Advanced Therapies Medicinal Products (ATMPs) is a term for a range of new, leading-edge therapies that expand science, meet significant unmet need, and gain expedited market access. Check out the executive summary of ATMPs: Access, Pricing, and Ethical Issues in Perspective which explores ATMPs, the market access and commercial challenges facing these innovations, solutions to those challenges, and patient and clinical ethical considerations.

Challenges in Access and Commercialization of ATMPs

ATMPs present the opportunity to bring curative treatments to underserved patients, however, major challenges remain in areas such as market access, manufacturing, and logistics, requiring innovation.

• Patient pathway: Finding patients to treat—especially in clinical trials—is often challenging, particularly for rare diseases that were previously untreatable. And not all healthcare facilities have the right expertise to become a clinical facility.
• Market access: Most advanced therapies are costly, and health systems, especially in the US, are not set up for one-time large payments. Legal and regulatory reforms to enable multiyear payment models may be required for these therapies to become broadly accessible.
• Clinical evidence: The innovative clinical trial designs enabled by—or required because of—small patient populations and high unmet need allow therapies to get to market faster, however, clinical questions, such as long-term safety and efficacy, are often left unresolved.
• Manufacturing and logistical complexity: Traditional supply chain management does not necessarily create patient value in emerging therapy value chains. Operations should be the backbone of a distribution model that delivers these products efficiently and effectively.

Innovative Solutions for ATMPs Access

Alternative Reimbursement Strategies

Facing pressure on reimbursement for these expensive drugs, payers are open to exploring innovative payment systems that ensure a more sustainable model for both healthcare systems and patients. Establishing a value-based contract (VBC) is one of the potential solutions to address the existing challenges in the financing and access of ATMPs. There are different categories based on the uncertainties whose impact they are intended to resolve, including financial-based and outcome-based VBC.

Accelerating Time to Patient Access of Innovation Through Artificial Intelligence

Digitization and analytics in the discovery and research and development (R&D) of ATMPs are part of the innovative solution to increase opportunities for patient benefit, although their implementation presents some challenges.

Challenges of applying artificial intelligence (AI) to R&D for ATMPs:

• Limited experimental data and costly data generation
• Functional complexity and wide variety of potential solutions
• Differences between laboratory experimentation and “in silico” research

Opportunities to apply AI along the R&D value chain for ATMPs:

• Target identification
• Payload design optimization
• Translational and clinical development
• Chain digitization

The different applications of AI can bring benefits in the management of ATMPs, from optimizing the selection of patients with the greatest potential benefit to promoting patient adherence and understanding of the disease.