Global Rare Disease Drug Market Size Clinical Trials Insight 2028

Global Rare Disease Drugs Clinical Trials Insight Report Highlights:

  • Clinical Insight On More Than 400 Marketed Orphan Drugs
  • Clinical Insight On More Than 1300 Orphan Drugs in Clinical Trials
  • Orphan Designated Drug Clinical Status by Indication & Country
  • Global Market Opportunity More Than USD 350 Billion
  • Market Exclusivity & Patent Protection Criteria for Orphan Drugs
  • Global & Regional Orphan Drug Market Sales Opportunity
  • Orphan Drug Reimbursement Policy
  • Orphan Clinical Pipeline Overview Company, Drug Class, Formulation, Indication, Region, Priority Status, Patient Segment, Phase

For Report Sample Contact

Rare disease is disorder which affects a small percentage of total population. In last few years, there has been increased interest among pharmaceutical companies for the development of novel orphan drugs against rare diseases. Although there are more than 7,000 known rare diseases, only 5% of these diseases have FDA approved treatment. Currently, more than 500 orphan drugs are available in market which is indicated for the management of various cancers, Pompe disease, fabry disease, cystic fibrosis, spinal muscular atrophy, and several others.

The development of orphan drug is considered a profitable business to the pharmaceutical companies which is mainly attributed to the large benefits associated with them. The clinical trials for orphan drug medications are significantly less expensive in comparison to other diseases as trial sizes are naturally smaller than for more common diseases with larger patient population. Further, many regulatory agencies across the world also offer several incentives for the development of orphan drugs. These incentives include financial benefits, faster approval, less stringent clinical data requirements, patent exclusivity, and many more. Several countries including US, Europe, Japan, Australia, South Korea, India, and other offer these incentives which drive the development of orphan drugs in the region.

In contrast to non-rare conditions, rare diseases are associated with several challenges which make their management more difficult. The major barrier to pharmaceutical companies is the small patient pool, which lacks the epidemiological data available and making research both difficult and costly. Furthermore, launching an orphan drug requires additional planning which requires forward thinking, thoughtfulness, progressive ideation, a patient-centric focus, and consideration of every detail. In addition to this, the affordability of orphan drugs is also a major challenge for payers and is thus a strong driver of tensions between the stakeholders.

Although there are several obstacles, but it is believed that orphan drugs provides a million dollar opportunity to the pharmaceutical companies. The advancement in the field of genomics and biotechnology is increasing the knowledge about the progression of diseases, which further boost the research and development sector. The major key players in the companies include AbbVie, Roche, Amgen, Novartis, Eli Lilly, Sanofi, Pfizer, and others. These players are implementing several strategic acquisitions, mergers, collaborations and partnerships in order to expand customer base and strengthen their industrial position. The investment by these players in recent years has created opportunities for the global expansion of the industry.

US orphan drug market hold the maximum share in the market during the forecast period on account of the improved economy and rising healthcare expenditure resulting in increasing the development of research institutions in the country. Growing number of government initiatives in the region is also contributing to the market growth trends in the country. The orphan drugs act under the US legislation allows manufacturers to request FDA to grant orphan drugs status to drugs in order to treat a rare disease or condition. Hence, such initiatives have the potential to expand the industry in the country.

Japan dominated the rare disease market in Asia-Pacific region. The highest market size is owing to the increase in biomedical and regenerative medicine research in the country. Japanese government has established support systems and special regulatory authorities to augment the development of novel drugs for rare disease treatment. The support system measure in Japan includes grant-in-aid for research programs along with price control policies negotiated by Japanese National Health Insurance (NIH) and pharmaceutical companies, and medical reimbursement for 56 diseases. Apart from this, India is seeing an increased focus on developing orphan drugs which is mainly due to high burden of rare diseases, which is driven by large population.


Neeraj Chawla

Research Head

Kuick Research


Back to news