Rare Disease Drug Market Sales Clinical Trials Drug Price Insight


Global Rare Disease Drug Market Opportunity More Than USD 350 Million With More Than 1100 Drugs in Clinical Trials Says Kuick Research

Delhi, June 30, 2023 (GLOBE NEWSWIRE) — Global Orphan Drugs Market & Clinical Trials Insight Report 2028 Highlights:

  • Clinical Insight On More Than 600 Marketed Orphan Drugs
  • Clinical Insight On More Than 1100 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


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The rare disease drug market applies to the pharmaceutical industry segment devoted to the development and commercialization of treatments for rare diseases. Rare diseases, sometimes known as orphan diseases, are diseases that affect a limited number of persons in the population, commonly less than 200,000 in the United States. Though rare diseases do not have a substantial influence on the healthcare industry individually, they impose a significant burden on the industry as a whole due to rising prevalence and mortality rates, as well as a lack of adequate treatment for many diseases. Only a small percentage of the diagnosed rare diseases have therapeutic interventions, and many of these existing treatments focus on controlling symptoms rather than considering underlying causes. However, as science advances, these diseases are becoming more visible due to their sluggish but growing patient base.

On the same note, orphan drugs are treatments that are developed to meet the unmet medical requirements of patients suffering from rare diseases such as pancreatic cancer, or less common indications and patient subsets of more common diseases such as glioblastoma multiforme. Many rare diseases are hereditary in nature, and individuals with these conditions frequently suffer from considerable morbidity and poor quality of life. The development of therapies for rare diseases attempts to address these unmet medical requirements and provide patients with therapeutic choices. Furthermore, the rare disease drugs industry has grown significantly in recent years. This can be associated with a number of things, including better diagnostic tools, increased awareness of and understanding of rare diseases, and improvements in research in science and medicine.

Regulation-related subsidies for orphan drug development are also offered by regulatory bodies like the EMA and US FDA. Initiatives have been put in place to promote the development of medications for rare diseases. These consist include accelerated approval procedures, increased market exclusivity, and monetary rewards to boost R&D initiatives. Drug producers might benefit from market exclusivity for a predetermined duration, tax credits, and fee exemptions when regulatory agencies designate their products as orphan drugs. Drug development for rare diseases has received financial support from pharmaceutical corporations thanks to these incentives. Over 140 drugs and compounds were granted orphan drug designation in the first six months of 2023, with close to 40 receiving marketing authorization. Over 400 drugs and compounds were designated as orphan in 2022, with a little more than 70 approved for their orphan indications. Both figures have risen in recent years, indicating a growing interest in these untapped indications in the pharmaceutical sector.

However, treating rare diseases with these medications sometimes comes with challenges, one of which is reimbursement. Because of the limited number of patients and the costs resulting from research, development, and manufacturing, rare disease drugs are expensive. These high prices can make patient access and reimbursement difficult, resulting in continuing discussions and arguments about pricing methods and affordability. To ensure patient access to these medications, the US government provides reimbursement support for orphan drugs through a variety of programs and initiatives. Medicare, Medicaid, and the 340B Drug Pricing Program are among these programs. Similarly, commercial health insurance firms play a major role in the region’s drug reimbursement sector.

Collaborations and partnerships between pharmaceutical corporations, academic institutions, patient support groups, and government organizations are widespread in the rare disease drug market due to the specialized nature of rare diseases and the tiny patient populations. These agreements aid in the advancement of research, development, as well as accessibility to drugs. For example, Novartis AG agreed to pay up to US$ 3.5 billion for Seattle-based Chinook Therapeutics in order to add two promising therapies in advanced testing for a rare kidney condition. Similarly, Amgen is attempting to acquire Horizon Therapeutics for approximately US$ 28 billion in its largest-ever deal.

The market for rare disease drugs represents a substantial opportunity and medical need. The discovery of medications for rare diseases has been accelerated by breakthroughs in genomics, personalized medicine, and targeted therapy. Despite the difficulties, the development of orphan medications has considerably improved the lives of those suffering from rare diseases. These therapies offer targeted treatments, disease management techniques, and potential solutions for diseases that were previously ignored or had no therapeutic alternatives. Continuous research, collaboration, and regulatory backing are required to satisfy the requirements of patients with rare diseases. The market is likely to expand further as more research discovers the underlying causes of rare diseases and novel therapeutics to treat these conditions are produced.

CONTACT: Neeraj Chawla Kuick research Research Head [email protected] https://www.kuickresearch.com 

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