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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.  

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As more innovative gene therapies progress toward – and achieve – FDA approval, we must bring new thinking to how they are valued. 

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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.

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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.

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To start, we worked with our council members to identify the most important elements of value to patients, the healthcare system, and society.

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THE MOST IMPORTANT ELEMENTS OF VALUE TO PATIENTS, THE HEALTHCARE SYSTEM, AND SOCIETY, INCLUDE:

DISEASE STATE FACTORS

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Burden on Patient

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Addressing an Unmet Medical or Clinical Need

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Severity and Heterogeneity of Disease

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Rarity of Disease

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Family and Caregiver Burden

TREATMENT FACTORS

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Short-term and Long-term Treatment Effects

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Lifetime Impact and Durability

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Patient Reported Outcomes

SYSTEM IMPACT

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Direct and Indirect Medical Costs

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Decreased Hospitalizations

SOCIETAL IMPACT

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Advancing Health Equity

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Goodwill & the Value of Hope

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Giving Patients and Families More Time Together

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Extending Patients’ Ability to Contribute to Communities

We believe the following principles are critical to any assessment of a gene therapy’s value:

  1. No public payer or private sector organization should place a dollar value on a patient’s quality of life, health, and worth.
     

  2. One-size-fits-all value assessments are ineffective and discriminatory.
     

  3. The patient, family, and caregiver perspective and preferences must be given priority.
     

  4. Determinations of value may be subject to change based upon evolving real-world evidence.
     

  5. Value assessments should adapt and reflect the available evidence for rare and ultra-rare disease treatments.
     

  6. Determinations of value should uphold FDA’s scientific authority over drug approvals.
     

  7. Value is just one component of a comprehensive and complex coverage decision process that prioritizes patient’s medical needs and available treatment options.
     

  8. An informed healthcare assessment will incorporate the vital elements of value across the four element domains.

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