The orphan drugs are developed to treat orphan or rare diseases. These drugs are called “orphan” because its market is very limited; few pharmaceutical companies have attention to develop such products only for public health, not for economic reasons. There are many patients lack a proper diagnosis and most are left without effective treatment, so research for rare diseases is urgently needed. For this, many research centers, pharmaceutical companies, and other organisations are:

  • developing more possibilities to diagnose rare diseases
  • highlighting the main resources to accelerate the research and development of treatments for rare diseases by 2025

Current Market Scenario and Forecast of Orphan Drugs

Currently, it is estimated that around 7000 different types of rare diseases are present across the world and around 350 million people suffer from a rare disease. The market of orphan drugs is showing persistent growth due to increasing demand in the healthcare sector. The sales of the orphan drug are expected to reach $136 billion in 2019, which is increased by 3.7% from the 2018 sale outcome. As per growing awareness, the incidence of rare diseases, improved incentives by regulatory authorities and increase demand for orphan drugs, it is estimated that orphan drug sales further will be increased by 12% per year and reach to 242 billion dollars by 2024. In this way, the development of the orphan drug market is expected to be almost double the overall prescription drug market by 2024.

Key Market Drivers of Orphan Drugs

In the past years, research on orphan drugs was less than 10% because pharmaceutical companies were not focusing on the development of orphan drugs as they provide low profitability due to the rarity of the diseases. For now, the new business model of niche busters could offer an integrated healthcare solution that enables pharmaceutical companies to develop a new area of therapeutics, treatment, diagnosis, and patient support. Including this, the following key drivers also play a significant role in increasing the growth of orphan drugs:

  • Market exclusivity for the orphan drug developer
  • Focus on awareness program for orphan drug
  • Rising prevalence of rare diseases
  • Research grants and government policies provided by the organization to the academics, research centers, institutes, and pharmaceutical industries for the development of rare drugs

Orphan Drugs Regulations

Orphan drugs follow the same regulatory development path as other medicinal products follow, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety, and efficacy. But orphan drug gets accelerated approval from the health authority in phase 2 because fewer patients affected with a rare disease which unable to meet the target patient number of phase 3. With this, orphan drug regulation acts exist across the world for the development of drugs, which are discussed below:

  • Orphan Drug Regulation in the United States: The Orphan Drug Act (ODA) first time passed in 1983 in the United States (US) for the development of orphan drugs. Process of getting approval is same as follow for other drugs, but under this law, drug developer gets orphan drug designation via ODA if the prevalence of the disease is less than 200,000, then drug developer get benefits in the form of tax credits, an accelerated approval process, and seven years exclusive marketing.
  • Orphan Drug Regulation in European Countries: Regulation (EC) No. 141/2000 act established in the European Union (EU) in 1999 for orphan drug development. If the prevalence of the disease is ≤5 people in 10,000, then this act provides incentives to drug developers, completely reduced fees for follow-up and protocol assistance, reduced fee for marketing approval up to 50%, and provides market exclusivity for 10 years.
  • Orphan Drug Regulation in Japan: Japan’s Ministry of Labour and Welfare (MHLW) established this orphan drug regulation act in 1993. As per this act, if the targeted disease is not treated previously and prevalence of the is ≤ 50,000, then then this act provides tax exemptions up to 6% on research and development (R&D), reduced drug development cost by 50%, expedited approval, and 10 years market exclusivity to the drug developer.
  • Orphan Drug Regulation in Other Countries: Orphan drug act adopted in Australia, Taiwan, and South Korea successfully promoted R&D investments and subsidies to develop new pharmaceutical products for the treatment of rare diseases.

Challenges Face by Orphan Drug Developer

Besides the benefits of orphan drug regulation acts, still, orphan drug developer faces many challenges for the development of medicines to treat rare diseases. The main challenges are poor understanding of rare diseases due to lack of prior clinical studies to create a template for study execution, the clinical management of patients, to find correct individuals, and to retain the target patients for a long time is very hard in the orphan drug clinical trials. These challenges can be overcome if drug developer adopts emerging methods like digital health tools, the social media network, and smartphones to search, recruit, attract, and engage the patients in the study for the required time.


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