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An evidence map of randomised controlled trials evaluating genetic therapies

Eric A Apaydin1,2, Andrea S Richardson3, Sangita Baxi4

  • 1Southern California Evidence-based Practice Center, Health Care, RAND Corporation, Santa Monica, California, USA eapaydin@rand.org.

BMJ Evidence-Based Medicine
|November 11, 2020
PubMed
Abstract

Insights

Genetic therapies show promise for treating diseases, with most improving symptoms. However, serious adverse events are common, and long-term effectiveness data is limited, necessitating further research.

Area of Science:

  • Genetics and Genomics
  • Clinical Trials
  • Therapeutics

Background:

  • Genetic therapies offer potential cures by targeting disease-causing genes.
  • Unlike traditional treatments, genetic therapies aim for a one-time application rather than symptom management.
  • Randomized controlled trials (RCTs) are crucial for evaluating the efficacy and safety of these novel interventions.

Purpose of the Study:

  • To provide a comprehensive overview of genetic therapies evaluated in RCTs.
  • To map the existing evidence on the safety and efficacy of genetic interventions.
  • To identify gaps in research regarding long-term outcomes of genetic therapies.

Main Methods:

  • Systematic search of multiple databases (PubMed, EMBASE, Web of Science) and clinical trial registries.
  • Inclusion of only RCTs to minimize bias in safety and efficacy assessments.
  • Data abstraction on study characteristics, safety, and efficacy outcomes, visualized in an evidence map.

Main Results:

  • 119 RCTs on genetic therapies for diverse conditions were identified.
  • Adenovirus vectors were common (40%), primarily for cardiovascular diseases (29%).
  • While 60% of trials showed symptom improvement, nearly half (45%) reported serious adverse events; long-term improvement data was sparse (19 trials).

Conclusions:

  • Genetic therapies demonstrate potential for symptom improvement across various diseases.
  • The co-occurrence of symptom improvement and serious adverse events highlights a complex risk-benefit profile.
  • Significant gaps exist in evidence for long-term effectiveness and safety, impacting clinical adoption and ethical considerations.
Keywords:
evidence mapgeneticstherapeutics

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