Darrow JJ, Avorn J, Kesselheim AS. The FDA breakthrough-drug designation - Four years of experience. N Engl J Med 2018;378:16 de abril. [Ref.ID 102515]

Anónimo. Cannabinoid-based drug improved symptoms in adults with Tourette syndrome in phase 2 trial. DIA Daily 2018;10 de abril:3. [Ref.ID 102508]

Mershon E. STAT Plus: It’s not over: After defeat on ‘right to try,’ GOP vows to renew push. STAT Daily Recap 2018:2. [Ref.ID 102468]

Harrington M, Sigal EV. Opinion: ‘Right-to-try’ law threatens patient safety and rational drug development. STAT Daily Recap 2018:6. [Ref.ID 102467]

Ott PA, Hu Z, Keskin DB, Shukla SA, Sun J, Bozym DJ, Zhang W, Luoma A, Giobbie-Hurder A, Peter L, Chen C, Olive O, Carter TA, Li S, Lieb DJ, Eisenhaure T, Gjini E, Stevens J, Lane WJ, Javeri I, Nellaiappan K, Salazar AM, Daley H, Seaman M, Buchbinder EI, Yoon Ch, Harden M, Lennon N, Gabriel S, Rodig S, Barouch DH, Aster JC, Getz G, Wucherpfennig K, Neuberg D, Ritz J, Lander ES, Fritsch EF, Hacohen N, Wu CJ. An immunogenic personal neoantigen vaccine for patients with melanoma. Nature 2017;547:217-21. [Ref.ID 101662]

Ataga KI, Kutlar A, Kanter J, Liles D, Cancado R, Friedrisch J, Gualandro S, Colella MP, Smith WR, Rollins SA, Stocker JW, Rother RP. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med 2017;37:429-39. [Ref.ID 101410]

Anónimo. Vanucizumab fails to meet primary endpoint of progression-free survival for metastatic colorectal cancer patients. DIA Daily 2016:3. [Ref.ID 100838]

Anónimo. Kite Pharma’s experimental cancer therapy shows encouraging results, but doubts remain. DIA Daily 2016:7. [Ref.ID 100762]

Margulies KB, Hernández AF, Redfield MM, Givertz MM, Oliveira GH, Cole R, Mann DL, Whellan DJ, Kieman MS, Felker M, McNulty SE, Anstrom KJ, Shah MR, Braunwald E, Cappola TP, for the NHLBI Heart Failure Clinical Research Network. Effects of liraglutide on clinical stability among patients with advanced heart failure and reduced ejection fraction: a randomized clinical trial. JAMA 2016;316:500-8. [Ref.ID 100686]

Dal-Ré R. Desarrollo clínico de nuevos medicamentos. Seguridad de los ensayos clínicos en voluntarios sanos. Med Clin (Barc) 2016;147:154-6. [Ref.ID 100648]

Mayor S. “Impressive results” in stem cell treatment for multiple sclerosis. BMJ 2016;353:i3269. [Ref.ID 100429]

Lonial S, Weiss BM, Usmani SZ, Singhal S, Chari A, Bahlis NJ, Belch A, Krishnan A, Vescio RA, Mateos MV, Mazumder A, Orlowski RZ, Sutherland HJ, Bladé J, Scott EC, Oriol A, Berdeja J, Gharibo M, Stevens DA, LeBlanc R, Sebag M, Callander N, Jakubowiak A, White D, de la Rubia J, Richardson PG, Lisby S, Feng H, Uhlar CM, Khan I, Ahmadi T, Voorhees PM. Daratumumab monotherapy in patients with treatment-refractory multiple myeloma (SIRIUS): an open-label, randomised, phase 2 trial. Lancet 2016;387:1551-60. [Ref.ID 100228]

Rajkumar SV. Daratumumab in multiple myleoma. Lancet 2016;387:1490-2. [Ref.ID 100220]

Eichler H-G, Hurts H, Broich K, Rasi G. Drug regulation and pricing — Can regulators influence affordability?. N Engl J Med 2016;374:1807-9. [Ref.ID 100113]

Thaçi D, Simpson EL, Beck LA, Bieber T, Blauvelt A, Papp K, Soong W, Worm M, Szepietowski JC, Sofen H, Kawashima M, Wu R, Weinstein SP, Graham NMH, Pirozzi G, Teper A, Sutherland ER, Mastey V, Stahl N, Yancopoulos GD, Ardeleanu M. Efficacy and safety of dupilumab in adults with moderate-to-severe atopic dermatitis inadequately controlled by topical treatments: a randomised, placebo-controlled, dose-ranging phase 2b trial. Lancet 2016;387:40-52. [Ref.ID 99988]

Baerlocher GM, Leibundgut EO, Ottmann OG, Spitzer G, Odenike O, McDevitt MA, Röth A, Daskalakis M, Burington B, Stuart M, Synder DS. Telomerase inhibitor imeteelstat in patients with essential thrombocythemia. N Engl J Med 2015;373:920-8. [Ref.ID 99517]

Abdulqawi R, Dockry R, Holt K, Layton G, McCarthy BG, Ford AP, Smith JA. P2X3 receptor antagonist (AF-219) in refractory chronic cough: a randomised, double-blind, placebo-controlled phase 2 study. Lancet 2015;385:1198-205. [Ref.ID 98993]

Sulkowski M, Hezode C, Gerstoft J, Vierling JM, Mallolas J, Pol S, Kugelmas M, Murillo A, Weis N, Nahass R, Shibolet O, Serfaty L, Bourliere M, DeJesus E, Zuckerman E, Dutko F, Shaughnessy M, Hwang P, Howe AYM, Wahl J, Robertson M, Barr E, Haber B. Efficacy and safety of 8 weeks versus 12 weeks of treatment with grazoprevir (MK-5172) and elbasvir (MK-8742) with or without ribavirin in patients with hepatitis C virus genotype 1 mono-infection and HIV/hepatitis C virus co-infection (C-WORTHY): a randomised, open-label phase 2 trial. Lancet 2015;385:1087-97. [Ref.ID 98985]

Neuschwander-Tetri BA, Loomba R, Sanyal AJ, Lavine JE, Van Natta ML, Abdelmalek MF, Chalasani N, Dasarathy S, Diehl AM, Hameed B, Kowdley KV, McCullough A, Terrault N, Clark JM, Tonascia J, Brunt EM, Kleiner DE, Doo E, for the NASH Clinical Research Network. Farnesoid X nuclear receptor ligand obeticholic acid for non-cirrhotic, non-alcoholic steatohepatitis (FLINT): a multicentre, randomised, placebo-controlled trial. Lancet 2015;385:956-65. [Ref.ID 98959]

Dyer O. Experimental drug that injured UK volunteers resumes in human trials. BMJ 2015;350:h1831. [Ref.ID 98941]