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Robert D. Truog, "Charlie Gard and Right-to-Try Laws"


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December 12, 2017

The Sixth Annual Health Law Year in P/Review symposium featured leading experts discussing major developments during 2017 and what to watch out for in 2018. The discussion at this day-long event covered hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

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Robert D. Truog, "Charlie Gard and Right-to-Try Laws"

  1. 1. Robert D. Truog, MD Charlie Gard and Right-to-Try Laws Frances Glessner Lee Professor of Medical Ethics, Anaesthesia, & Pediatrics, Harvard Medical School Director, Center for Bioethics, Harvard Medical School Senior Associate in Critical Care Medicine, Boston Children's Hospital
  2. 2. Charlie Gard • Born with the mitochondrial DNA depletion syndrome • Most common: TK2 mutation – affects only muscles • Charlie: RRM2B – very rare, more severe, causes brain damage Truog RD. The United Kingdom Sets Limits on Experimental Treatments: The Case of Charlie Gard. JAMA 2017;318:1001
  3. 3. Charlie Gard • Age 2 months, admitted to Great Ormond Street Hospital, London (GOSH) • Over several months became ventilator dependent, developed uncontrollable seizures leading to severe brain damage
  4. 4. Dr. Michio Hirano, Columbia University • Uses experimental nucleoside therapy for MDS • Has treated 18 pts with the TK2 defect • Most in Spain, Italy • One American child
  5. 5. Arturito Estopinan • 7 years old, has been treated for 6 years • Has regained some movement of fingers and toes • Still ventilator dependent, requires full care
  6. 6. Request for transfer to New York • Requested transfer to Columbia University • Columbia agreed to provide treatment as compassionate use • Parents raised $1.6 million • The GOSH physicians refused to permit transfer
  7. 7. The Court Battle • The court noted that, under UK law, the case must be decided solely upon the objective best interests of Charlie Gard • GOSH experts: – Only limited evidence of benefit for patients with the TK2 mutation, the mildest form of this disease – No experience with Charlie’s mutation, which is much more severe and with brain involvement – In addition, he had uncontrolled seizures which had resulted in substantial additional brain injury – Argued that his best interests were to have palliative care and withdrawal of life support
  8. 8. The Court Battle • Dr. Hirano – Acknowledged his recommendation was theoretical and based on very limited evidence – He said that the chances of meaningful brain recovery would be small, even “vanishingly small” – He acknowledged that the brain damage was to a large extent, if not entirely, irreversible. – “I would just like to offer what we can. It is unlikely to work, but the alternative is that he will pass away.”
  9. 9. The Court Battle • United Kingdom – High Court, Court of Appeal, and the Supreme Court • European Court of Human Rights • All courts affirmed the initial ruling that withdrawal of life support and palliative care were in Charlie’s best interest
  10. 10. International Attention • Both Pope Francis and President Trump expressed support for the parents • The Vatican offered to accept Charlie “for the time it will take for him to live.”
  11. 11. The end of the story… • His parents finally agreed to withdrawal of life support • He died on July 28, 2017
  12. 12. Right-to-Try Laws
  13. 13. Right-to-Try Laws • Laws that purport to give patients access to experimental therapies that have completed Phase 1 testing but that have not been approved by the FDA • Emotionally compelling – “These are real people facing their mortality with no hope” Senator Ron Johnson, (R) WI • Exceptionally popular – 38 states have enacted such laws (not MA) – FDA Reauthorization Act of 2017
  14. 14. Right-to-Try Laws: Political candy • Do not require physicians to prescribe experimental therapies • Do not require insurance companies to pay for them • Do not require manufacturers to provide them • FDA not an obstacle – 99% of requests approved
  15. 15. “Right-to-Try” is essentially “Right-to-Ask” • Pharma have developed voluntary programs to give patients access to experimental drugs outside of clinical trials – Expanded access programs: requests by groups of persons with the same disease – Compassionate use programs: requests by individuals Darrow JJ, Sarpatwari A, Avorn J, Kesselheim AS. Practical, legal, and ethical issues in expanded access to investigational drugs. N Engl J Med 2015;372:279
  16. 16. Requirements for Janssen Pharmaceuticals • The patient must have a serious or life-threatening condition • The patient must have exhausted all other options • The patient is not eligible for a clinical trial • Sufficient evidence that the benefits of the investigational medicine outweigh the risks • Providing pre-approval access will not jeopardize the overall program to support FDA approval
  17. 17. Compassionate Use - Janssen • Compassionate Use Advisory Committee (CompAC) – Advisory only – Includes physicians, bioethicists, patients, and patient advocates • Patients selected using principles of justice – PRIMARY: not harming patients, needing to exhaust all existing approved treatments, the scientific likelihood of an efficacious response, patient functionality – SECONDARY: prior participation in clinical trials, length of coping with the disease, support for dependents, and patient age. Caplan AL, Ray A. JAMA 2016;315:979-80.
  18. 18. Are there lessons from Charlie Gard for right-to-try laws and compassionate use programs?
  19. 19. Consumerism vs Professional Regulation • Consumerism Model – Charlie’s parents were able and willing to pay for his treatment at Columbia – Dr. Hirano and Columbia were willing to provide the treatment • Most compelling in the context of an isolated relationship between patient and investigator • Less compelling is settings that involve use of communal resources
  20. 20. Professional Regulation: Setting standards of reasonableness • Should the FDA take a more proactive role in setting standards for reasonableness? – FDA not well suited for making highly contextualized judgments – Right-to-try laws may disqualify FDA from this role
  21. 21. Professional Regulation: Setting standards of reasonableness • Should CompACs be made mandatory? – Better suited for contextualized case-by-case decisions – But decisions are only partially patient-centered – compassionate use is denied if it jeopardizes the program supporting drug approval
  22. 22. Charlie Gard: tentative observations • In my view, nucleoside therapy was not a “professionally reasonable” option for Charlie Gard • The GOSH physicians were correct in recommending palliative care
  23. 23. Charlie Gard: tentative observations • The case points to the need for professional oversight of access to experimental therapies – The FDA is not well suited to the task, and may be disqualified by current federal regulations – CompACs are an imperfect solution, given their COI between patient and corporate welfare, but are better than a market solution