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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
What is Genetic Engineering?00:49

What is Genetic Engineering?

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Pharmacogenetics and Pharmacogenomics: Overview01:29

Pharmacogenetics and Pharmacogenomics: Overview

Pharmacogenetics and pharmacogenomics examine how genetic factors influence an individual's response to drugs. While pharmacogenetics focuses on the impact of specific genetic variants on drug effects, pharmacogenomics takes a broader approach, studying how genetic variation across populations contributes to differences in drug responses. These fields aim to explain why individuals may experience varying levels of efficacy or adverse reactions to the same medication.Variability in drug...

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Gene therapy and cost-effectiveness analysis: Current issues and future research.

Scott Johnson1, Marric Buessing1, Thomas O'Connell1

  • 1Medicus Economics LLC, Boston, MA, USA.

Med (New York, N.Y.)
|June 4, 2026
PubMed
Summary
This summary is machine-generated.

Gene therapies offer one-time cures for genetic diseases but challenge traditional cost-effectiveness analysis (CEA). New methods are needed to assess their value and ensure patient access.

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Area of Science:

  • Healthcare Economics
  • Biotechnology
  • Genetics

Background:

  • Gene therapies represent a significant advancement in treating genetic diseases, offering potential one-time cures.
  • Traditional cost-effectiveness analysis (CEA) faces challenges in evaluating these novel, high-cost, and durable treatments.

Purpose of the Study:

  • To review the complexities of CEA for gene therapies.
  • To identify challenges and emerging solutions for economic assessment of gene therapies.

Main Methods:

  • Analysis of approved gene therapies and their pricing.
  • Review of US cost-effectiveness analyses (CEAs).
  • Examination of five distinguishing attributes complicating CEA: rarity, one-time administration, durable efficacy, high upfront costs, and financing.

Main Results:

  • Approved gene therapies range from $60,000 to over $4 million.
  • CEAs often find list prices above traditional thresholds but comparable to other advanced therapies.
  • Methodological challenges include limited data, inappropriate utility sources, durability uncertainty, and undervaluing transformative benefits.

Conclusions:

  • Developing appropriate economic assessment methods is crucial for gene therapy access.
  • Innovative financing and evolving value frameworks are emerging solutions.
  • Balancing patient access with innovation incentives requires refined economic evaluation strategies.