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ISSN 2063-5346
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“Navigating the Roadblocks: Insights and Advances in Orphan Drug Access and Affordability”

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Ashutosh Pandey, Chandan.C, Jeyaprakash MR
» doi: 10.48047/ecb/2023.12.4.194

Abstract

Orphan chemotherapeutics diseases that become uncommon in the community to cure them. A doctor might see one instance per year or worse due to the disease's rarity and a relative handful of individuals suffering. There are numerous medications used to treat rare diseases, including Tafinlar, Photobarr, Somavert, Onsenal, etc. These times, the pharmaceutical sector's rise of orphan drugs is more rapid, and it has great potential. For the creation of one particular new medicine, pharmaceutical corporations invest a significant sum. The nation as a whole can perceive the headway. The United States and Europe both observe Rare Disease Day on February 28 each year. I have covered the demographic impacted by rare diseases, the 1983 Orphan Drug Act, and its different qualifications and authorizations in this appraisal. The multinational orphan medicine market, its sales promotions, sales, and price strategies have also been covered. Due to the poor trading purpose and high cost of the drug, the estimated profit for curing a rare condition is lower. In the essay, the difficulties a pharmaceutical company faced while developing an orphan drug and its potential future are covered. A list of medications has also been provided, together with information on each one's generic name, level of endorsement, dosage, and manufacturer. Further details include the specific treatment, its therapeutic focus, mechanism of operation and layout.

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