Dr. Danial Arkwell, Head of Global Key Accounts, Pharma at Envirotainer highlights the vital role of efficient logistics in mitigating risk and finding the right solutions to get orphan drugs to patients in desperate need.
There are approximately eight thousand rare diseases in the world and many of these are chronic, life-threatening conditions. To fight these elusive illnesses, the medical community focuses on developing specialised medications known as orphan drugs. Originating from the United States Orphan Drug Act (ODA) in 1983, the term “Orphan Drug” reflects the lack of attention and investment necessary for their development.
The diseases targeted by orphan drugs often have low prevalence rates, making them economically unattractive to develop treatments for, due to the limited potential market. The result is that many of these diseases are being historically neglected as a result of the perceived low profitability.
The Orphan Drug Act and similar legislation aim to progress the development of drugs for rare diseases by offering various incentives to drug manufacturers. This includes tax credits for clinical research costs, seven years of market exclusivity upon approval, and assistance with clinical trials, design and funding.
Despite government support, orphan drugs have been priced significantly higher than non-orphan drugs, owing to the inherent complexities and high costs of production. The significant upfront investment required to bring these much-needed treatments to market not only slows down their development but also contributes to incredibly high prices for patients, potentially putting them out of reach for many.
Yet, there are other barriers to patients receiving life-saving treatments. One that’s frequently overlooked is the complexity of transporting these highly sensitive drugs from the lab to the end patient. If manufacturers cannot ensure safe, cost-effective delivery, then access to these rare treatments will remain out of reach for many patients. Efficient logistics must take centre stage.
