Value of Gene Therapies
THE VALUE OF GENE THERAPIES
Gene therapies are fundamentally different from medicines that are taken over the course of a patient’s entire lifetime to address the symptoms of disease. Today’s new transformational therapies are designed to target the source of many rare diseases at the genetic level often with one lasting dose. These differences make gene therapies ill-suited for the current tools that are used by payers to determine their cost effectiveness.
As more innovative gene therapies progress toward – and achieve – FDA approval, we must bring new thinking to how they are valued.
Getting the value equation right for gene therapies will have a real and tangible impact on patients’ ability to benefit from this game-changing field.
IGT is committed to educating stakeholders about the value of gene therapies, what it looks like to accurately and fully assess their value, and the consequences if we fall short.
To start, we worked with our council members to identify the most important elements of value to patients, the healthcare system, and society.
THE MOST IMPORTANT ELEMENTS OF VALUE TO PATIENTS, THE HEALTHCARE SYSTEM, AND SOCIETY, INCLUDE:
DISEASE STATE FACTORS
Burden on Patient
Addressing an Unmet Medical/Clinical Need/Severity of Disease
Rarity of Disease
Family and Caregiver Burden
Treatment Effects (Short- and Long-Term)
Patient Reported Outcomes
Medical Costs (Direct/Indirect)
Advancing Health Equity/Preventing Discrimination
Goodwill or the Value of Hope (Hope for families and physicians to treat their patients)