Reforming Health Technology Assessments to Embrace Gene Therapy Advances 

The advent of cell and gene therapies (CGTs) represents a paradigm shift in medicine, offering the potential to cure diseases through one-time interventions with lifelong benefits. However, despite their transformative potential, these therapies face significant challenges in market access and reimbursement due to limitations within current Health Technology Assessment (HTA) processes. 

Current HTA processes struggle to appropriately assess therapies that require substantial upfront costs but provide prolonged, sometimes lifelong, benefits. As a result, health systems, payers, and policymakers are increasingly confronted with the question: Are current HTA processes fit for purpose in evaluating cell and gene therapies? 

This article synthesises insights from a recent panel discussion – Cell and Gene Therapy Summit Insight hour: Are health technology assessments fit to judge the value of cell and gene therapies? This webinar was hosted by Johnson & Johnson, outlining the key barriers to access and proposing policy reforms necessary to ensure equitable, evidence-based decision-making for these groundbreaking treatments. 

The Fundamental Challenge: HTA Methodologies Discount Future Benefits 

​Traditional HTA methodologies often rely on economic evaluations, such as cost-effectiveness analyses, to assess the value of new treatments compared to existing options. Often, a discount rate is applied to future health benefits and costs within economic models to reflect their present value. While this methodology is effective for chronic treatments that distribute costs and benefits over time, it presents a fundamental misalignment when applied to gene therapies, which: 

• Incur costs upfront but deliver health benefits over decades. 

• Are assessed using discount rates that reduce the present value of long-term benefits, making them appear less cost-effective. 

• While the reliance on real-world data (RWD) at the time of launch varies across markets, the lack of sufficient RWD at launch could lead to conservative valuation by payers. 

Some countries, such as Sweden, Belgium, and the Netherlands, have begun implementing differential discount rates for long-term therapies to better reflect their true value. However, this practice remains inconsistent across countries.  

Policy Recommendation: 

HTA processes should adopt lower discount rates for cell and gene therapies to ensure long-term health gains are appropriately valued and not disproportionately devalued due to standard economic modelling conventions. 

Addressing the Financing Problem: Structuring Payments for Sustainability 

One of the principal objections to gene therapies is their high upfront cost, often reaching millions per patient. However, as highlighted by one of the panellists Prof. Lotte Steuten – the Deputy Chief Executive of the Office of Health Economics, this is not an issue of economic inefficiency but one of financing. 

“When purchasing a house, individuals do not pay the full amount upfront. Instead, they finance it over time. Why should healthcare systems approach gene therapy any differently?” 

Despite their high initial cost, gene therapies have the potential to reduce long-term healthcare expenditures by eliminating the need for lifelong treatment. Yet, current reimbursement models treat these therapies as one-time expenses, which places a significant burden on short-term healthcare budgets. 

Policy Recommendation: 

Healthcare systems should adopt innovative financing models that align payment structures with the long-term value of gene therapies. Potential approaches include: 

• Annuity-based payments, where costs are distributed over multiple years. 

• Outcome-based reimbursement, in which payments are contingent upon demonstrated clinical efficacy. 

• Subscription models, where payers contribute a fixed amount annually to cover eligible patients, mitigating financial shocks. 

By reframing gene therapy costs through financing rather than affordability, HTA can enable more sustainable reimbursement pathways without restricting patient access. 

Managing Uncertainty: The Role of Real-World Evidence in HTA Decision-Making 

HTA agencies often hesitate to approve gene therapies due to limited long-term clinical data at the time of market entry. This uncertainty results in payer reluctance and downward pricing pressure, which in turn: 

• Sets unsustainable pricing precedents for future therapies. 

• Disincentivizes innovation, leading to market withdrawals by smaller biotech firms. 

• Delays patient access as protracted negotiations stall reimbursement decisions. 

Panellists emphasised the necessity of adaptive approval mechanisms that allow treatments to enter the market while real-world evidence (RWE) is continuously collected. 

Policy Recommendation: 

HTA agencies should adopt adaptive decision-making frameworks, including: 

• RWE collection mandates, ensuring that pricing and reimbursement decisions evolve based on emerging data. 

• Managed entry agreements (MEAs), where pricing is adjusted dynamically in response to real-world performance. 

• Conditional reimbursement policies, allowing access under specific data-sharing and monitoring conditions. 

Addressing Global Inequities in Gene Therapy Access 

A central ethical concern raised during the discussion was the disparity in gene therapy access across different jurisdictions. Some patients can receive life-saving treatments, while others are denied due to regional variations in HTA methodologies and budget constraints. 

Avril Daly – the Chief executive of Retina International referred to this phenomenon as a form of “therapeutic apartheid”, highlighting the ethical dilemma of denying treatment based on geography rather than medical need. 

Policy Recommendation: 

HTA agencies should work toward greater international harmonization to reduce disparities in access. This could include: 

• Joint clinical assessments across regulatory regions (e.g., EU-level HTA assessments). 

• Shared pricing and reimbursement models to ensure consistency in valuation. 

• Pooling RWD across borders, accelerating evidence generation. 

Ensuring equitable access to gene therapy is not solely a financial question but a reflection of societal priorities and global health equity. 

Ethical and Societal Considerations in Value Assessment 

HTA is often framed as a technical exercise, but it ultimately reflects societal values. Avril added: 

“If someone falls into the Atlantic Ocean, we spend millions on a rescue operation without hesitation. Why do we struggle to fund gene therapy for a child with a rare disease?” 

This comparison highlights the inconsistencies in how societies allocate healthcare resources, particularly for high-cost, high-impact interventions. If we are willing to fund other expensive public health initiatives (e.g., intensive care, emergency medical services, or military healthcare) without strict cost-effectiveness thresholds, why are curative therapies subjected to different standards? 

Policy Recommendation: 

HTA agencies should consider integrating societal and ethical considerations into their decision-making, including: 

• Citizen panels and deliberative processes to ensure assessments reflect public values. 

• Greater emphasis on non-clinical outcomes, such as improved quality of life, caregiver burden reduction, and societal productivity. 

• Ethical frameworks that assess long-term societal return on investment, beyond immediate healthcare costs. 

Conclusion 

Gene therapy represents one of the most significant medical breakthroughs of our time, yet its full potential is constrained by an outdated system of value assessment and reimbursement. If HTA frameworks are to remain relevant in the era of advanced therapies, they must evolve to: 

1. Adjust discount rates to reflect the transformative impact of curative treatments. 
2. Implement alternative financing models to align cost with long-term value. 
3. Embrace RWE as a critical component of value assessment. 
4. Harmonize global HTA processes to ensure equitable access. 
5. Incorporate ethical considerations into funding decisions. 

The time for incremental reform has passed. The future of healthcare demands a new paradigm—one that ensures medical innovation is not just developed, but accessible to those who need it most. If you need any support or would like to discuss how we can help, please get in touch.