Breakthrough Therapy late May 2014 Serious Condition
Just to review what we applied for:
A. Serious Condition
1. Whether a Condition Is Serious
FDA intends to interpret the term serious as it has done in the past for the purposes of accelerated approval and expanded access to investigational drugs for treatment use.6 A serious disease or condition is defined in the expanded access regulations as follows:
. . . a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible if it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.
2. Whether the Drug Is Intended to Treat a Serious Condition
As referenced in section IV, the statutory and regulatory eligibility criteria for expedited programs require that a drug be intended to treat a serious condition. To satisfy this criterion, a drug must be intended to have an effect on a serious condition or a serious aspect of a condition, such as a direct effect on a serious manifestation or symptom of a condition or other intended effects, including the following:
• A diagnostic product intended to improve diagnosis or detection of a serious condition in a way that would lead to improved outcomes
• A product intended to mitigate or prevent a serious treatment-related side effect (e.g., serious infections in patients receiving immunosuppressive therapy)
• A product intended to avoid or diminish a serious adverse event associated with available therapy for a serious condition (e.g., product that is less cardiotoxic than available cancer therapy)8
• A product intended to prevent a serious condition or reduce the likelihood that the condition will progress to a more serious condition...