End-to-End Solutions Optimize Rare Disease Clinical Trials and Product Commercialization

A growing number of pharmaceutical manufacturers now rely upon AscellaHealth as a single resource for managing all phases of rare disease clinical trials and implementing go-to-market strategies. As a trusted Specialty Pharmacy solutions partner, our team cost-effectively eases the burdens of dealing with multiple vendors, streamlines processes and speeds patient time-to-treatment.

While the challenges associated with rare disease clinical trials persist, our team of professionals has the deep experience and comprehensive expertise to support manufacturers worldwide in their quest to serve specialty populations with the novel drugs, cell and gene therapies (CGT) that they desperately need.

Defining Cell and Gene Therapies

According to the American Society of Gene + Cell Therapy: gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Cell therapy involves the transfer of cells with the relevant function into the patient. Some protocols utilize both gene therapy and cell therapy.

The AscellaHealth team provides a complete suite of end-to-end solutions that addresses some of the most pressing key market challenges.

Challenge: Clinical Trial Strategy Planning and Management, Patient and Physician Recruitment, Regulatory and Logistics Issues

Timing is key for patients who are battling rare disease and fail to get enrolled in a clinical trial that could provide life-changing treatment. Yet in every corner of the world, the clinical trial industry is struggling to identify, screen and recruit trial participants for specialty drugs or CGTs -- with 50-70% of sites failing to even recruit one patient for a study. Additionally, many physicians outside of academia say there are too many obstacles that stand in the way for them to participate and conduct research. Finally, it is sometimes challenging to identify clinical research centers in a designated geographic area that have previous experience in treating patients with a rare disease and conducting a clinical trial.

AscellaHealth Solution

From pre-clinical and feasibility studies to protocol reviews and pre-trial planning, the AscellaHealth advisory team provides guidance and solutions for logistics that continue throughout all phases of a trial. Our complete range of services also supports preparing for pre-investigational new drug (IND) meetings for a drug sponsor's first formal meeting with the FDA as they begin the process of bringing a drug to market.

With longstanding collaborative relationships throughout the rare disease community, our team can readily tap into patient organizations, registries and advocacy groups in order to accelerate participant recruitment and re-populate a trial when patients drop out. Social media has also been playing a key role in raising awareness and creating disease-specific patient communities and groups, representing an innovation that has enhanced recruitment strategies.

For patients and families who cannot travel the long distances or cover the high cost of travel, lodging and other expenses to reach trial sites, we also manage de-centralized trials and digital communications -- those executed through telemedicine and mobile/local healthcare providers. This option, popularized during the pandemic, is predicted to grow in 2023 and beyond as sponsors acknowledge the need for a degree of flexibility for patients who have to juggle their personal lives and trial participation.

AscellaHealth’s expertise also supports physician recruitment and allows us to draw upon provider practices that specialize in treating specialty populations. Our trained and experienced care coordinators welcome each physician office, provide ongoing support and build program trust. These activities include setting up proactive reminders regarding lab testing and telehealth visits and establishing communications channels to share patient feedback.

We ‘speak the language’ of rare diseases, with expert communications management that:

• Enhances patient engagement and adherence to therapy throughout the trial. A dedicated care coordinator is assigned to manage program enrollment, welcome patients/caregivers, advance their understanding of therapy and build a patient profile that enables real world evidence (RWE) tracking.
• Manages interaction with trial sites and call centers
• Rapidly enlists key opinion leaders (KOLs)

Challenge: Patient Testing, Site Visits, Product Delivery, Reporting

Today, trial sponsors face increased complexities of managing and conducting patient testing, which now includes getting product to the patient’s home for de-centralized trials. This also involves arranging for site visits and generating accurate, timely reporting of RWE and testing results.

AscellaHealth Solution:

Our care coordinators are positioned to ask all the right questions. Through ongoing communications that lead to enhanced patient engagement, they gather RWE that uncovers life changes. They document feedback, such as determining if the patient missed work or school, or if they experienced physical changes, i.e., swelling or other adverse events, reactions and exacerbations. Following established protocols for adverse event reporting, they also complete all required forms and templates and submit documentation to the appropriate government authorities.

This multi-faceted approach to patient care coordination and RWE reporting includes activities for monthly clinical care planning, site assessment and pharmacy consultation. A clinical education team manages and supports patient engagement compliance with treatment, with special attention to patient monitoring that is tailored to the requirements of longer-term trials and longitudinal studies.

For example, CGTs typically require one treatment, while other specialty drugs may necessitate administration over prolonged time periods. AH provides monitoring services and RWE collection for whatever timeframe is determined by the trial sponsor and continues these activities through post treatment follow-up, establishing a basis for patient journey mapping.

To ensure patient access to therapies, trial product fulfillment and coordination services make certain that product is delivered to the physician office, site or home, as needed. Shipment updates inform all stakeholders and site of care support teams manage required Institutional Review Board (IRB) approvals. To maximize cost savings for our trial sponsor partners and further optimize patient compliance with treatment, we select only the most efficient methods of transportation for safe, timely product distribution. All services are designed to scale when new sites or patients are added to the trial.

Our approach also incorporates a health economics-driven Medical Record System to:

• Record and publish data
• Articulate pre-booking strategies with payers and insurance brokers
• Develop a foundation for value-based contracting
• Confirm trial milestones
• Support prior authorization

Challenge: Getting Patients on to Therapy

When a clinical trial ends, transitioning patients on to therapy can be cumbersome. Time-sensitive barriers to physician prescribing, ensuring product delivery and monitoring therapy administration all pose challenges. Consider the hurdles of obtaining a verbal authorization for the drug from a physician at a trial site in California and making sure that the patient who is located in Iowa gets the prescription filled. There are just too many potential snags in communications and product fulfillment that would upend the process and delay patient access to treatment.

AscellaHealth Solution:

We make the process seamless with an incredible track record of getting patients on to therapy faster: 96% of patients who finish a trial are on therapy within 30 days.

Dedicated care coordinators close all the gaps in care transitions and facilitate timely communications among patients, physicians, pharmacists, insurance companies, labs and other participants in the care continuum.

The AscellaHealth service model supports product development within Phase 2, Phase 3, Expanded Access Programs (EAP) to commercialization and beyond. All custom programs are part of our services supporting clinical trials, AscellaHealth coordinators gather RWE and build patient populations that expand registries and increase opportunities for drug utilization.

The FDA advises that expanded access may be appropriate when all the following apply:

• Patient has a serious or immediately life-threatening disease or condition.
• There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
• Patient enrollment in a clinical trial is not possible.
• Potential patient benefit justifies the potential risks of treatment.
• Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

Accelerated time-to-treatment is one of the many benefits of working with a single Specialty Pharmacy solutions partner that provides end-to-end solutions throughout the product life cycle – from clinical trials and pre-commercialization through product launch, market access and distribution.

Data collection and RWE evidence reporting from one source ensures consistency and accuracy that smooths the transition from research and development to market introduction. The bonus for pharmaceutical companies is getting rare disease patients the lifesaving treatments and therapies they have long been waiting for.

AscellaHealth is attending the Asembia Specialty Pharmacy Summit in Las Vegas, April 30 – May 4, 2023. To schedule a meeting, please click here.