TY - JOUR AU - Eichler, H. G. AU - Bloechl-Daum, B. AU - Bauer, P. AU - Bretz, F. AU - Brown, J. AU - Hampson, L. V. PY - 2016 DA - 2016// TI - “Threshold-crossing”: a useful way to establish the counterfactual in clinical trials? JO - Clin Pharmacol Ther VL - 100 UR - https://doi.org/10.1002/cpt.515 DO - 10.1002/cpt.515 ID - Eichler2016 ER - TY - STD TI - Beckman RA, Clark J, Chen C. Integrating predictive biomarkers and classifiers into oncology clinical development programmes. Nat Rev Drug Discov [Internet]. 2011[cited 2018 may 2];10(10):735–748. Available from: http://www.ncbi.nlm.nih.gov/pubmed/21959287. Accessed 2 May 2018. UR - http://www.ncbi.nlm.nih.gov/pubmed/21959287 ID - ref2 ER - TY - STD TI - Thomas N. Historical Control. 2008 [cited 2018 Apr 9];1–3. Available from: https://onlinelibrary.wiley.com/doi/pdf/https://doi.org/10.1002/9780471462422.eoct347. ID - ref3 ER - TY - STD TI - Hill AB. THE ENVIRONMENT AND DISEASE: ASSOCIATION OR CAUSATION? Proc R Soc Med [Internet]. 1965 May;58:295–300. Available from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2626428/. Accessed 9 Apr 2018. UR - https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2626428/ ID - ref4 ER - TY - STD TI - Byar DP, Schoenfeld DA, Green SB, Amato DA, Davis R, De Gruttola V, et al. Design considerations for AIDS trials. N Engl J Med [Internet] 1990;323(19):1343–1348. Available from: http://www.nejm.org/doi/abs/https://doi.org/10.1056/NEJM199011083231912. ID - ref5 ER - TY - STD TI - Walton MK. Natural History Studies. 2012 [cited 2018 Apr 9]; Available from: https://events-support.com/Documents/Walton.pdf. Accessed 9 Apr 2018. UR - https://events-support.com/Documents/Walton.pdf ID - ref6 ER - TY - STD TI - Beach ML, Baron J. Bias from Historical Controls. 2005. https://doi.org/10.1002/0470011815.b2a16007. ID - ref7 ER - TY - STD TI - Gehan EA. The evaluation of therapies: Historical control studies. Stat Med [Internet]. 1984 [cited 2018 Apr 10];3(4):315–324. Available from: http://doi.wiley.com/https://doi.org/10.1002/sim.4780030405. ID - ref8 ER - TY - STD TI - Cber; CDRH. Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices - Guidance for Industry and Food and Drug Administration Staff. [cited 2018 Apr 9]; Available from: https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf. Accessed 9 Apr 2018. UR - https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf ID - ref9 ER - TY - STD TI - Sherman RE, Anderson SA, Dal Pan GJ, Gray GW, Gross T, Hunter NL, et al. Real-World Evidence — What Is It and What Can It Tell Us? N Engl J Med [Internet]. 2016[cited 2018 Aug 13];375(23):2293–2297. Available from: http://www.nejm.org/doi/https://doi.org/10.1056/NEJMsb1609216. ID - ref10 ER - TY - STD TI - Salman RA-S, Beller E, Kagan J, Hemminki E, Phillips RS, Savulescu J, et al. Increasing value and reducing waste in biomedical research regulation and management. Lancet [Internet]. 2014[cited 2019 mar 25];383(9912):176–185. Available from: http://www.ncbi.nlm.nih.gov/pubmed/24411646. Accessed 25 Mar 2019. UR - http://www.ncbi.nlm.nih.gov/pubmed/24411646 ID - ref11 ER - TY - STD TI - Smith MA, Joffe S. Will my child do better if she enrolls in a clinical trial? Cancer [Internet]. 2018[cited 2019 mar 25];124(20):3965–3968. Available from: http://www.ncbi.nlm.nih.gov/pubmed/30291807. Accessed 25 Mar 2019. UR - http://www.ncbi.nlm.nih.gov/pubmed/30291807 ID - ref12 ER - TY - STD TI - Clarke M, Loudon K. Effects on patients of their healthcare practitioner’s or institution’s participation in clinical trials: a systematic review. Trials [Internet]. 