Unrealized potential of drug repositioning in Europe during COVID-19 and beyond: a physician’s perspective

References

• Ban TA. The role of serendipity in drug discovery. Dialogues Clin Neurosci. 2006;8(3):335–344.
   PubMedGoogle Scholar
• Mulder CJ, van Asseldonk DP, de Boer NK. Drug rediscovery to prevent off-label prescription reduces health care costs: the case of tioguanine in the Netherlands. J Gastrointestin Liver Dis. 2014;23(2):123–125.
   Web of Science ®Google Scholar
• Allen B. Thalidomide regains respectability as new benefits are discovered. Pharmaceutical J. 2001;267.
   Google Scholar
• Sheskin J. Thalidomide in the treatment of lepra reactions. Clin Pharmacol Ther. 1965;6:303–306.
   PubMed Web of Science ®Google Scholar
• Iyer CG, Languillon J, Ramanujam K, et al. WHO co-ordinated short-term double-blind trial with thalidomide in the treatment of acute lepra reactions in male lepromatous patients. Bull World Health Organ. 1971;45(6):719–732.
   PubMed Web of Science ®Google Scholar
• Reyes-Teran G, Sierra-Madero JG, Martinez del Cerro V, et al. Effects of thalidomide on HIV-associated wasting syndrome: a randomized, double-blind, placebo-controlled clinical trial. AIDS (London, England). 1996;10(13):1501–1507.
   Web of Science ®Google Scholar
• Rehman W, Arfons LM, Lazarus HM. The rise, fall and subsequent triumph of thalidomide: lessons learned in drug development. Ther Adv Hematol. 2011;2(5):291–308.
   PubMedGoogle Scholar
• D’Amato RJ, Loughnan MS, Flynn E, Folkman J. Thalidomide is an inhibitor of angiogenesis. Proc Natl Acad Sci U S A. 1994;91(9):4082–4085.
   PubMed Web of Science ®Google Scholar
• Kenyon BM, Browne F, D’Amato RJ. Effects of thalidomide and related metabolites in a mouse corneal model of neovascularization. Exp Eye Res. 1997;64(6):971–978.
   PubMed Web of Science ®Google Scholar
• Ito T, Ando H, Suzuki T, et al. Identification of a primary target of thalidomide teratogenicity. Science. 2010;327(5971):1345–1350.
   PubMed Web of Science ®Google Scholar
• Kumar S, Witzig TE, Timm M, et al. Expression of VEGF and its receptors by myeloma cells. Leukemia. 2003;17(10):2025–2031.
   PubMed Web of Science ®Google Scholar
• Hussein MA, Bolejack V, Zonder JA, et al. Phase II study of thalidomide plus dexamethasone induction followed by tandem melphalan-based autotransplantation and thalidomide-plus-prednisone maintenance for untreated multiple myeloma: a southwest oncology group trial (S0204). J Clin Oncol. 2009;27(21):3510–3517.
   PubMed Web of Science ®Google Scholar
• Weber D, Rankin K, Gavino M, Delasalle K, Alexanian R. Thalidomide alone or with dexamethasone for previously untreated multiple myeloma. J Clin Oncol. 2003;21(1):16–19.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency - EU/3/01/067 https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu301067. Accessed 31 May 2020.
   Google Scholar
• Simsek M, de Boer NKH, Mulder CJJ. [Continued development of drugs: the path of thioguanine]. Nederlands tijdschrift voor geneeskunde. 2018;162:D1757.
   Google Scholar
• Bayoumy AB, Simsek M, Seinen ML, et al. The continuous rediscovery and the benefit-risk ratio of thioguanine, a comprehensive review. Expert Opin Drug Metab Toxicol. 2020:1–13.
   Google Scholar
• Dubinsky MC, Hassard PV, Seidman EG, et al. An open-label pilot study using thioguanine as a therapeutic alternative in Crohn’s disease patients resistant to 6-mercaptopurine therapy. Inflamm Bowel Dis. 2001;7(3):181–189.
   PubMed Web of Science ®Google Scholar
• Simsek M, Meijer B, van Bodegraven AA, de Boer NKH, Mulder CJJ. Finding hidden treasures in old drugs: the challenges and importance of licensing generics. Drug discovery today. 2018;23(1):17–21.
