Framework for “N-of-1” Experimental Therapies

To the Editor: The editorial by Woodcock and Marks (Oct. 24 issue)¹ on the use of a personalized antisense oligonucleotide to treat a child with Batten’s disease is important, not least because of right-to-try legislation in both the United States and the United Kingdom.^2,3 More than a decade ago, recognizing the increased use of “N-of-1” therapies and the lack of governance of these therapies, we proposed a framework⁴ and developed the type of process Woodcock and Marks suggested, in order to support both clinicians and patients and to accompany any other regulatory authorization. Clinicians, scientists, parents, and indeed children meet . . .