Rare diseases are mostly serious, chronic and life-threatening, associated with both psychological as well as financial burdens. In addition, only a few of them have effective drug treatment available. The European Union (EU) definition of a rare disease is one that affects fewer than 5 in 10,000 people. These affect 6–8% (or about 30 million people) out of the 508 million population of EU countries. So, pharmaceutical companies refrain from investing in them.
This the reason they are called orphan drugs as they are not commercially viable. When medicine is granted an orphan designation, various incentives are provided to the sponsor of the clinical trial.
Treatment for Rare Diseases is still a major obstacle which we need to overcome. Out of 6000 known rare diseases, cures for only 200 are known.
Fortunately, in recent years, pharmaceutical companies have made advancements in developing drugs for Rare Diseases. The reason being improvements in technology to identify the causes behind them. The financial aid provided for their clinical trials which has resulted in more than 400 drugs getting approved. Also, the global orphan drug market is estimated to reach USD 209 billion by 2022 accounting for 21.4% of total branded prescription drug sales.
But their high prices confine the patients’ access and make it impossible to bear by any health-care system as well.
For instance, Nusinersen, an antisense oligonucleotide, is given via intrathecal injection, at a cost of USD 125000 per injection. And the whole treatment costs a lot more than this. Imiglucerase, an enzyme replacement therapy to treat Gaucher’s disease, might cost as much as USD 400,000 per year for an adult patient. Eculizumabt, which treats Paroxysmal Nocturnal Hemoglobinuria, costs up to USD 500,000 per patient per year.
The monopoly of pharmaceutical companies in any specific drug making, patent rights, sparse estimates of the cost of development, less knowledge of the drug are some of the reasons behind higher prices of the orphan drug.
With the high unmet drug needs and the absence of substitutes, the cost factor becomes more of a bigger problem.
Although the policymakers have started paying attention to the unaffordable prices of these drugs but there is still more to be implemented in this area. The components on which the cost is based and made available to patients is one thing to be kept in mind. Broad and sustainable access to DRUGS is important. A global and cohesive strategy is needed while deciding the pricing of treatment. Drugs’ prices need to be regulated carefully.
Because in the end providing affordable health care should be the priority of the companies and policymakers.