Unleashing the Potential of Gene Therapies: Overcoming HTA Challenges

By Staff Writer

October 16, 2023

The Promise and Challenges of Gene Therapies

Gene therapies offer transformational benefits to patients, health systems, and society. However, several challenges hinder timely patient access. A report commissioned by Pfizer, penned by Besley et al. (2022), discusses the difficulties of health technology assessment (HTA) of gene therapies and proposes potential solutions.

 



Gene therapies, as defined by the European Medicines Agency (EMA), are medicines containing genes that lead to therapeutic, prophylactic, or diagnostic effects. They work by inserting ‘recombinant’ genes into the body to treat a variety of diseases, including genetic disorders, cancer, or long-term diseases.

Recommendations for Overcoming HTA Challenges

Based on a review of the literature and insights from an international panel of HTA and health economics experts, the authors propose six overarching recommendations. These recommendations aim to address the challenges in fully capturing the potential value of gene therapies as part of the HTA process and to improve the quality and acceptability of the evidence generated.

 


The recommendations are not specific to gene therapies and should be consistently applied across HTA of other treatments. However, due to the unique challenges presented by the HTA of gene therapies, if implemented, these recommendations are likely to have a larger impact on the assessment of gene therapies.

The report also assesses the extent to which these recommendations are being achieved in selected European countries, Australia, and Canada. It reviews the HTA outcomes of key gene therapies conducted in these countries to provide further understanding of the current state of play.

 

Reference url

Recent Posts

UK Medicine Payment Rates
Data Analysis Drug pricing Europe HTA Innovative medicines and medical devices Innovative Tech 

Revitalizing Innovation: The Impact of UK Medicine Payment Rates

João L. Carapinha

💡 Are the UK’s medicine payment rates stifling innovation in life sciences?

Recent analysis reveals that the staggering 23.5% payment rate for pharmaceuticals is not just limiting NHS spending but also curtailing R&D investments, delaying new drug launches, and threatening workforce stability. If we can lower these rates to below 10%, we could unleash significant growth and restore the UK’s competitive edge in the global life sciences landscape.

Discover the critical insights and recommendations that could reshape the future of healthcare in the UK.

#SyenzaNews #healthcarepolicy #innovation

Blinatumomab benefit assessment
Diseases Europe Health economic evaluations Health Policy High-priced drugs HTA HTA Agencies HTA policies Innovative medicines and medical devices Innovative Tech Oncology and cancer research Rare diseases and orphan drugs 

Blinatumomab Benefit Assessment for ALL and Health Economics

João L. Carapinha

🚀 How does a groundbreaking therapy change the landscape of acute lymphoblastic leukemia treatment?

The recent benefit assessment of Blinatumomab by the German Federal Joint Committee reveals critical insights into its potential as a consolidation therapy for adults with Philadelphia chromosome-negative B-cell precursor ALL. With its orphan drug status promising automatic benefits, the article explores the implications of its high cost and economic impacts, shedding light on the balance needed between innovation and sustainability in healthcare.

Jump into the full analysis to understand how this could impact clinical practice and health economics.

#SyenzaNews #HealthEconomics #MarketAccess

Enhertu reimbursement analysis
Clinical Practice Clinical trials Cost-effectiveness analysis Cost-utility analysis Diseases Drug pricing Europe Evidence-based medicine Health Policy HTA HTA Agencies HTA policies Innovation Innovative medicines and medical devices Innovative Tech Oncology and cancer research Policymakers 

Enhertu Reimbursement Analysis: Navigating Cost-Effectiveness Challenges

João L. Carapinha

💡 How do we balance groundbreaking cancer treatments with budget realities?

The recent analysis by the Dutch Healthcare Institute on Enhertu shines a light on the complexities of integrating innovative therapies for HER2-low metastatic breast cancer into national insurance packages. While the clinical efficacy is evident, the substantial cost implications and demand for steep price reductions bring significant challenges to market access.

Explore the nuances of this vital discussion on clinical effectiveness, economic evaluation, and the future of pricing strategies in oncology.

#SyenzaNews #HealthEconomics #MarketAccess

When you partner with Syenza, it’s like a Nuclear Fusion.

Our expertise are combined with yours, and we contribute clinical expertise and advanced degrees in health policy, health economics, systems analysis, public finance, business, and project management. You’ll also feel our high-impact global and local perspectives with cultural intelligence.
PARTNER WITH US
schedule a meeting

SPEAK WITH US

+1 617 219 9400

CORRESPONDENCE ADDRESS

1950 W. Corporate Way, Suite 95478
Anaheim, CA 92801, USA

WRITE US

inquiry2023@syenza.com

JOIN NEWSLETTER




SERVICES

QUICK LINKS

LINKEDIN
TWITTER
© 2025 Syenza™. All rights reserved.
Made with by Frogiez
schedule a meeting