Navigating Targeted Therapy Access: Innovations and Challenges in High-Cost Treatments
Evolving Landscape of Targeted Therapies and Access Challenges
Expanding targeted therapy access remains a critical challenge amid the pharmaceutical market’s shift toward high-cost innovations, such as orphan drugs and advanced therapy medicinal products (ATMPs), which target smaller patient populations, often for rare diseases. As detailed in the BMJ article by Tarang Sharma and colleagues, this evolution creates tension between policy incentives driving innovation and barriers to equitable access, even in high-income countries, due to prohibitive costs exceeding €2 million per treatment and immature evidence from small-scale trials. The authors advocate for enhanced international collaboration, patient engagement in value assessments, and managed entry agreements to balance innovation with sustainable reimbursement, drawing on initiatives like the Oslo Medicines Initiative and the WHO’s Access to Novel Medicines Platform.
Inadequacies in Traditional Value Assessment Frameworks
Standard health technology assessment (HTA) frameworks fall short for evaluating costly new drugs, prioritizing cost-effectiveness and budget impact while regulators focus solely on efficacy and safety, leading to divergent stakeholder perspectives. For instance, the authors cite that over half of orphan drugs approved in Europe between 2000 and 2017 relied on government incentives for viability, yet these therapies often achieve blockbuster status through approvals for multiple rare conditions, complicating value determination. They support this with examples like the European Medicines Agency’s approval of 29 ATMPs by 2025 and the US Food and Drug Administration’s projection of 10-20 annual cell and gene therapies, emphasizing how single-dose, high-cost models with long-term benefits create uncertainty for payers. These insights highlight the need for broader value perspectives incorporating patient experiences to align assessments across stakeholders, fostering more holistic decision-making that improves targeted therapy access.
Methodological Foundations from Collaborative Initiatives
The article’s claims are grounded in the Oslo Medicines Initiative (2020-2022), a collaboration between the WHO Regional Office for Europe, Norway’s Ministry of Health and Care Services, and the Norwegian Medicines Agency, which analyzed policies for equitable access to costly drugs through stakeholder consultations and technical reports. Building on this, the Access to Novel Medicines Platform facilitates ongoing dialogue among governments, industry, and patients to pilot solutions, such as joint value assessments and procurement, exemplified by voluntary collaborations like the Beneluxa Initiative and Nordic Pharmaceutical Forum. Methodologically, the BMJ article integrates literature reviews, such as studies on managed entry agreements in six countries showing a 6% average list price increase, and qualitative evidence from patient consultations in countries like Canada and Norway, which incorporate lived experiences into HTA. This evidence-based approach supports the discussion toward practical reimbursement reforms, highlighting how these initiatives address legal, confidentiality, and trust barriers through consensus-building to enhance targeted therapy access.
Expanding Managed Entry Agreements for Risk Sharing
Managed entry agreements—financial or outcomes-based arrangements between industry and payers—serve as a key mechanism to manage uncertainty for high-cost drugs, allowing conditional coverage while generating real-world evidence. The authors reference a 2023 review of ATMP reimbursement across 20 countries, which calls for harmonized frameworks to enhance affordability, and a Norwegian study from 2021-2022 demonstrating how such agreements balance early access with evidence development. For example, outcomes-based models like “pay for performance” tie payments to milestones, mitigating risks from surrogate endpoints in rare disease trials, while financial discounts address budgetary impacts. The article suggests expanding these agreements via platforms like the Access to Novel Medicines Platform, which develops checklists for patient-industry-payer collaboration, potentially increasing market access for industry while reducing inequities and supporting targeted therapy access.
Role of Patient Engagement and Public-Private Partnerships in Value Determination
Patient involvement serves as an important element, advocating for incorporating qualitative evidence of lived experiences into value assessments to capture holistic benefits beyond clinical endpoints. It points to mechanisms like the Mechanism of Coordinated Access to orphan medicinal products, which has expedited access for 23 rare disease treatments by including patients in early dialogues with payers and regulators. Public-private partnerships are highlighted through examples like the COVID-19 Recovery trial, which demonstrated rapid innovation via collaborative data collection, and calls for public funding in ATMP development under EU hospital exemptions. These elements support the broader argument for lifecycle value pricing, where post-patent competition and evolving evidence inform cost-effectiveness.
Critical Examination of Collaborative Approaches
The article advocates for international collaboration and patient engagement to enhance access to costly therapies, however, it does not consider the feasibility and unintended consequences. Emphasizing managed entry agreements overlooks their opacity, as confidential terms often preserve high list prices through undisclosed discounts, potentially inflating global pricing via international reference mechanisms rather than truly sharing risks. Moreover, the reliance on voluntary platforms like the Access to Novel Medicines assumes high levels of trust and alignment among diverse national systems. Also, legal barriers and varying HTA priorities could exacerbate inequities, favoring larger economies and leaving smaller ones behind. This optimism about patient-driven value assessments may also undervalue the challenges of integrating subjective lived experiences into rigorous economic models, risking biased or inconsistent reimbursements that strain public budgets without guaranteeing superior outcomes.
Broader Implications for Health Economics and Market Access
The article’s findings suggest a shift toward dynamic, multi-stakeholder value frameworks that incorporate real-world evidence and societal values, potentially reducing access disparities in reimbursement decisions. In market access and pricing terms, joint international negotiations could enhance bargaining power for payers, as seen in Baltic Procurement Initiative successes, while managed entry agreements mitigate fiscal risks for novel therapies amid rising ATMP approvals. From an outcomes research perspective, the emphasis on post-launch data collection via pragmatic trials aligns with trends toward precision medicine, though it necessitates investments in health system readiness. Reflecting on broader trends, such as the EU’s 2025 joint clinical assessments under Regulation (EU) 2021/2282, these proposals could foster sustainable innovation, ensuring evidence-based mechanisms evolve to support equitable, long-term patient outcomes without compromising fiscal responsibility.
