel therapeutic drugs has facilitated the growth of the Rare Disease Drugs market. Additionally, growing product developments is leading to the implementation of technologically driven innovative devices in the diagnosis of rare genetic disorders.

Moreover, favourable government policies aiming at creating awareness pertaining to the Rare Disease treatment are leading to increased adoption of effective treatments of rare disorders. However, there are several factors hindering the efficient and effective clinical trials of drug development of rare disorders including low patient numbers, limited understanding of pathology and progression, and lack of established endpoints. Therefore, several regulatory bodies are engaged in implementing regulatory standards for drug approval for Rare diseases to ensure patient safety and efficacy of drugs.

Furthermore, improved detection and increased rates of diagnosis of Rare diseases are likely to contribute to rising costs. While the humanitarian benefits of Rare Disease therapies are indisputable, concerns regarding high treatment costs play a meaningful role in how payers administer and manage healthcare benefits that ultimately dictate patient access to orphan drugs. Moreover, the biological drugs are used for treating Rare diseases such as cancer with fewer side effects that have a high prevalence rate in the developed world.

Scope of the Report

The report presents the analysis of the Rare Disease Drugs market for the historical period of 2017-2021 and the forecast period of 2022-2027.
The report analyses the Rare Disease Drugs Market by Value (USD Billion).
The report analyses the Rare Disease Drugs Market by Drug Type (Biologics, Non-Biologics).
The report analyses the Rare Disease Drugs Market by Therapeutic Area (Infectious Disease, Oncology, Neurology, Metabolic, Others).
The report analyses the Rare Disease Drugs Market by Patients (Pediatric, Adults).
The report analyses the Rare Disease Drugs Market by Route of Administration (Oral, Injectable).
The report analyses the Rare Disease Drugs Market by Distribution Channel (Hospital & Retail Pharmacies, Online Pharmacies).
The Global Rare Disease Drugs Market has been analysed By Region (North America, Europe, Asia Pacific and LAMEA).
The Global Rare Disease Drugs Market has been analysed By Country (United States, Canada, Germany, United Kingdom, France, Italy, China, Japan, India, South Korea).
The attractiveness of the market has been presented by region, by Drug Type, by Therapeutic area, by patients, by Route of Administration and by Distribution Channel
Also, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report.
The report tracks competitive developments, strategies, mergers and acquisitions and new product development.
The companies analysed in the report include Takeda Pharmaceutical Company Limited, Sanofi, Merck KGaA, F. Hoffman-La Roche Ltd., Pfizer, Novartis AG, Bayer Group, Biogen, GlaxoSmithKline, AbbVie Inc.
Key Target Audience
Rare Disease Drugs Manufacturers
Healthcare and Pharmaceutical Companies
Consulting and Advisory Firms
Government and Policy Makers
Regulatory Authorities