2011[cited 2019 mar 25];12:16. Available from: http://www.ncbi.nlm.nih.gov/pubmed/21251306. Accessed 25 Mar 2019. UR - http://www.ncbi.nlm.nih.gov/pubmed/21251306 ID - ref13 ER - TY - STD TI - Gliklich RE, Dreyer NA. Registries for Evaluating Patient Outcomes: A User’s Guide [Internet]. Registries for Evaluating Patient Outcomes: A User’s Guide. Agency Healthc Res Qual (US); 2010 [Cited 2019 mar 25]. Available from: http://www.ncbi.nlm.nih.gov/pubmed/21204321. Accessed 25 Mar 2019. UR - http://www.ncbi.nlm.nih.gov/pubmed/21204321 ID - ref14 ER - TY - STD TI - Richesson RL. Data Standards in Diabetes Patient Registries. J Diab Sci Technol [Internet]. 2011[cited 2019 Mar 25];5(3):476–485. Available from: http://journals.sagepub.com/doi/https://doi.org/10.1177/193229681100500302. Accessed 25 Mar 2019. ID - ref15 ER - TY - STD TI - Mitchel J, Laschinger J, Health T, Laschinger J. CTTI Registry Trials Project : Eval Des Registries Conducting Clin Trials. 2017 [cited 2018 Apr 9]; Available from: https://www.nihcollaboratory.org/Pages/GR-Slides-07-21-17.pdf. Accessed 9 Apr 2018. UR - https://www.nihcollaboratory.org/Pages/GR-Slides-07-21-17.pdf ID - ref16 ER - TY - STD TI - Brooke EM. The current and future use of Registeries in Health Information Systems. [cited 2018 Apr 10]; Available from: http://apps.who.int/iris/bitstream/handle/10665/36936/WHO_OFFSET_8.pdf?sequence=1&isAllowed=y. UR - http://apps.who.int/iris/bitstream/handle/10665/36936/WHO_OFFSET_8.pdf?sequence=1&isAllowed=y ID - ref17 ER - TY - STD TI - FDA. Natural History Studies for Rare Diseases [Internet]. Available from: https://www.youtube.com/watch?v=2GDU7f75MVI. Accessed 10 Apr 2018. UR - https://www.youtube.com/watch?v=2GDU7f75MVI ID - ref18 ER - TY - STD TI - Groft SC, de la Paz MP. Rare diseases – avoiding misperceptions and establishing realities: the need for reliable epidemiological data. In: Adv Exp Med Biol [Internet]. 2010 [cited 2018 Apr 10]. p. 3–14. Available from: http://www.ncbi.nlm.nih.gov/pubmed/20824436. Accessed 26 Mar 2019. UR - http://www.ncbi.nlm.nih.gov/pubmed/20824436 ID - ref19 ER - TY - STD TI - FDA, CDER, CBER. Rare Diseases: Common Issues in Drug Development Guidance for Industry DRAFT GUIDANCE. FDA [Internet]. 2019. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-common-issues-drug-developmentguidance-industry-0. UR - https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-common-issues-drug-developmentguidance-industry-0 ID - ref20 ER - TY - STD TI - Chen C, Li N, Shentu Y, Pang L, Beckman RA. Adaptive Informational Design of Confirmatory Phase III Trials With an Uncertain Biomarker Effect to Improve the Probability of Success. Stat Biopharm Res. 2016 [cited 2018 May 2];8(3):237–247. Available from: https://doi.org/10.1080/19466315.2016.1173582. ID - ref21 ER - TY - STD TI - Beckman RA, Chen Cong. Informational Designs and Potential Applications to Rare Diseases. In: "Handbook of Biomarkers and Precision Medicine”, C Carini, M Fidock, & A Van Gool, eds, Chapman & Hall/CRC press, Taylor & Francis Group, Boca Raton, Florida. 