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Clinical Efficacy Testing for Orally Inhaled Drugs Robert J. Meyer, MD Acting Director, DPDP

Clinical Efficacy Testing for Orally Inhaled Drugs Robert J. Meyer, MD Acting Director, DPDP. Introduction. Observations About Inhaled Drugs Efficacy Determination for Inhaled Pulmonary Products Safety Determination for Inhaled Pulmonary Products. Clinically Characterizing Inhaled Drugs.

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Clinical Efficacy Testing for Orally Inhaled Drugs Robert J. Meyer, MD Acting Director, DPDP

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  1. Clinical Efficacy Testing for Orally Inhaled Drugs Robert J. Meyer, MD Acting Director, DPDP

  2. Introduction • Observations About Inhaled Drugs • Efficacy Determination for Inhaled Pulmonary Products • Safety Determination for Inhaled Pulmonary Products

  3. Clinically Characterizing Inhaled Drugs • Intended Site of Action • Locally Acting vs. Systemic • Locally Acting: • Mechanism of Action • Extent of Systemic Absorption • Consequences of Systemic Absorption

  4. Clinically Characterizing Inhaled Drugs • For Locally Acting, Orally Inhaled Drugs: • Delivery to site of action cannot be inferred from serum pharmacokinetics • Clinical Study Design Depend on Drug Class / Action • Local safety (I.e., respiratory tract safety) cannot be inferred from PK • Systemic safety may be approached by PK studies (depending on reference)

  5. Clinically Characterizing Inhaled Drugs Patient Device Drug

  6. Clinically Comparing Inhaled Drugs • Assuring “equivalent” delivery characteristics for efficacy does not fully address safety • Assuring “equivalent” systemic delivery characteristic does not address relative efficacy • In-vitro data, Scintigraphy data, PK data all useful in developing drugs, but not definitive for clinical characterization

  7. Efficacy Testing of Inhaled Drugs Bronchodilators • Direct Measures of Action • Serial Spirometry (bronchodilation) • Protection against bronchoprovocation • Peak Flow Rate Assessments • Supportive measures • Symptoms and signs assessments

  8. Efficacy Testing of Inhaled Drugs Corticosteroids • Direct Measures of Action • None • Exploratory - eNO, airway inflammatory markers • Indirect Measures of Action • Serial Trough Spirometry • Exacerbation rates / assessment of control • symptoms / rescue use • Protection against bronchoprovocation

  9. Efficacy Testing of Inhaled Drugs Others - Cromolyn • Direct Measures of Action • ??? • Indirect measures of Action • “trough” PFTs over time • Symptoms / rescue use • Protection against bronchoprovocation • EIB, AMP,,…

  10. Safety Testing of Inhaled Drugs • Tolerability of Drug Product • Excipient issues • Local safety • Systemic safety • PK useful, when possible and when well characterized reference exists • If PK cannot be done, PD and AE assessments

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