Correspondence

BH, NJD, and MV declare no competing interests. African region and Turkey but around Novartis, PTC Therapeutics, Synedgen (Synspira),
BH has received funding from the University of 75% in northern Europe.2 By contrast, Vertex Pharmaceuticals, Vivus (Janssen), and Actelion
Sydney–Yonsei University Partnership Collaboration (Johnson & Johnson), for which his institution
Awards. NJD receives doctoral funding from the Naji in the USA, Schrijver and colleagues3 (University of Alabama at Birmingham, Birmingham,
Foundation, grant funding from the Fetzer Franklin reported that F508del homozygosity AL, USA) received payment; consulting fees from
Fund, and has been employed on grants from the occurs in 50% of non-Hispanic white, Bayer, Celtaxys, Novartis, Renovion, Synedgen
Good Thinking Society and the Laura and John (Synspira), and Vertex Pharmaceuticals; fees for
Arnold Foundation. MV’s work is funded by the
27% of Hispanic, 19% of black, and advisory board participation for Vertex
Office of Research Integrity of the US Department of 21% of Asian people with cystic Pharmaceuticals; and honoraria from Genentech and
Health and Human Services and by the Oklahoma fibrosis. In our study, only five of Boeringher Ingelheim. JW reports grants from
Center for the Advancement of Science and AbbVie, Allergan, Concert, Janssen, Proteostasis,
Technology.
107 participants (4·7%) reported being and Vertex Pharmaceuticals, for which his institution
Hispanic or Latino (three of them (New York Medical College, New York, NY, USA)
Bennett Holman, *Nicholas J DeVito, participated in the control group and received payment; and non-financial support from
Matt Vassar two in the active treatment group). Vertex Pharmaceuticals.
[email protected]
The inclusion of people with cystic *Karen S McCoy, Harry Heijerman,
Underwood International College Yonsei fibrosis of racial and ethnic minorities Jennifer L Taylor-Cousar, David Waltz,
University, Seoul, South Korea (BH); Faculty
of Humanities, University of Johannesburg, in our trial is consistent with the trend Patrick R Sosnay, Bonnie W Ramsey,
Johannesburg, South Africa (BH); Nuffield seen in other cystic fibrosis studies4 Steven Rowe, John Welter
Department of Primary Care Health Sciences, and suggests that efforts to increase [email protected]
University of Oxford, Oxford OX2 6GG, UK (NJD);
and Department of Psychiatry and Behavioral trial enrolment of under-represented Department of Pediatrics, Nationwide Children’s
Hospital, Ohio State University, Columbus,
Sciences, Oklahoma State University Center for minority individuals with cystic fibrosis
OH 43210, USA (KSM); Department of Pulmonology,
Health Sciences, Tulsa, OK, USA (MV) should be a goal for the cystic fibrosis University Medical Center Utrecht, Utrecht,
1 Heijerman HGM, McKone EF, Downey DG, et al. community. Netherlands (HH); Department of Medicine and
Efficacy and safety of the elexacaftor plus Pediatrics, National Jewish Health, Denver, CO, USA
tezacaftor plus ivacaftor combination regimen As new clinical trials of cystic
(JLT-C); Vertex Pharmaceuticals, Boston, MA, USA
in people with cystic fibrosis homozygous for fibrosis transmembrane conductance (DW, PRS); Department of Pediatrics, Seattle
the F508del mutation: a double-blind,
randomised, phase 3 trial. Lancet 2019;
modulators for people with cystic Children’s Hospital, University of Washington School
394: 1940–48. fibrosis are expected to address cystic of Medicine, Seattle, WA, USA (BWR); Division of
Pulmonary, Allergy, and Critical Care Medicine,
2 Centers for Medicare and Medicaid Services. fibrosis caused by ultra-rare mutations, University of Alabama at Birmingham, Birmingham,
Open payments. 2019. https://www.cms.gov/
openpayments/ (accessed Nov 14, 2019). future studies should enrol a greater AL, USA (SR); and Division of Pediatric Pulmonology,
3 The International Committee of Medical proportion of minoritised people with Allergy, Immunology, and Sleep Medicine, New York
Journal Editors. Conflicts of Interest. Medical College, New York, NY, USA (JW)
cystic fibrosis.
http://www.icmje.org/conflicts-of-interest/ 1 Heijerman HGM, McKone EF, Downey DG, et al.
(accessed Nov 14, 2019). Bennett Holman and colleagues Efficacy and safety of the elexacaftor plus
noted that disclosure statements for tezacaftor plus ivacaftor combination regimen
Authors’ reply some of the authors1 were inconsistent in people with cystic fibrosis homozygous for
the F508del mutation: a double-blind,
For Open Payments see https:// We appreciate the opportunity to with information available on Open randomised, phase 3 trial. Lancet 2019;
openpaymentsdata.cms.gov/ respond to Meghan McGarry’s queries Payments. We recognise that it is 394: 1940–48.
2 Bobadilla JL, Macek M Jr, Fine JP, Farrell PM.
about our study.1 the responsibility of each author to Cystic fibrosis: a worldwide analysis of CFTR
We agree with McGarry that it provide a complete and accurate mutations—correlation with incidence data
would be optimal if the distribution reporting of potential competing and application to screening. Hum Mutat
2002; 19: 575–606.
of race and ethnicity in clinical trials interests, in alignment with the 3 Schrijver I, Pique L, Graham S, Pearl M,
For the International in people with cystic fibrosis reflected International Committee of Medical Cherry A, Kharrazi M. The spectrum of CFTR
Committeee of Medical variants in non-white cystic fibrosis patients.
the overall cystic fibrosis population. Journal Editors’ recommendations. The Implications for molecular diagnostic testing.
Journal Editors’ guidance see
http://www.icmje.org/ Our trial was designed to evaluate authors have rectified their disclosures J Mol Diagn 2016; 18: 39–50.
recommendations/ the effect of the elexacaftor plus in the corrected Article. In all cases, 4 McGarry ME, McColley SA. Minorities are
underrepresented in clinical trials of
tezacaftor plus ivacaftor combination these omissions were unintentional. pharmaceutical agents for cystic fibrosis.
regimen in people with cystic fibrosis KSM reports research grants from AbbVie, Alcresta, Ann Am Thorac Soc 2016; 13: 1721–25.
who are homozygous for the cystic Allergan, Corbus, Novoteris, Gilead, Janssen, Laurent,
Novoteris, Proteostasis, Savara, Translate Bio, Vertex,
fibrosis transmembrane conductance and Vivus for which her institution (Nationwide
regulator mutation. The prevalence Children’s Hospital, Ohio State University, Columbus,
of this mutation is much higher in OH, USA) received payment; and non-financial
support from Chiesi. BWR reports research grants
people originating from northern
from Vertex, for which her institution (Seattle
Europe than in people from other Children’s Hospital, University of Washington School
regions. of Medicine, Seattle, WA, USA) received payment;
For instance, the prevalence of this non-financial support from Novartis; and honoraria
from Vertex. SR reports research grants from
mutation among people with cystic AstraZeneca, Bayer, Celtaxys, Eloxx, Forest Research
fibrosis is only 15–25% in the north Institute, Galapagos (AbbVie), N30 (Nivalis),

602 www.thelancet.com Vol 396 August 29, 2020