Description
Foreword, ix Acknowledgement, xi Author’s note on the cover design, xiii Introduction, xv 1 What is off-label medication, and how prevalent is it?, 1 What is ‘off-label’ medicine?, 2 Scope of the issue, 8 2 Where it all went right: new uses for existing drugs supported by good evidence, 19 Examples where products have been through regulatory approval for a secondary use, 19 Finasteride: pseudohermaphroditism and hair growth, 19 Sildenafil: re-tasking the blue pill for a life-threatening illness, 21 Doxycycline: from killing bugs to protecting gums, 22 Raloxifene: from cancer to bone disease and back again, 23 Galantamine: using snowdrops to improve memory, 24 Cyclosporine: preventing immune attack on organs and skin, 25 Dimethyl fumarate: a remarkable drug for multiple sclerosis, 26 Botox: a drug to kill or cure you, 27 Examples where evidence is uncertain and not to regulatory standards, 28 Tricyclic antidepressants: for curing more than emotional pain, 28 Aspirin for cancer, 30 Retrospective data: looking back to create future therapies, 30 3 Shared decision making and consent, 33 Viewpoint of the patient, 34 Viewpoint of the prescriber, 37 Professional guidelines, 38 Patient awareness, 41 Practitioner attitudes, 41 Diagnosis, 44 4 Gaming the system: the role of the pharmaceutical industry, 47 Normal drug development and drug repurposing development, 48 Gaming the system, 53 Orphan use, 56 Pharmaceutical marketing, 61 Expanding uses for non-pharmaceuticals, 64 DTC advertising, 64 Patents and genericisation, 65 Conclusion, 68 5 Do no harm: Safety and efficacy, 71 Relative safety, 73 Different therapeutic uses, 73 Chronic versus acute dosing, 78 Different dose, 81 Differences between children and adults, 82 Other patient populations, 86 Fatal ADRs, 87 Quality of evidence, 88 Strong evidence, 89 Poor evidence, 91 Doctors do not know evidence, 94 Proximity of off-label to on-label, 96 Debunking medical myths, 101 6 Liability, injustice and reimbursement: who should pay?, 105 A prescriber’s ethical and professional duties, 105 Medical professional participation in off-label promotion, 105 A prescriber’s legal position, 106 Consent, 106 Liability, 111 Reimbursement, 113 Compendia, 115 NICE, 117 Compassionate access, 120 Cost, as a driver for off-label medicine, 121 7 The role of regulation in off-label medicine, 125 Regulators do not regulate medical practice, 126 Off-label marketing, 128 Off-label fines, 130 Whistle-blowers, 134 European situation, 134 Tip of the iceberg, 136 Free speech, 138 8 Justifying unapproved medicine, 143 Constraints on making changes, 144 Moves to enhance off-label medicine, 145 Diagnosis shifting, 146 A partial solution: clinical trial transparency, 147 A solution based on increased regulatory supervision, 152 My solutions, 153 Professional standards, 153 Reimbursement and pricing, 156 Outcomes, 159 Conclusion, 173 References, 175 Index, 191