2019. p. 183–188. ID - ref22 ER - TY - STD TI - Prakash Bhuyan, Pfizer, Inc.; Chao Chen, GlaxoSmithKline; Jigar Desai, Pfizer I., Christopher Hart, AstraZeneca; Leah Helvering, Eli Lilly; Daniel Jia AJ, Lim, GlaxoSmithKline; Jiandong Lu, Janssen; Jingyi Liu ELSM, Bristol-Myers Squibb; Josephine Wolfram, Astellas; Edward Bowen, Pfizer I. DEVELOPMENT AND IMPLEMENTATION OF A PHARMA-COLLABORATIVE LARGE HISTORICAL CONTROL DATABASE [Internet]. Available from: http://www.transceleratebiopharmainc.com/wp-content/uploads/2015/04/TransCelerate-PSoC-Data-Sharing-White-Paper.pdf. Accessed 9 Apr 2018. UR - http://www.transceleratebiopharmainc.com/wp-content/uploads/2015/04/TransCelerate-PSoC-Data-Sharing-White-Paper.pdf ID - ref23 ER - TY - STD TI - Duke Margolis. Utilizing innovative statistical methods and trial designs in rare disease settings [Internet]. 2018. Available from: https://www.youtube.com/watch?v=Ftp6Zgan_hE&feature=youtu.be. Accessed 9 Apr 2018. UR - https://www.youtube.com/watch?v=Ftp6Zgan_hE&feature=youtu.be ID - ref24 ER - TY - STD TI - CDER; CBER, Fda CC. Guidance for Industry E 10 Choice of Control Group and Related Issues in Clinical Trials Guidance for Industry. Control [Internet]. 2001 [cited 2018 Apr 9];(May):301–827. Available from: http://www.fda.gov/cder/guidance/index.htm. Accessed 9 Apr 2018. UR - http://www.fda.gov/cder/guidance/index.htm ID - ref25 ER - TY - STD TI - European Medicines Agency (EMA). ICH TOPIC E 10 CHOICE OF CONTROL GROUP IN CLINICAL TRIALS. [cited 2018 Apr 9]; Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002925.pdf. Accessed 9 Apr 2018. UR - http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500002925.pdf ID - ref26 ER - TY - JOUR AU - Papageorgiou, S. N. AU - Koretsi, V. AU - Jäger, A. PY - 2017 DA - 2017// TI - Bias from historical control groups used in orthodontic research: a metaepidemiological study JO - Eur J Orthod VL - 39 UR - https://doi.org/10.1093/ejo/cjw035 DO - 10.1093/ejo/cjw035 ID - Papageorgiou2017 ER - TY - STD TI - Sacks H, Chalmers TC SHJ. Randomized versus historical controls for clinical trials. Am J Med [Internet]. 1982[cited 2018 Apr 9];72(2):A51. Available from: http://linkinghub.elsevier.com/retrieve/pii/0002934382908336. Accessed 9 Apr 2018. UR - http://linkinghub.elsevier.com/retrieve/pii/0002934382908336 ID - ref28 ER - TY - STD TI - Pocock SJ. The combination of randomized and historical controls in clinical trials. J Chronic Dis [Internet]. 1976[cited 2018 Apr 9];29(3):175–188. Available from: http://linkinghub.elsevier.com/retrieve/pii/0021968176900448 Accessed 14 Aug 2018. UR - http://linkinghub.elsevier.com/retrieve/pii/0021968176900448 ID - ref29 ER - TY - STD TI - Coo C. CDISC Standards in the Regulatory Submission Process Webinar Agenda. 2012;(January). ID - ref30 ER - TY - STD TI - Beat Neuenschwander. Statistical Issues in Using Historical Data in Clinical Trials. 2012 [cited 2018 Apr 10]; Available from: https://www.researchgate.net/profile/Beat_Neuenschwander/publication/274890005_Statistical_Issues_in_Using_Historical_Data_in_Clinical_Trials/links/552b92170cf2e089a3aa461f/Statistical-Issues-in-Using-Historical-Data-in-Clinical-Trials.pdf. Accessed 9 Apr 2018. UR - https://www.researchgate.net/profile/Beat_Neuenschwander/publication/274890005_Statistical_Issues_in_Using_Historical_Data_in_Clinical_Trials/links/552b92170cf2e089a3aa461f/Statistical-Issues-in-Using-Historical-Data-in-Clinical-Trials.pdf ID - ref31 ER - TY - STD TI - Dejardin D, Delmar P, Patel K, Warne C, Van Rosmalen J, Lesaffre E. Dynamic borrowing of historical data: Performance and comparison of existing methods based on a case study. [cited 2018 Apr 9]; Available from: http://bbs.ceb-institute.org/wp-content/uploads/2017/07/Dejardin-BBS_dynamic_borrowing.pdf. Accessed 26 Mar 2019. UR - http://bbs.ceb-institute.org/wp-content/uploads/2017/07/Dejardin-BBS_dynamic_borrowing.pdf ID - ref32 ER - TY - STD TI - FDA, CDER, CBER. Adaptive Designs for Clinical Trials of Drugs and Biologics Guidance for Industry DRAFT GUIDANCE. Fda [Internet]. 