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 or Clinical Need
Severity and Heterogeneity of Disease
Rarity of Disease
Family and Caregiver Burden
Short-term and Long-term Treatment Effects
Lifetime Impact and Durability
Patient Reported Outcomes
Direct and Indirect Medical Costs
Advancing Health Equity
Goodwill & the Value of Hope
Giving Patients and Families More Time Together
Extending Patients’ Ability to Contribute to Communities
We believe the following principles are critical to any assessment of a gene therapy’s value:
No public payer or private sector organization should place a dollar value on a patient’s quality of life, health, and worth.
One-size-fits-all value assessments are ineffective and discriminatory.
The patient, family, and caregiver perspective and preferences must be given priority.
Determinations of value may be subject to change based upon evolving real-world evidence.
Value assessments should adapt and reflect the available evidence for rare and ultra-rare disease treatments.
Determinations of value should uphold FDA’s scientific authority over drug approvals.
Value is just one component of a comprehensive and complex coverage decision process that prioritizes patient’s medical needs and available treatment options.
An informed healthcare assessment will incorporate the vital elements of value across the four element domains.