With five FDA programmes moving drugs through approval to commercialisation, the speed of scale-up is quickening, shortening time to market by months.
To make the most of these expedited programmes, drug developers should evaluate their entire process to eliminate bottlenecks and non-value-added tasks. This evaluation should include logistics, where planning temperature-control strategies as early as possible in the development process helps avoid delays and get medications to patients more quickly.
To put the trend toward speedy approvals in context, 200 of the 367 novel drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) between 2011 and 2018 used one or more of its expedited development programmes. Of those 200, 12 per cent received the Accelerated Approvalii designation. The remainder were expedited under Fast Trackiii, Breakthrough Therapy, Regenerative Medicine Advanced Therapy, or Priority Review designations.
A growing percentage of those drugs require cold-chain or controlled room temperature shipping and handling. Specifically, in 2018, 44 per cent of the FDA-approved drugs required controlled temperature handling, and the percentage increases each year. The 2019 Biopharma Cold Chain Sourcebookvii estimates that by 2023, approvals for temperature-controlled pharmaceuticals will increase 59 per cent from 2017 figures.
Biologics are the main reason for this increase. Today’s novel therapeutics often are developed by innovative small to mid-sized biopharmaceutical companies that thrive on rapid development. To deliver therapeutics as quickly as possible, they often include rare and orphan diseases applications in their portfolios in the hopes of qualifying for expedited approval designations.
That’s an effective strategy, but it doesn’t follow the strategic template used for blockbuster drugs. For example, development and manufacturing activities for rare and orphan diseases are much smaller. Clinical trials and even post-approval therapeutics may involve only a handful of patients in any given region. Therapeutics for rare and orphan diseases (as well as investigational therapeutics that are administered as last resorts under the Right to Try Actviii) have limited markets.
To maximise the potential of today’s small-batch, personalised medicines, biopharmaceutical companies need managers who understand the newer, faster pace and start planning early. By planning early, they can deliver potentially-life-saving therapeutics to patients as quickly and effectively as possible.
Strategies to Anticipate Your Cold Chain Needs
Participating in one of the FDA’s expedited programmes is only one step to commercialising and delivering innovative therapeutics quickly. There are additional steps drug developers can take to further speed the process. Consider these points:
1 Map Your Cold Chain Supply Early
Begin discussing your controlled temperature challenges during the drug development stage. Identify drug stability issues that may be affected by temperature, light exposure, shock, or vibration. Then research packaging solutions and data monitoring systems that meet the requirements of the data required by the regulators from your planned launch regions and the World Health Organization’s (WHO’s) Good Distribution Practices (GDP) and your own logistics team. By beginning early, you can find cost-effective solutions for all transportation options and speed time to market.
Also, use development time to create more stable formulations with formulation exposure to environmental hazards with simulated transportation. This simplifies packaging and logistics needs and can reduce logistics expenses. Confirming formulation robustness can also expand the market for novel and existing therapeutics (especially in the developing world, where refrigeration is unreliable).
At the same time, use the development time to identify potential cost savings throughout the cold supply chain. This may mean more efficient routes or logistics partners, or new or improved technologies that support delivery methods that were unthinkable until recently. For example, UPS and CVS deployed drones for the first time in November for last-mile delivery, and Merck is testing drones for disaster response. Earlier, improved reefers and condition monitors have made maritime and rail shipping as reliable and economical for pharmaceuticals as they have been for more durable goods.
2 Adopt GMP and Continuous Manufacturing
Develop your products with good manufacturing processes (GMP) and continuous manufacturing (CM) in mind from the beginning. Designing processes for scale-up from their inception minimises the need to rework formulations and resubmit regulatory data when ingredients change. Likewise, CM enhances flexibility in batch-saving ways thanks to continuous optimisation. Both strategies decrease development time from preclinical to commercial stages.
3 Speed Global Access to Individualised Therapies
Developing a global regulatory cold chain strategy rather than a country-by-country approach is another time-saver. Once the markets are identified, determine their regulatory commonalities and differences. Then design the studies to meet the most rigorous regulatory requirements of those countries. (Note that even regions with similar requirements, like the U.S. and EU, are not perfectly aligned.) Developing a multi-nation strategy during the drug development process is faster and more cost-effective than waiting until after commercialisation in your primary markets.
Early Planning Matters
Accelerated drug approval pathways are making significant impacts on patient health as well as the health of the companies that develop them. Those gains can be unwittingly sabotaged, however, when drug developers neglect the big picture.
Planning early for GMP, scalable and continuous manufacturing, global regulatory requirements, and temperature-controlled logistics, helps companies advance with certainty. Planning early helps minimise delays and setbacks and makes the most of the opportunities afforded by the FDA’s expedited drug programmes.
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