How efficient logistics can change the lives of rare disease patients

There are approximately 8000 rare diseases in the world and many of these are chronic, life-threatening conditions.¹ 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 the investment necessary for their development.

The Orphan Drug Act and similar legislations aim to progress the development of drugs for rare diseases by offering various incentives to drug manufacturers.

Despite government support, orphan drugs have been priced significantly higher than non-orphan ones owing to the inherent complexities and high costs of production.²

Yet, there are other barriers to patients receiving life-saving treatments.³ One that’s frequently overlooked is the complexity of transporting these highly sensitive drugs from the lab to the end user.

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.

Logistical barriers stand tall

Companies developing orphan drugs and other rare treatments often do so at a net financial loss.⁴ This cost does not rest solely with research and production, the safe transport and distribution of these specialised treatments is also a consideration.

One of the most significant challenges when shipping orphan drugs is their temperature sensitivity. Many of these products are biologics, gene therapies or other advanced therapies that require strict temperature control to protect their efficacy. 

Some require storage at extremely low temperatures, often below freezing, whereas others may need to be stored at controlled room temperatures or other conditions. Even a small temperature deviation during shipment can compromise the product and render the drug ineffective or even harmful to patients. 

Despite the small patient pool, orphan drugs often need to be delivered to different, sometimes hard to reach, locations around the world, adding another layer of complexity to the process.

Import and export regulations and differing international standards can create bottlenecks and delays, increasing the risk of temperature deviations if a shipment is stuck in customs.

Last mile delivery is another critical challenge when ensuring patients receive their treatments safely and efficiently. Unlike many medications, orphan drugs often require direct delivery to patients’ homes or specialised medical facilities, introducing additional requirements to consider.

Challenges such as geographical remoteness, limitations in infrastructure and the need for personalised handling further add to the complexities of last mile delivery.

All these costs and logistical challenges only push the cost of treatment up higher, creating significant barriers to affordable treatments and potentially pricing-out patients who need the treatment most.⁵ 

Finding the right balance

For the pharmaceutical industry, efficient logistics isn’t just about cost savings; it’s about ensuring that life-saving medications reach the right patients, at the right time, in the right condition. Finding the optimal shipping solution involves balancing performance, environmental impact, service level and cost. 

It’s crucial to choose packaging that’s designed to maintain a consistent temperature range throughout the shipping process.

It’s vital that the optimal packaging for the shipment mitigates the risk of temperature deviations, prevents potential product loss and any consequential increase in transport costs and drug usability at the destination.
 
However, there are strategies to mitigate risk, such as “smart” secondary packing solutions with real-time temperature and location monitoring that send alerts in real-time if an unexpected event occurs during transit.

This facilitates the implementation of corrective action that prevents costly product spoilage and reshipping, saving money in the long run. 

Both active and passive containers have their role to play, but the best choice depends on varying factors including product temperature requirements, tradeline complexity, Total Landed Cost (TLC) and, ultimately, the level of risk you are willing to take with your medicine.

Because orphan drugs are manufactured in smaller quantities, they often share cargo space with other pharmaceutical products, potentially increasing the risk of exposure to unsuitable conditions.

To mitigate this risk, manufacturers can prioritise collaboration and co-ordination with logistics partners to optimise transportation. By leveraging shared logistics networks and implementing just-in-time inventory strategies, distribution processes can be streamlined while also minimising costs and maximising efficiency.

The bottom line of efficient logistics

The journey of an orphan drug, from research lab to patient delivery, is a complex process and often an expensive one. However, the cost of rare disease drugs isn’t just in development, it’s in delivery. Inefficient logistics can eat up resources and can even lead to patients not getting the treatment they so desperately need.

Yet, the barriers to shipping rare disease treatments can be negated. This means that more patients have access to the drugs they need, regardless of how rare their condition is. After all, it’s not just about saving money, it’s about saving lives.

References

1. www.statista.com/topics/2493/orphan-drugs/#topicOverview.
2. www.ncbi.nlm.nih.gov/pmc/articles/PMC9957503/.
3. www.pharmaceutical-technology.com/features/navigating-distribution-challenges-for-rare-disease-drugs/.
4. www.envirotainer.com/resources/industry-insights/2023/orphan-drugs/.
5. www.ncbi.nlm.nih.gov/pmc/articles/PMC9957503/.