2018;(September). Available from: https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. Accessed 26 Mar 2018. UR - https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm ID - ref33 ER - TY - JOUR AU - Neuenschwander, B. AU - Capkun-Niggli, G. AU - Branson, M. AU - Spiegelhalter, D. J. PY - 2010 DA - 2010// TI - Summarizing historical information on controls in clinical trials JO - Clin Trials VL - 7 UR - https://doi.org/10.1177/1740774509356002 DO - 10.1177/1740774509356002 ID - Neuenschwander2010 ER - TY - STD TI - Gsteiger S, Neuenschwander B. Historical controls in clinical trials : the meta-analytic predictive approach applied to over-dispersed count data. 2012;(May). ID - ref35 ER - TY - STD TI - Neuenschwander B, Schmidli H, Roychoudhury S, Gsteiger S. Robust borrowing from Historical data with Meta-analytic-predictive mixture priors. 2013; Available from: http://www.researchgate.net/profile/Beat_Neuenschwander/publication/274889986_Robust_Borrowing_from_Historical_Data_with_Meta-Analytic-Predictive_Mixture_Priors/links/552b911f0cf29b22c9c1c0c6.pdf. Accessed 23 Oct 2018. UR - http://www.researchgate.net/profile/Beat_Neuenschwander/publication/274889986_Robust_Borrowing_from_Historical_Data_with_Meta-Analytic-Predictive_Mixture_Priors/links/552b911f0cf29b22c9c1c0c6.pdf ID - ref36 ER - TY - JOUR AU - Viele, K. AU - Berry, S. AU - Neuenschwander, B. AU - Amzal, B. AU - Chen, F. AU - Enas, N. PY - 2013 DA - 2013// TI - Use of historical control data for assessing treatment effects in clinical trials JO - Pharm Stat [Internet] VL - 13 UR - https://doi.org/10.1002/pst.1589 DO - 10.1002/pst.1589 ID - Viele2013 ER - TY - STD TI - Neuenschwander B. Meta-Analytic Approaches to Using Historical Data in Clinical Trials. 2014;(June). ID - ref38 ER - TY - STD TI - Neuenschwander B, Wandel S, Roychoudhury S. An Industry Approach to Bayesian Phase I Oncology Trials : Methodology and Implementation. 2015;(May). ID - ref39 ER - TY - STD TI - Neuenschwander B, Roychoudhury S, Branson M. Predictive evidence threshold scaling: does the evidence meet a confirmatory standard? Stat Biopharm Res [Internet] 2017;6315:0–0. Available from: https://www.tandfonline.com/doi/full/https://doi.org/10.1080/19466315.2017.1392892. ID - ref40 ER - TY - STD TI - Swallow E, Signorovitch J, Yuan Y, Kalsekar A. Indirect comparisons for single-arm trials or trials without common comparator arms □ Single-arm trials and trials without common comparators □ Comparison of methods and criteria for evaluation Randomized controlled trials ( RCTs ) are the gold standard –. 2015;1–32. ID - ref41 ER - TY - STD TI - He H, Hu J, He J. Statistical causal inferences and their applications in public Health Research. 2016; Available from: http://link.springer.com/https://doi.org/10.1007/978-3-319-41259-7. ID - ref42 ER - TY - STD TI - Rosenbaum PR, Rubin DB. The Central Role of the Propensity Score in Observational Studies for Causal Effects. Biometrika [Internet]. 