   PubMed Web of Science ®Google Scholar
• Simsek M, Lissenberg-Witte BI, van Riswijk MLM, et al. Off-label prescriptions of drugs used for the treatment of Crohn’s disease or ulcerative colitis. Aliment Pharmacol Ther. 2019;49(10):1293–1300.
   Web of Science ®Google Scholar
• Dresser R, Frader J. Off-label prescribing: a call for heightened professional and government oversight. J Law Med Ethics. 2009;37(3):476–486 396.
   PubMed Web of Science ®Google Scholar
• Eguale T, Buckeridge DL, Winslade NE, Benedetti A, Hanley JA, Tamblyn R. Drug, patient, and physician characteristics associated with off-label prescribing in primary care. Arch Intern Med. 2012;172(10):781–788.
   Google Scholar
• Wittich CM, Burkle CM, Lanier WL. Ten common questions (and their answers) about off-label drug use. Mayo Clin Proc. 2012;87(10):982–990.
   PubMed Web of Science ®Google Scholar
• Breckenridge A, Jacob R. Overcoming the legal and regulatory barriers to drug repurposing. Nat Rev Drug Discov. 2019;18(1):1–2.
   Web of Science ®Google Scholar
• Kumar Kakkar A, Dahiya N. The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space. Drug Dev Res. 2014;75(4):231–234.
   Web of Science ®Google Scholar
• Moliner AM, Waligora J. The European Union policy in the field of rare diseases. Advances in experimental medicine and biology. 2017;1031:561–587.
   PubMed Web of Science ®Google Scholar
• Luzzatto L, Hyry HI, Schieppati A, et al. Outrageous prices of orphan drugs: a call for collaboration. Lancet. 2018;392(10149):791–794.
   PubMed Web of Science ®Google Scholar
• Simoens S, Cassiman D, Dooms M, Picavet E. Orphan drugs for rare diseases: is it time to revisit their special market access status? Drugs. 2012;72(11):1437–1443.
   Web of Science ®Google Scholar
• The Lancet. The balancing act of orphan drug pricing. Lancet. 2017;390(10113):2606.
   Web of Science ®Google Scholar
• Daniel MG, Pawlik TM, Fader AN, Esnaola NF, Makary MA. The orphan drug act: restoring the mission to rare diseases. Am J Clin Oncol. 2016;39(2):210–213.
   Web of Science ®Google Scholar
• Jayasundara K, Hollis A, Krahn M, Mamdani M, Hoch JS, Grootendorst P. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet journal of rare diseases. 2019;14(1):12.
   PubMed Web of Science ®Google Scholar
• Radboud UMC. Radboud UMC concerned about drug accessbility for patients with rare neuromuscular disease (in Dutch). https://www.radboudumc.nl/nieuws/2019/radboudumc-bezorgd-over-toegankelijkheid-medicijn. Published 2019. Accessed 17 November 2019.
   Google Scholar
• Dooms M, Carvalho M. Compounded medication for patients with rare diseases. Orphanet J Rare Dis. 2018;13(1):1.
   Google Scholar
• Scheepers HP, Langedijk J, Neerup Handlos V, Walser S, Schutjens MH, Neef C. Legislation on the preparation of medicinal products in European pharmacies and the Council of Europe Resolution. Eur J Hosp Pharm. 2017;24(4):224–229
   Google Scholar
• James O, Cullen J, Bouchier IA. Chenodeoxycholic acid therapy for gallstones: effectiveness, toxicity and influence on bile acid metabolism. Q J Med. 1975;44(174):349–367.
   Google Scholar
• Berginer VM, Salen G, Shefer S. Long-term treatment of cerebrotendinous xanthomatosis with chenodeoxycholic acid. N Engl J Med. 1984;311(26):1649–1652.
   PubMed Web of Science ®Google Scholar
• Stokel-Walker C. If a medicine is too expensive, should a hospital make its own? https://mosaicscience.com/story/price-essential-orphan-drug-self-compounding-pharmacy-leadiant-amsterdam-umc-ctx-cdca/. Published 2019. Accessed 17 November 2019.
