Re-Think. Re-Consider. Re-Calibrate.

If there is one word to describe the best go-forward approach for pre-commercialisation and market access planning it is CHANGE. 2023 will be a time to move away from legacy models that may have traditionally worked and shift toward longer, more adequate timeframes – as long as 3-4 years -- that ensure reaching potential patients, providers and payers.

For complex products such as cell and gene therapies (CGT), there are new considerations throughout the pre-commercialisation journey to leverage unique financial programmes, finalize payment and risk-sharing mechanisms between pharmaceutical companies, and payers to proactively remove financial barriers to enhance medication access, while optimising outcomes. This extended planning process is also critical to ensure the therapy itself is readily available since there is little room for failures, bottlenecks or delays throughout the supply chain.

Given these challenges, it is critical that manufacturers work with an experienced Specialty Pharmacy (SP) solutions partner who can help them navigate challenges at each step—from market access and clinical trials to commercialisation and patient support solutions to increase compliance. A consultative approach results in seamless new product launches, improved medication access and enhanced clinical outcomes for patients with specialty conditions and rare diseases.

Successful partnerships with manufacturers depend upon the execution of end-to-end solutions and a complete range of programmes across the product lifecycle and beyond to ease the administrative burden of managing multiple vendors. When manufacturers are able to work with a single vendor for all program and drug launch milestones, the opportunities for a flawless launch are exponentially greater- not to mention the clean, actionable data which results in programme optimisations.

When and How to Prepare the Market

The experienced AscellaHealth market access professionals are steeped in knowledge and understanding of the rare disease community and the complexity of new specialty drugs and CGTs. This expertise enables our team to plan communications outputs that effectively educate and engage with patients, caregivers, payers, and healthcare systems.

Success metrics are tied to multiple capabilities that should be benchmarked throughout the process. Here’s a compendium of resources that Terebellum brings to its partners:

• Support for patients and caregivers is a key to success, since the diagnostic odyssey for a rare disease can be 5 years or longer. Global Genes, a rare disease advocacy nonprofit, reports that “patients living with rare disease visit an average of 7.3 physicians before receiving an accurate diagnosis.”

That’s why our approach goes well beyond a standard information hotline or static web information. We developed well-designed patient services, support infrastructure and the personalized attention of care coordinators who provide education and support with one-to-one outreach and documentation of progress throughout the treatment journey.

• An astute market access team is a “must-have” in market preparation, ensuring streamlined access to therapies and optimal pricing on the day of launch. Our professional team has the experience, contacts and know-how to develop strategic pricing strategies that enable manufacturers to launch a successful product into a competitive market.

• Innovative financial services are becoming an essential component of go-to-market strategies, allowing the high one-time costs of gene therapies and other expensive medication to be converted into small, predictable payments over time. This option grows more critical as new therapies become available with price tags as high as $3.5-Million for the new Hemophilia Gene Therapy.

• A focus on data collection is essential. The right SP partner will help manufacturers to demonstrate long-term outcomes, providing real-world evidence that supports payers with early visibility into the total health system costs related to a particular disease. A partner that focuses on data collection is highly valuable, because the disease burden is often not quantified for rare subpopulations indicated for CGT or certain orphan drugs. Data is also needed to engage in outcomes-based contracts and establishing the causal relationships between the disease and outcomes.

• Optimising the supply chain cannot be overlooked during pre-commercialisation, with demand forecasting beginning as early as three years before the actual launch to ensure that patient-specific doses are delivered just in time to sites of care. Many manufacturers are embracing our exclusive drug distribution services since medications for rare diseases may require special handling needs, such as refrigeration, overnight delivery, and shipment tracking. An SP partner that specializes in rare diseases already uses these techniques to provide uninterrupted therapy. As your partner, we can offer exclusive, national distribution, ensuring patients have access to therapies they need when they need them most. This model can also offer manufacturers significant cost savings.

Prepare NOW

When manufacturers choose Terebellum as a single-source SP partner, they take the most important step forward in ensuring that the market, the product and the company itself are fully prepared for an effective launch. Time is of the essence to resolve the challenges of commercialising drugs and therapies, updating organisational models for efficiency and scale and going to market with a fail-safe approach that rests upon meticulous planning and disciplined execution.

It's always important to keep in mind that a single thread runs through all activities: the patient is the focal point of every program.

Visit terebellumltd.com to learn more about Terebellum’s comprehensive portfolio of business consulting, supply chain, distribution, fulfilment and unique financial solutions for Life Sciences manufacturers.