1983 [cited 2018 Apr 9];70(1):41–55. Available from: http://links.jstor.org/sici?sici=0006-3444%28198304%2970%3A1%3C41%3ATCROTP%3E2.0.CO%3B2-Q. Accessed 9 Apr 2018. UR - http://links.jstor.org/sici?sici=0006-3444%28198304%2970%3A1%3C41%3ATCROTP%3E2.0.CO%3B2-Q ID - ref43 ER - TY - STD TI - Rosenbaum PR, Rubin DB. Reducing Bias in Observational Studies Using Subclassification on the Propensity Score. J Am Stat Assoc [Internet]. 1984 [cited 2018 Apr 9];79(387):516–524. Available from: http://links.jstor.org/sici?sici=0162-1459%28198409%2979%3A387%3C516%3ARBIOSU%3E2.0.CO%3B2-6. Accessed 9 Apr 2018. UR - http://links.jstor.org/sici?sici=0162-1459%28198409%2979%3A387%3C516%3ARBIOSU%3E2.0.CO%3B2-6 ID - ref44 ER - TY - STD TI - King Richard Nielsen G, Abadie A, Dafoe A, Grimmer J, Hainmueller J, Hazlett C, et al. Why Propensity Scores Should Not Be Used for Matching *. 2016 [cited 2018 Apr 9]; Available from: https://gking.harvard.edu/files/gking/files/psnot.pdf. Accessed 9 Apr 2018. UR - https://gking.harvard.edu/files/gking/files/psnot.pdf ID - ref45 ER - TY - STD TI - Chen Z, Zhao Y, Cui Y, Kowalski J. Methodology and application of adaptive and sequential approaches in contemporary clinical trials. J Probab Stat. 2012;2012. ID - ref46 ER - TY - JOUR AU - Galwey, N. W. PY - 2017 DA - 2017// TI - Supplementation of a clinical trial by historical control data: is the prospect of dynamic borrowing an illusion? JO - Stat Med VL - 36 UR - https://doi.org/10.1002/sim.7180 DO - 10.1002/sim.7180 ID - Galwey2017 ER - TY - STD TI - Thabane L, Mbuagbaw L, Zhang S, Samaan Z, Marcucci M, Ye C, et al. A tutorial on sensitivity analyses in clinical trials: the what, why, when and how. BMC Med Res Methodol [Internet]. 2013;13(1):92 Available from: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=3720188&tool=pmcentrez&rendertype=abstract. Accessed 23 Oct 2018. UR - http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=3720188&tool=pmcentrez&rendertype=abstract ID - ref48 ER - TY - JOUR AU - Greenland, S. PY - 1996 DA - 1996// TI - Basic methods for sensitivity analysis of bias JO - Int J Epidemiol VL - 25 UR - https://doi.org/10.1093/ije/25.6.1107 DO - 10.1093/ije/25.6.1107 ID - Greenland1996 ER - TY - STD TI - Medicines Agency E. Reflection paper on extrapolation of efficacy and safety in. 2016 [cited 2018 Apr 9]; Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2016/04/WC500204187.pdf. Accessed 9 Apr 2018. UR - http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2016/04/WC500204187.pdf ID - ref50 ER - TY - STD TI - Bonate PL. Clinical Trial Simulation in Drug Development. Pharm Res [Internet]. 2000 [cited 2018 Apr 9];17(3):252–256. Available from: http://link.springer.com/https://doi.org/10.1023/A:1007548719885. ID - ref51 ER - TY - STD TI - Mayer C, Perevoskaya I, Leonov S, Dragalin V, Pritchett Y, Bedding A, et al. Simulation Practices For Adaptive Trial Designs In Drug And Device Development. Stat Biopharm Res [Internet]. 2019[cited 2019 Mar 26];1–20. Available from: https://www.tandfonline.com/doi/full/https://doi.org/10.1080/19466315.2018.1560359. ID - ref52 ER - TY - STD TI - Dejardin D, Delmar P, Warne C, Patel K, van Rosmalen J, Lesaffre E. Use of a historical control group in a noninferiority trial assessing a new antibacterial treatment: A case study and discussion of practical implementation aspects. Pharm Stat [Internet]. 