Assessment of Regulatory Strategies for Orphan Drug Development and Approval Process in USA & EU

Orphan medicines are pharmaceutical drugs or vaccines supposed to deal with, preventing or diagnose of a rare disease (viz., ailment, Huntington's disease, syndrome, etc.). The meaning of rare sicknesses varies throughout some jurisdictions, however, generally contemplates the ailment of occurrence, severity, and life of alternate healing options. An uncommon ailment isn't worldwide and depends on the rules and guidelines officially adopted and approved via each region or U.S.A. The Orphan Drug Act, 1983 (ODA) has been recognized and adopted in numerous nations, international (United States of America (USA) and European Union (EU).) in the preceding 35 years, and has effectively advanced R and D ventures to widen new pharmaceuticals for the remedy of rare sickness. The rate of occurrences of such diseases were outpaced at an extra pace than the speed with which medicines are researched and developed to treat rare diseases. One of the main reasons is that the pharmaceutical industry is not interested in researching the enhancement of orphan medicines since they no longer have a significant large market. Despite the multiple incentives provided by the orphan drug act, this is the current reality. However, in this article, we've tried to concentrate on the current regulatory framework, current concepts of rare disease, regulatory challenges for rare disease drug development, and orphan drug approval in the United States & the European Union.

Dercum’s disease: estimating the prevalence of a rare painful loose connective tissue disease

Background: In the USA, the Orphan Drug Act of 1983 defines a rare disease as affecting under 200,000 individuals. Dercum’s disease (DD) is a loose connective (adipose) tissue disease characterized by painful lipomas. While considered a rare disease, the prevalence of DD has not been systematically assessed previously. The objective of this paper is to estimate the prevalence of DD to determine if it is rare or not. Results: Estimates of prevalence of DD using PubMed, the UK Biobank, the US Agency for Health Research and Quality Healthcare Cost and Utilization, physician practices, social media forums and internet searches found the prevalence of DD to be less than 200,000 individuals in the US. These prevalence likely overestimate the disease; however, underestimation may also occur because DD is not well known and may be misdiagnosed. Conclusion: DD meets requirements of the Orphan Drug Act to be classified as a rare disease. Further research should focus on representative population samples in the USA to better estimate the prevalence of DD. Estimating the prevalence is an important first step to increase recognition, research efforts and patient care for people living with DD.

Dercum’s Disease: Estimating the prevalence of a rare painful loose connective tissue disease

Background In the United States (US), the Orphan Drug Act of 1983 defines a rare disease as affecting less than 200,000 individuals. In Europe, a rare disease or disorder is defined as affecting 5 individuals in 10,000 which the European commission reports to be approximately 246,000 individuals. Dercum’s disease, also known as Adiposis Dolorosa, is a rare loose connective (fat) tissue disease that is characterized by painful lipomas (fatty masses). Orphanet, the National Organization for Rare Disease (NORD) and the United States National Institutes of Health (NIH) have classified Dercum’s disease as a rare disorder. The prevalence of Dercum’s disease is not well studied or understood. The objective of this paper is to estimate the prevalence of Dercum’s disease, whether it is in fact a rare disease, and increase awareness for this painful disease. Results An in-depth literature review was conducted in PubMed, the UK Biobank, the U.S. Agency for Health Research and Quality Healthcare Cost and Utilization, by incidence of disease in medical practice, in social media forums and by internet search in order to understand, estimate and determine the prevalence of Dercum’s disease. The prevalence of Dercum’s disease was found to range from 150 to 150,000 in the US population. Conclusion The data collected in this paper meet the requirements of the Orphan Drug Act which requires 200,000 individuals or less to be affected by a disorder for it to be classified as rare. These conclusions may apply to Europe as supporting data was utilized from both Sweden and the UK. Further research must be conducted to better understand subclassifications of this rare disorder.

The Orphan Drug Act: An Appropriate Approval Pathway for Treatments of Rare Diseases?

The Orphan Drug Act provides financial incentives to pharmaceutical manufacturers to develop treatments for rare diseases affecting limited patient populations. Since passage of the Act in 1983, the U.S. Food and Drug Administration (FDA) has approved more than 600 orphan drug indications from greater than 450 distinct drug products. The annual number of orphan drug designation approvals has increased significantly in the recent past with much of this increase driven by approval of secondary indications for previously approved treatments. This recent increase has led to concerns regarding the Act as some pharmaceutical manufacturers have reaped outsized financial benefits while avoiding the regulatory requirements and costs associated with nonorphan drug development.