   Google Scholar
• Dorleijn DMJ, Luijsterburg PAJ, Reijman M, et al. Intramuscular glucocorticoid injection versus placebo injection in hip osteoarthritis: a 12-week blinded randomised controlled trial. Ann Rheum Dis. 2018;77(6):875–882.
   Web of Science ®Google Scholar
• Verrips A, Dotti MT, Mignarri A, Stelten BML, Verma S, Federico A. The safety and effectiveness of chenodeoxycholic acid treatment in patients with cerebrotendinous xanthomatosis: two retrospective cohort studies. Neurological Sciences. 2020;41(4):943–949
   Web of Science ®Google Scholar
• Sheldon T. Dutch hospital makes own drug for rare condition after manufacturer hikes price to euro170 000. Bmj. 2018;361:k2103
   Google Scholar
• Zorginstituut Nederland. GVS-advies chenodeoxycholzuur (Chenodeoxycholic acid Leadiant®) bij de behandeling van cerebrotendineuze xanthomatose (CTX). https://www.zorginstituutnederland.nl/publicaties/adviezen/2018/11/22/gvs-advies-chenodeoxycholzuur-chenodeoxycholic-acid-leadiant-bij-de-behandeling-van-cerebrotendineuze-xanthomatose-ctx. Published 2018. Accessed 21-07-2019.
   Google Scholar
• Liu Z, Jiang Z, Fang H, et al. Perception, price and preference: consumption and protection of wild animals used in traditional medicine. PLoS One. 2016;11(3):e0145901.
   Web of Science ®Google Scholar
• Serafin MB, Bottega A, Foletto VS, da Rosa TF, Hörner A, Hörner R. Drug repositioning is an alternative for the treatment of coronavirus COVID-19. Int J Antimicrob Agents. 2020;105969.
   Google Scholar
• Gordon DE, Jang GM, Bouhaddou M, et al. A SARS-CoV-2 protein interaction map reveals targets for drug repurposing. Nature. 2020.
   Google Scholar
• Harrison C. Coronavirus puts drug repurposing on the fast track. Nat Biotechnol. 2020;38(4):379–381
   PubMed Web of Science ®Google Scholar
• Beigel JH, Tomashek KM, Dodd LE, et al. Remdesivir for the treatment of Covid-19 — preliminary report. New England Journal of Medicine. 2020.
   Google Scholar
• United States Food & Drug Administration - Remdesivir EUA Letter of Authorization. https://www.fda.gov/media/137564/download. Published 01 May 2020. Accessed 06 May 2020.
   Google Scholar
• Scavone C, Brusco S, Bertini M, et al. Current pharmacological treatments for COVID-19: what’s next? Br Int J Antimicrob Agents. 2020;n/a(n/a).
   Google Scholar
• Ayman F, Ping W, Mahmoud A, Hesham S. Identification of FDA approved drugs targeting COVID-19 virus by structure-based drug repositioning. 2020.
   Google Scholar
• Rosa SGV, Santos WC. Clinical trials on drug repositioning for COVID-19 treatment. Rev Panam Salud Publica. 2020;44:e40.
   PubMed Web of Science ®Google Scholar
• Lythgoe MP, Middleton P. Ongoing clinical trials for the management of the COVID-19 pandemic. Trends Pharmacol Sci. 2020:S0165-6147(0120)30070-30075.
   Google Scholar
• Ciliberto G, Mancini R, Paggi MG. Drug repurposing against COVID-19: focus on anticancer agents. Journal of Experimental & Clinical Cancer Research. 2020;39(1):86.
   Web of Science ®Google Scholar
• European Medicines Agency - EU actions to support availability of medicines during COVID-19 pandemic – update #4. https://www.ema.europa.eu/en/news/eu-actions-support-availability-medicines-during-covid-19-pandemic-update-4. Published 2020. Accessed 06 May 2020.
   Google Scholar
• Bahrke J. European Commission - Coronavirus: commission launches data sharing platform for researchers. https://ec.europa.eu/commission/presscorner/detail/en/IP_20_680. Published 2020. Accessed 06 June 2020.
   Google Scholar