To incentivize pharmaceutical companies to address the unmet needs of patients with rare diseases, the Orphan Drug Act of 1983 was passed. Since the passage of this Act, the number of orphan drugs approved in the U.S. has risen dramatically. In line with this increased resource, further consideration needs to be taken on how to safely transport and distribute these specialized treatments to the patients who need them.
Handling Requirements for Rare Disease and Orphan Drugs: Understanding the Complexities
Even though the number of potential patients may be relatively small, orphan drugs often need to be delivered to more locations around the world than non-orphan drugs. In addition to this, when orphan drugs are developed as personalized medicines, the transportation complexities increase. Some of these complexities include:
Temperature Sensitivity: One of the most significant challenges in shipping orphan drugs is their temperature sensitivity. Many of these products are biologics, gene therapies, or other advanced therapies that require precise temperature control to protect their efficacy. Even a small temperature deviation during the shipment can cause degradation to the product – potentially making it ineffective or even harmful to patients.
Cold Chain Logistics: A strong cold chain is central to the transportation of many orphan drugs. This requires effective transportation solutions, experienced partners, and appropriately trained stakeholders at every stage in the transportation process.
Temperature Extremes: Some orphan drugs require storage at extremely low temperatures, often below freezing. Others may need to be stored at controlled room temperatures or slightly cooler conditions. Any deviation from these requirements can compromise the drug's quality and efficacy.
Global Accessibility: Transporting these drugs across international boundaries can add additional layers of complexity to the process. Customs procedures, import/export regulations, and differing international standards can create bottlenecks and delays in the shipping process.
Selecting a Packaging Solution: Finding the right balance
Ensuring that the right dose reaches the right patient at the right time and under the right conditions can be challenging. To find the most effective shipping solution for such precious cargo, you must not only understand the requirements of the product being shipped, but also assess any solutions based on both performance and the level of service you should expect from the provider. This includes:
Precise Temperature Control: For drugs that demand strict temperature control, always select a packaging solution that is designed to maintain a consistent temperature range throughout the shipping process – regardless of even the most extreme ambient conditions. Consider a provider’s past performance in terms of reliability relating to temperature excursions and how long a solution can keep a stable temperature for if there were to be any delays in the shipment.
Cold Chain Expertise: Always choose a provider that has extensive expertise in shipping temperature-sensitive products. They should understand the rigorous standards of the pharmaceutical industry and should be able to offer you advice tailored to your specific product and operations. By leveraging their cold chain know-how, you can work together to ensure a successful shipment and maintain the efficacy of your product.
Real-time Monitoring and Data Logging: Solutions should be able to monitor temperature, location, and other environmental conditions in real time. This level of monitoring allows for complete visibility into the status of your product throughout the shipment. With true shipment monitoring services, alerts can be sent if an unexpected event occurs during transit, enabling swift corrective action. This proactive approach ensures that patients receive their drug in optimal condition and reduces the risk of product waste.
Regulatory Compliance: When qualifying a new solution, make sure that your supplier can provide you with detailed documentation and qualification support. This can help you to streamline the qualification process and, in many cases, help you to get the product to the patient more quickly.
Global Reach: Ensure that your solution provider can offer you global support and has a good network around the world. This will enable efficient and reliable transportation to various regions, helping to address the global accessibility challenge. A strong presence in key pharmaceutical hubs ensures that temperature-controlled shipments can be seamlessly managed across international borders.
The journey of an orphan drug from research lab to patient delivery is a complex process. The challenges of storage, handling, and regulatory compliance, can hinder the shipping of these life-changing therapies and if done incorrectly, can ultimately affect patient safety. However, with the right packaging solution, ongoing collaboration, and a patient-centric approach, the barriers to shipping rare disease treatments can be overcome, ensuring that every patient, regardless of how rare their condition, has access to the therapies they desperately need.
Envirotainer, a leading provider of temperature-controlled packaging solutions, plays a crucial role in addressing the complex challenges associated with shipping and handling orphan drugs. Our experienced Sales and Operations teams work directly with our customers to meticulously plan shipments, ensuring the integrity and efficacy of these critical medications throughout the supply chain.
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