Pharmacy Benefits Glossary
A guide to general pharmacy benefits terminology.
What Is Gene Therapy?
Gene therapy is a unique approach to treating or preventing disease by correcting the underlying genetic disease at the source. Gene therapy treats these diseases by altering a person’s genetic makeup in place of drugs or surgical options aimed at reducing the effects of disease. Some diseases gene therapy treats are:
- Inherited retinal diseases
- Spinal muscular atrophy (SMA)
- Hemophilia
- Sickle cell disease
Gene therapy medications are medical breakthroughs used to prevent or cure complex diseases by modifying the patient’s genes. It can provide long-term, potentially permanent therapeutic solutions through several processes, including:
- Replacing a disease-causing gene with a healthy gene
- Adding new copies of broken genes
- Deactivating disease-causing genes
There continue to be many unknowns in the field, but promising results of ongoing clinical trials suggest there will be increasing long-term benefits for a broader range of previously incurable diseases.
Challenges and Considerations in Gene Therapy
Gene therapies are highly experimental, even with the early years of promising results from clinical trials. The following are some of the primary challenges in the field.
Safety concerns
Gene therapy comes with serious health risks, like toxicity, inflammation, and even cancer. With newer, more experimental forms of genetic therapies like CRISPR, little is known about long-term effects, and since some techniques deal with altering one’s DNA, there are concerns about the implications for future generations and inheritable traits.
We don’t know the full outcomes of gene therapy, and the unknown outcomes mean a therapy could pass an unintended or unknowable consequence down to future generations.
Cost
One of the greatest challenges for gene therapy is the cost of treatment. As a point of reference, the five most expensive FDA-approved gene therapies cost between $425,000 and $3.5 million.
The issue of cost isn’t just about the impact it has on those suffering from chronic disease. It goes to the heart of socioeconomic disparities and general access to potential cures for society. Lawmakers and science organizations in the private sector are seeking solutions to bring costs down and make therapy more accessible. But, much like the clinical trials, it’s a long, slow process.
Ethical Considerations
The concern over passing down unknown genetic traits to future generations, particularly through germline therapy (modifying egg and sperm cells), opens up ethical concerns regarding lack of consent. Future generations could end up paying for medical decisions they weren’t able to be a part of.
What Role Do Regulators Play Regarding Gene Therapy
The FDA is the primary regulatory body for gene therapy oversight and approval. Regulators help ensure the safety and effectiveness of gene therapies at multiple stages of research and development (R&D). This includes the following roles:
- Clinical trial oversight
- Adherence to ethical guidelines
- Manufacturing standards
- Tracking adverse events and long-term risks
In addition to the FDA’s role, the National Institutes of Health (NIH) provides safety guidelines for clinical trial protocols. The NIH Recombinant DNA Advisory Committee (RAC) deals with
medical, ethical, and safety issues with newer forms of genetic therapies. An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) are also involved in safety and oversight.
Future Trends
The future of gene therapy involves continued clinical trials for known techniques that have shown positive results for many years, while researchers learn from trials to improve efficacy, introducing breakthroughs in gene editing that may broaden the range of diseases that can be prevented or cured.
Discussions within the science community and with lawmakers will define ways to measure risk and ethical concerns with the potential for reducing, if not eradicating, fatal diseases. This may lead to future generations being spared from these conditions. The true question of where genetic therapies are headed lies in finding solutions to make them more widely affordable.
The future of gene therapy largely involves continuing to extend the science beyond monogenic diseases to cure or prevent complex multifactorial disorders.
Gene therapies will continue to evolve for many years to come. Radical changes and innovations like these are just some of the many factors that impact the complexity of managing data throughout the healthcare industry.
