If there is one word to describe the best go-forward approach for pre-commercialization 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 gene and cell therapies (GCT), there are new considerations throughout the pre-commercialization journey to leverage unique financial programs, finalize payment and risk-sharing mechanisms between pharmaceutical companies, and payers to proactively remove financial barriers to enhance medication access, while optimizing outcomes. This extended planning process is also critical to make sure 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 that can help them navigate challenges at each step—from market access and clinical trials to commercialization 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 programs 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 program optimizations.
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 GCTs. 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 AscellaHealth 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.
Our proprietary Site of Care (SOC) optimization program for infusion therapies benefits all stakeholders: AscellaHealth’s Home Infusion Pharmacy Network is a leading strategy for reducing costs without compromising quality of care for specialty biologic medications. This approach is improving outcomes by offering enhanced access to more convenient, quality-focused settings.** Specialty products include a wide range of components, such as vaccines, blood and blood components, allergenics, somatic cells, GCT, tissues and recombinant therapeutic proteins.
An astute payer negotiation 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 communicate with payers regarding pricing strategies that work for the manufacturer and the health plan or employer group.
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 for payers. 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.
Our comprehensive suite of copay assistance solutions supports manufacturers in their quest to garner transparency. Our Copay Advisor™ monitors and tracks manufacturer copay funds to ensure allotted funds are utilized to help offset costs of expensive therapies for patients.
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 GCT or certain orphan drugs. Data is also needed to engage in outcomes-based contracts and establishing the causal relationships between the disease and outcomes.
Optimizing the supply chain cannot be overlooked during pre-commercialization, 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.
Educate & Inform Physicians and Payers Pre-and-Post Launch
Educating clinicians, physicians and payers early in the process enhances opportunities for market access success.
With specialty drugs and GCTs, it’s important to remember that physicians deal with chronic conditions on a daily basis, but experience shows that doctors deal with few rare disease patients. Doctors do not receive the training and education necessary to recognize symptoms and understand treatment options for these rare diseases. For these reasons, it is critical to provide full support for the therapy itself by offering evidence-based information to providers and payers. One survey sought to promote understanding of the challenges healthcare providers face in diagnosing and treating rare disease patients. Consistent with small patient populations for each disease, lack of rare disease education, and symptom awareness were the most common challenges when addressing rare disease patients.
Additional challenges included shortages of physicians specializing in rare diseases, limited facilities dedicated to rare diseases and a lack of clinical trials for disease areas. However, increased education for physicians was the most commonly reported factor for improving rare disease diagnosis and treatment over the next five years—cited by more than half of respondents.
Manufacturers need an SP partner that recognizes specific challenges and is equipped to address and resolve the issues facing physicians. Physicians may be most familiar with "Specialty" drugs but may not be as familiar with gene therapies, largely because their practices may not include rare disease patients who would benefit and the low number of therapies approved. This is likely to become even more critical in market access with more than 900 Investigational New Drug (IND) applications for ongoing clinical studies of gene therapy products underway, and the FDA predicting they will be approving from 10 to 20 gene therapies per year.
When manufacturers choose AscellaHealth 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 commercializing drugs and therapies, updating organizational 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 central point of focus of every program.
Contact AscellaHealth to learn more about AscellaHealth’s comprehensive portfolio of services for Life Sciences that are customizable based on client needs, available “a la carte” and include: