Clinical supply chain teams manage some typical challenges almost every day of their work in handling clinical trial logistics for local or global clinical trials. These include those related to regulatory compliance, temperature control (or, excursions), labeling, actual shipment, or last-mile delivery.
These challenges are often compounded multi-fold when clinical trials are related to a field with special considerations, for example, in clinical trials for rare diseases.
Clinical trials for rare diseases – what’s so special?
Rare diseases are a highly active field currently in the clinical development industry globally. There are more than 7,000 rare diseases recognized worldwide and the number could vary across countries.
Unsurprisingly, there is a significant increase in the number of orphan drugs in clinical trials worldwide.
Read Rare Diseases: Clinical Trials & Drug Development for more information on clinical trials for rare diseases
Reasons include mainly unprecedented supports from governments across the globe. These supports encourage researchers to undertake clinical development of orphan drugs despite some extreme challenges faced by them.
Challenges in clinical trials for rare diseases include
- Finding patients from limited availability worldwide
- Patient and healthcare worker education
- Managing trials with young patients (mostly children)
- Limited drug supply
- Demanding storage and shipment conditions for the drugs
..and many others
Obviously, these changes percolate to all sections of a clinical trial, including clinical supply chain management.
Challenges in clinical supply chain management for rare disease clinical trials
- Patients are ‘rare’ or limited in numbers
- Mostly pediatric population
- Lack of awareness about rare disease
- Inexperienced clinical trial sites
- Limited availability of drug under investigation
- High customization required
It’s confounding to see how these challenges manifest into practical problems in clinical supply chain management for rare disease clinical trials.
For example, a limited number of available patients may translate into more challenges in logistics, storage, and repackaging. Since it may take years to find patients and close out a study, it makes shelf life and repackaging an ongoing consideration that must be addressed, as well as retention.
Also, with challenging patient enrollment, clinical trials may require broader geography, which introduces other logistics challenges e.g. product delivery to remote locations, the longer distance for patients to travel, etc.
Recent advances in the clinical trial methodology have led to the greater adaptation of remote clinical trials, including virtual clinical trials or hybrid clinical trials. Unlike traditional clinical trials, which require frequent patient visits to a central investigation site, remote clinical trials run using a virtual/hybrid model based in the patient’s home. This enables patients with mobility issues — such as the elderly or disabled — or patients who live in remote areas with limited transportation options to participate in the trial. 
Clinical supply chain teams have to deal with different challenges in direct to patient logistics. These entail significant customization of supply methodology, labeling, technology adaptations, and other considerations.
When working with remote and diverse locations, it’s possible that they may not have the required infrastructure both in terms of facilities and prior research skills, placing an increased burden on the sponsor to provide additional support and training.
As the drug is so rare and valuable, it might need to be conserved and only shipped when an eligible patient is identified, impacting storage and distribution strategy.
Special segments, e.g. Infectious disease or seasonality-driven diseases, may require a rapid response and agility in the supply chain.
How to overcome the challenges in clinical supply chain management for clinical trials in rare diseases?
- Leverage technology
- Adopt a flexible supply chain design model
- Incorporate redundancy in the plan as contingencies
- Improvise client delivery experience
- Ensure age-appropriate communications
- Engage with regulatory agencies
- Plan in advance
For clinical trials for rare diseases, it’s always important that the investigational products and biological research materials are delivered to target sites (which may be scattered and very few) and depots with determined controlled temperature ranges and within scheduled timelines.
Smart and intelligent clinical supply management teams manage this challenge with technology and flexible networks.
A robust Interactive Response Technology (IRT) or Randomization and Trial Supply Management (RTSM) system offers just the right support for this. Here is how IRT / RTSM may help.
– Staging drugs at significant depots globally for complex site initiation and randomization
– Flexibility to adapt to changes in patient population, enrollment, site location, etc.
– Management of resupply requirements, e.g. for biologics
– Supply forecasting and scenario planning for enrollment outperforming or under-performing cases
– Temperature control management
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Technology can also help improve education and support e.g. video consultations, e-Labels that access data/video via smartphones, apps that can provide translation, or drones for remote delivery, particularly with infectious diseases.
An amazing utilization of technology is in the management of direct to patient logistics, particularly in hybrid clinical trials or virtual clinical trials. With limited availability of drug supply as well as patients, technology ensures not just efficient direct to patient logistics, but also patient education, temperature tracking, and compliance.
Flexible supply chain design with small and frequent manufacturing runs can respond to unique demand requests efficiently.
Incorporating redundancy in the plan as a contingency, e.g. by sending test kits in advance, but holding the stock until patients are available, can be another smart move.
Enabling direct-to-patient shipment (to their home or care center), when the patient is unable to visit the site is a great example of improving client delivery experience.
With more than 50% of rare disease patients being children, age-appropriate communications for both the patient and their families go a long way in making clinical trials for rare diseases successful.
Another very important part is planning. Engaging regulatory agencies in advance helps a lot in preventing many unexpected situations.
With these considerations in place, one can plan for effective and successful clinical supply chain management in clinical trials for rare diseases. However, if you’re still up against challenges, do not hesitate to seek help from the experts.
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