Home use and sustainability opportunities leading to a renaissance in Lyophilized drugs

Can you tell us about your vision of self-administration at home becoming the next big shift in healthcare?

Bill said: Based on medical economic forces, much of therapy administration and chronic disease management will need to fall upon able patients or their caregivers because resources are spread so thin. We have seen other industries place tools in the hands of consumers leading to a more satisfying experience. For example, a decade or two ago, we would call a taxi company and hope the taxi shows up in 15-20 minutes. With Uber, Lyft and the like, we have much more transparency. We know where the driver is, whom to expect, when to expect them, what the cost will be, etc. I think health services can achieve many of the same efficiencies and customer experience gains with the right types of products, services and digital experiences.

There are novel compounds coming out all the time and clinics don’t necessarily have the capacity to administer every needed injection or dose for every patient. Patients also find convenience in managing their own therapies at home. Discovering and defining ways to make therapy delivery simple, easy, approachable and repeatable means that we as designers have a responsibility to imagine new solutions.

What kinds of solutions do you have in mind?

Bill said: One drug delivery method involves the reconstitution of medications. It arrives in dehydrated form in a small vial and patients can reconstitute the dose and potentially adjust the dose generally under the direction of the HCP based on biomarkers and other indicators before administering. We have seen many autoinjectors for at-home administration introduced over the last 10-15 years. While the convenience factor is wonderful, there is a lot of packaging and single-use plastics entailed in the delivery. Lyophilization – therapies distributed in powder form and reconstituted before use – can be a way to deliver more product to more people in a more shelf-stable way.

Another way to think about this is that it is not unlike what we’ve seen in many other fast-moving consumer goods. For example, we see more concentrated solutions for laundry detergent, dishwashing, etc. Smaller is better and more convenient. Manufacturers don’t need to ship air, and can ship less plastic packaging.

Why do you see lyophilization as a direction the drug delivery world should take?

Bill said: For one, lyophilized compounds, which have been converted to powder form, can reduce cold chain burden. They are often stable at shelf temperature and stable for longer. While not needing refrigeration might seem like a fairly nominal benefit, you need to consider the amount of energy that goes into refrigeration from the point of manufacture to storage, distribution and then to the patient. We have also heard complaints from patients not having the space in their refrigerator at home to store all of those autoinjectors in one carton.

Even though the preparation and administration of the solution might be more complex, there are sustainability gains to be had by making more compounds available in lyophilized form. It is a little more process intensive at the point of manufacture but based on the downstream considerations it ends up having a smaller carbon footprint.

If this is the case, why are we not seeing lyophilization used more often?

Bill said: It seems reflexively that drug developers assume that cold chain distribution is available and may not be testing the boundaries of the compounds they create. It requires the tweaking of the formulation and changing the behaviors of life scientists in terms of the care and handling of these compounds and how they are distributed. For example, getting medicines into remote parts of the world has been a longstanding challenge. Cold chain logistics are a part of that challenge. Not only is the carbon footprint more attractive but there is also a larger addressable market through shelf stability through lyophilization.

For the COVID mRNA vaccines, many of us had to visit infusion centers where vaccines were stored at extremely cold temperatures. Through lyophilization, we could potentially administer booster doses at home in the future.

What kind of investment is needed to educate patients to be able to reconstitute and selfadminister these drugs themselves?

Bill said: While they may not be for everyone, many people are perfectly capable of following the steps and developing the confidence to manage their own therapies. For many compounds, one needs to check for air bubbles or particulates and wait a few minutes before administering but that is even true for autoinjectors.

There are potential downsides to any drug delivery method. We have done work in a few conditions where people couldn’t leave home for more than a week or two because of the stability of their compound. Research we did around a mixing station that could reconstitute a drug in a repeatable way suddenly opened up the ability for people to be away from home for extended periods of time. While there may be more steps involved for administration, there are benefits as well.

How can companies integrate lyophilization into the already complex manufacturing process?

Bill said: The market for lyophilization equipment is growing and projected to continue to grow at 8-10% annually, even without high-volume therapies or vaccinations such as mRNA. While investment is needed at the manufacturing stage, downstream logistics will be simplified which can enable drug manufacturers to ostensibly pay themselves back through energy savings and packaging savings.

Can you tell us about lessons learned from PA Consulting’s projects in lyophilization?

Bill said: In one program we are formalizing a reconstitution platform that will be appropriate for a number of therapies. It doesn’t achieve all of the resource savings gains in terms of single-use plastics, etc, but it is certainly a step in the right direction. These compounds are stable at room temperature and in other benchmarking we have seen an up to 96% reduction in primary packaging resources volume based on drug reformulations. There are indicators we are finding to give us more courage behind our conviction to invite drug developers to consider lyophilization not as a concession but as an opportunity.

Anything else?

Bill said: There is no single magic bullet on making drug reconstitution approachable for all patients on all occasions, but the majority of doses for many types of compounds can be delivered in this way provided thoughtful design of the reconstitution process, the components required, and the sustainability and reusability of accessories or products. These are opportunities we would love to explore with biopharma organizations.