Pharmaceutical companies have been increasingly focusing on developing treatments for rare diseases in recent years. This is largely due to technological advancements that make identifying the genetic causes of rare diseases easier. Because of their high prices, orphan medications have a lot of potential to generate income. By 2022, the global orphan medication industry is expected to have grown to $209 billion, accounting for 21.4 percent of all branded prescription drug sales. The high pricing connected with some orphan drugs have become a point of contention as the number of approved orphan drugs has grown. The high expense of drug development and recouping these costs by a smaller patient pool are the most common reasons for orphan medicine prices. We can't use cost-based pricing strategies unless we fully grasp the costs of orphan medicine research. If the patient population is limited enough, orphan medications with relatively low research costs may nonetheless require very high pricing to recoup those expenditures. Even if a more accurate cost of medication development at the drug level becomes accessible, the question of how much profit should be allowed above the cost of drug development remains. There is still a lot of work to be done in this area to better understand how to reward innovation while keeping drug expenditures in check.
Title : The foundation for rare disease and its role in the european rare disease research landscape
Daniel Scherman, Foundation for Rare Diseases, France
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