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The logistics of orphan drugs

Managing a rare disease global access programme, the goal is to minimise risk and get the drug to the patient on time

Orphan drugs and rare diseases

Close your eyes and put a pin on a map of the world. That location might be where a patient is in urgent need of a new therapeutic option. Depending on where your pin landed, it can be a significant challenge to ensure that a drug can get to the patient in time.

When patients with an unmet medical need are unable to access a drug via the traditional clinical trial or commercial framework, a global access programme can be an effective way of ensuring the much needed medicine reaches the patient. These programmes facilitate access to medicines that are in clinical development, are unlicensed in a specific market but licensed elsewhere, or are in the marketing authorisation application (MAA) process. Governments around the world have established regulations enabling this form of access and as a result, demand may come from the most remote regions of the world with little, if any, advanced notice and a limited amount of time for the pharmaceutical company to respond.

For those pharmaceutical companies more used to shipping bulk quantities of drugs to a wholesaler or a limited number of clinical sites, a shipment earmarked for an individual patient presents a unique challenge and the requirement for specialist expertise and skills.

Critical success factors

From a logistical standpoint, several factors are critical to success when managing the logistics of a global access programme: security, timing, individuality and an end-to-end view.

Security: In a mass market situation with bulk shipments, the supply chain is fairly predictable; consolidated shipments routinely go to the same wholesalers and to major population centres. In many cases, shipments are made at ambient temperature and are not under urgent time pressures.

With global access programmes, shipments are made to individual patients via their physicians and local pharmacies; as a result, the supply chain becomes longer and typically requires more steps. In many cases, the patient is in dire need of the drug and so the supply chain must operate at a much more rapid pace. The drug must be managed efficiently through customs and cannot be subjected to temperatures that may be damaging.

When managing global access programmes it is important to maintain complete visibility of all steps in the supply chain along with storage conditions, from the moment the drug leaves the warehouse to the time it gets to the patient's physician. An example of a recent shipment managed to Malaysia highlights the points in the process that should be monitored. Departure from the warehouse in Singapore to the airport was monitored and then reports were received at all subsequent steps:

•    When the shipment arrived on the plane

•    When the plane landed at its destination

•    When the shipment entered and cleared customs

•    When the shipment was finally delivered.

In addition to the location of the shipment, reports of the condition the shipment is in are available.

Due to the unpredictable nature of the locations, programmes often require creativity while maintaining the integrity of the supply chain. When recently managing a shipment to a patient in a remote area of the Northwest Territory of Canada, in order to deliver the drug to the patient, the services of the Royal Canadian Mounted Police had to be enlisted to drive the package to the hospital.

Timing: Patients who are part of a global access programme may require very rapid delivery of the drug, in some cases within 24-48 hours. With a longer supply chain and patients often located in distant time zones, efficiency is critical.

Anyone who has shipped even the most simple of packages overseas knows that the customs process can be a bottleneck. In some countries, customs are more unpredictable with individually interpreted import controls. In these cases, it is important to closely monitor the situation so as to prevent the shipment from getting delayed. Relationships with local freight carriers and distribution agents are essential for timely delivery as they can facilitate documentation procedures.

Individuality: A successful programme must be global in nature to reach those patients in need of the drug and leverage a dynamic, flexible approach to logistics, integrating both global and local or regional capability. Operating at the level of individual packs/shipments for individual patients, these programmes start with a global network of freight carriers but must become localised at the far end of the supply chain. Once shipments have arrived into one country, individual orders go onward towards different hospitals and may need to be handled at different temperatures.

For a shipment to a patient in Mongolia, for example, one would be best served by starting with a large carrier and then leveraging local carriers with better knowledge of local conditions as the shipment nears the patient.

An end-to-end view: In order to strengthen the supply chain, a best practice is to put in place an agreed method of onward transport with distribution partners. Checks should be put in place for the conditions to be fulfilled before partners are allowed to take product from distributors. It is also crucial to carefully monitor the pathway a drug takes to get to the service provider's warehouse from the manufacturer. A product won't be accepted into the supply chain unless it is known exactly where it is from and it is possible to prove where it has been from the moment it left the manufacturer.

When managing logistics for a global access programme, the goal must be to minimise risk and get the drug to the patient on time. The clock is ticking from the moment the request comes in and at the end of the supply chain is a patient in need. Supply chains should be 'challenged' on a regular basis to reveal any weak points, identifying critical hand-off points and which transactions may present difficulty. In conducting these exercises, one can then assign resources where needed most to ensure a smooth delivery and a successful outcome.

Free webinar: Early Access for Patients with Unmet Medical Needs, 4 December 2013, 2pm (UK time)

Article by
John Arnold

is Commercial Operations Director for the Clinigen Group. He can be reached at

29th November 2013

Article by
John Arnold

is Commercial Operations Director for the Clinigen Group. He can be reached at

29th November 2013

From: Research, Sales



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