How Cell and Gene Therapy Companies Can Adapt in an Evolving Commercial Landscape

For Cell and Gene Therapy (CGT) manufacturers, the strategies for bringing these life-changing treatments to market were never going to be particularly simple.However, the addition of the COVID-19 pandemic has further strained regulators, clinical trial recruitment, the global supply chain, and sites of care, adding commercial complications where they are not needed nor welcomed.

According to the Alliance for Regenerative Medicine, there are 1,220 ongoing clinical trials for regenerative medicines and advanced therapies worldwide; 152 of those are in Phase III. FDA was already taking steps internally to keep up with growing CGT demand, predicting the approval of 10 to 20 CGT products each year.¹ However, since the onset of the pandemic, priorities have had to shift, in-person surveillance and pre-approval inspections face new challenges, and global alignment efforts around gene therapies have been disrupted.²

As if a tougher-than-usual regulatory environment wasn't challenging enough, an influx of new cold-chain products, including the COVID-19 vaccines, have increased demand for cold and ultra-cold storage with supply chain partners and sites of care. In recent years, as many as half the drugs approved by FDA were cold chain products.³

"Distribution vendors, like 3PL [third-party logistics] partners, have already made the adjustments to accommodate the growing demand for cold storage," says Joel Wayment, Vice President of Operations, Cardinal Health Third Party Logistics. "Sites of care are different. They're laser-focused on patient care, and managing pandemic care has put added stress on their systems, which may stretch their ability to accommodate the growing needs of frozen and ultra-frozen products."

The extra hurdles CGT manufactures are currently facing may sound overwhelming, but challenges and opportunities often go hand-in-hand.Companies willing to reassess and pivot strategically may realize unforeseen commercial advantages.

Changes for commercial approach CGT

Take good care of those who care for your patients by prioritizing site of care preferences. Manufacturers can make great strides in developing CGT products that can fit into existing site of care operational workflows rather than reinventing care pathways that can be significant disruptors.

Douglas Bock, Partner, Archbow Consulting, points out that "CGT manufacturers should challenge themselves to make their products as easy as possible to order, store, prepare, and administer. These can and should be pre-clinical or Phase I conversations."

Hospitals, healthcare systems, and other sites of care can be instrumental in guiding successful operational decisions when it's still relatively easy to make product design changes.

Archbow Consulting recently conducted market research with US hospitals on behalf of a gene therapy company entering Phase I to understand how a new therapy's target product profile would be received by these essential sites of care. The research revealed operational complexities that would likely have a negative impact on future product adoption. Meaningful opportunities to ease logistical complications were identified, allowing the company to course-correct early in development.

This commitment to prioritizing the needs of sites of care can even extend to understanding how they work with supply chain partners. "Minimizing complexities for sites of care, like how products are ordered, stored, and invoiced, can simplify the management of a complex yet exciting therapy.The focus should be on the patient and not complicating the order-to-cash process.It is important to engage the sites of care early to help them understand any nuances to that process so that there is time to adjust and implement," says Wayment.

Begin engaging commercialization partners (much) earlier. Moving a CGT from clinical trials to commercial sales requires 24-36 months of careful planning. In light of the longer lead times needed in a pandemic environment, Archbow Consulting recommends that CGT clients allow additional lead time for crucial initiatives:

• Launch minus 36 months: Discovery and planning phase that includes strategy design, market research, advisory boards, and mapping the patient and product journey
• Launch minus 24 months: Global (ROW) distribution planning that includes selecting global distribution partners, inventory management, apheresis coordination (if needed), quality release planning, and order-to-cash strategies
• Launch minus 24 months: Pricing and reimbursement strategies that include US and global pricing and contracting, HEOR research, and US payer strategies
• Launch minus 21 months: Patient support decisions, customer engagement (sites of care), packaging and handling decisions, and development of a data strategy
• Launch minus 21 months: US distribution planning that includes state licensing, selection of US distribution partners, inventory management decisions, and order-to-cash strategies

It's important for manufacturers to know that vendor partner engagement does not necessarily equal expense. Most programs will not charge monthly fees until launch, eliminating any financial benefit to waiting.

Challenge internal teams and vendor partners to innovate in ways that benefit sites of care without disrupting them. Innovation discussion prompts may include:

• How can (ultra) cold-chain shippers or dewars be better designed to act as self-contained storage units to reduce storage requirements for sites of care?
• How well does shipper tracking and monitoring technology meet the needs of sites of care?
• Sophisticated hospital finance departments may not need traditional HUB-type reimbursement services.What type of support will they need?
• For products that meet safety requirements, are alternative (non-hospital) sites of care an option for administration going forward because those sites have invested in freezer storage for the COVID-19 vaccine?
• Could new COVID-19 safety protocols in sites of care alter therapeutic timelines for your product, effectively dictating administration locations (e.g., outpatient vs. inpatient) and impacting financial viability?What changes can your team make in operational design to avoid undesirable operational hurdles?
• With sites of care largely focusing on their pandemic response, how can obstacles to the onboarding and CGT training processes be minimized?
• What value can Specialty Pharmacies add to the distribution of CGTs?
• CGT products might necessitate collection of different data than traditional biologic products. How can existing partners (distribution, HUB, etc.) help to capture this data without disrupting processes at the site of care?

In the current environment, CGT commercial strategies are certainly not without obstacles. But that doesn't mean all hope is lost. Making these changes now, regardless of the pre-clinical or clinical trial phase, will better prepare products for commercial success.

References

1. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics
2. https://www.raps.org/news-and-articles/news-articles/2020/10/fda-officials-experts-discuss-impact-of-covid-19-o
3. https://healthdelivered.org/2020/09/18/pharmaceutical-distributors-are-at-the-forefront-of-cold-chain-logistics/

Katie Rapp, Vice President, Archbow Consulting