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How to successfully launch a rare disease treatment in a patient-centric world

  • By Abhyodaya Siddhartha
  • •  Dec 14, 2021

The introduction of all drugs is complicated, but these are especially the introduction of treatments for orphan diseases. In our experience, companies that bring rare disease treatments to market need to excel in four areas.

Patients with rare illnesses often find that there is no cure for their condition. Of the 7,000 known diseases in this category, 95% (so-called orphan diseases) do not receive a single FDA-approved medication. Rare diseases, by definition, affect a small number of patients and do not attract historically  significant drug investments. 

But recently, major pharmaceutical companies are paying more attention to rare diseases due to government incentives and the increased likelihood that treatment of often life-threatening or  debilitating illnesses will be effective for both drugs and  patients. I started to turn it. The government incentives sidebar lists several measures taken by the government and regulators  to promote innovation in diseases with low prevalence and high unmet needs.

As a result, 45 new treatments for rare diseases were approved by the FDA's Center for Drug Evaluation and Research in 2015. This is well above the average of 28 approved over the last nine years. 10.5% 4 per year predicts that 19% of global prescription drug sales will be $ 176 billion by 2020 patient benefits. 

However, the way pharmaceutical companies bring their products to market with new orphan drugs is essential to their success. This is because the launch of all drugs is complex, but the launch of treatments for rare diseases is also complex. When a large company introduces such a treatment, it is usually the first time it enters the treatment area. As a result, they may lack both expertise in the disease and a deep understanding of the health ecosystem and the patient's experience with the disease. This condition is rare, so launch teams have few, if any, analogs  to learn. Also, the company is likely to have purchased the drug from a small biotechnology company  late in development, so it may not have enough time to prepare for  launch.


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