2018[cited 2018 Apr 9];17(2):169–181. Available from: http://www.ncbi.nlm.nih.gov/pubmed/29282862. Accessed 9 Apr 2018. UR - http://www.ncbi.nlm.nih.gov/pubmed/29282862 ID - ref53 ER - TY - STD TI - Viele K. The use of Historical information in clinical trials. 2014;. ID - ref54 ER - TY - STD TI - Rothman KJ, Greenland S, Lash TL, editors. Modern epidemiology. Lippincott Williams & Wilkins; 2008. ID - ref55 ER - TY - STD TI - Dempster AP, Selwyn MR, Weeks BJ. Combining Historical and Randomized Controls for Assessing Trends in Proportions. J Am Stat Assoc [Internet]. 1983[cited 2018 Apr 10];78(382):221–227. Available from: http://www.tandfonline.com/doi/abs/https://doi.org/10.1080/01621459.1983.10477953. ID - ref56 ER - TY - STD TI - Ibrahim JG, Ryan LM, Chen M-H. Using Historical Controls to Adjust for Covariates in Trend Tests for Binary Data. J Am Stat Assoc [Internet]. 1998 [cited 2018 Apr 10];93(444):1282–1293. Available from: http://www.tandfonline.com/doi/abs/https://doi.org/10.1080/01621459.1998.10473789. ID - ref57 ER - TY - STD TI - Tamura RN, Young SS. The incorporation of historical control information in tests of proportions: simulation study of Tarone’s procedure. Biometrics [Internet]. 1986 [cited 2018 Apr 10];42(2):343–349. Available from: http://www.ncbi.nlm.nih.gov/pubmed/3755626. Accessed 10 Apr 2018. UR - http://www.ncbi.nlm.nih.gov/pubmed/3755626 ID - ref58 ER - TY - STD TI - Tarone RE. The Use of Historical Control Information in Testing for a Trend in Proportions. Biometrics [Internet]. 1982[cited 2018 Apr 10];38(1):215. Available from: https://www.jstor.org/stable/2530304?origin=crossref Accessed 10 Apr 2018. UR - https://www.jstor.org/stable/2530304?origin=crossref ID - ref59 ER - TY - STD TI - Cook TD, Campbell DT. Quasi-Experimentation: Design and Analysis Issues for Field Settings [Internet]. Houghton Mifflin; 1979 [cited 2018 Apr 10]. Available from: https://www.scholars.northwestern.edu/en/publications/quasi-experimentation-design-and-analysis-issues-for-field-settin. Accessed 10 Apr 2018. UR - https://www.scholars.northwestern.edu/en/publications/quasi-experimentation-design-and-analysis-issues-for-field-settin ID - ref60 ER - TY - STD TI - Rosenbaum PR. Observational Studies. Springer Series in Statistics. New York: Springer; 2002. ID - ref61 ER - TY - STD TI - Keiding N. Historical Controls in Survival Analysis. In: Encyclopedia of Biostatistics [Internet]. Chichester, UK: John Wiley & Sons, Ltd; 2005 [cited 2018 Apr 9]. Available from: http://doi.wiley.com/https://doi.org/10.1002/0470011815.b2a11036. ID - ref62 ER - TY - STD TI - U.S Food and Drug Administration. Real World Evidence Program. 2018;(February). ID - ref63 ER - TY - STD TI - Priya Kishnani. Lessons Learned from Pompe Disease [Internet]. [cited 2018 Apr 9]. Available from: https://events-support.com/Documents/Summary-NHS.pdf. Accessed 9 Apr 2018. UR - https://events-support.com/Documents/Summary-NHS.pdf ID - ref64 ER - TY - STD TI - Moscicki R. A Retrospective Natural History Study in Fabry Disease: Challenges and Uses. [cited 2018 Apr 9]; Available from: https://events-support.com/Documents/Moscicki.pdf. Accessed 9 Apr 2018. UR - https://events-support.com/Documents/Moscicki.pdf ID - ref65 ER - TY - STD TI - van Rosmalen J, Dejardin D, van Norden Y, Löwenberg B, Lesaffre E. Including historical data in the analysis of clinical trials: is it worth the effort? Stat methods med res; 2017. ID - ref66 ER -