To ensure better consistency in reported outcomes in trials in the same clinical area, core outcome sets (COS) have been developed. 2004–002508-13, the mechanism of randomisation was not described and changes in the protocol from the original version were not specified. In a Major Setback to the Healthcare System, Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic. 2006–001246-13 did not provide specific information on the masking of participants. Visit GlobalData Store, Enhancing Oral Formulations of Large and Polar APIs, Rubber Parts for Primary Packaging and Medical Devices Solutions, Laboratory Testing and Project Management for Clinical Trials, 14 January 2020 (Last Updated January 14th, 2020 16:13). Because of the missing clear justification for using LOCF [4], the risk of bias due to missing outcome data was assessed as with ‘some concerns’. Spark Therapeutics, Pfizer, and UniQure all have gene therapy products in Phase III development. If approved, BioMarin’s candidate will be the first gene therapy product to ever become marketed in the entire haemophilia market, including both haemophilia A and B. Haemophilia A and B are congenital, genetic X-linked deficiencies in the blood clotting factor VIII (FVIII) and clotting factor IX (FIX), respectively. In this domain, RCTs were reviewed for risk of bias that arose because of selection of the reported results [4]. This domain focuses mainly on differential errors related to intervention assignment. Challenges in conducting CT with ATMP are also posed by their biological nature and specific manufacturing process. A review of available COS for the clinical field of analysed RCTs during the period they were conducted was performed as well as a comparison between trial reports and their protocols concerning consistency in planned and published outcome measures and analyses. Pre-Clinical. The authors report no conflict of interest. There was no consensus reached on optimal phase II endpoints in acute or chronic heart failure trials [16] and study no. The approval of valoctocogene roxaparvovec will greatly contribute to BioMarin’s profits and further growth. The three-year follow-up of the Phase I/II clinical trial showed positive results, with the median number of annualized treated bleeding events at zero and a reduction of the median use of exogenous factor VIII from 138.5 infusions to 0 infusions per year, in 7 out of 15 participants. We use cookies to ensure that we give you the best experience on our website. Registered in England & Wales No. The definition of the CT as double-blind was likely considered by the trialists as sufficient, with no need for additional data regarding of outcome assessors masking. Gene therapies are anticipated to address the significant unmet need for therapies that can reduce the number of weekly prophylactic infusions and alleviate some of this treatment burden. In three of the reviewed RCTs, blinding was described only with the broad term ‘double blind’. We use cookies to improve your website experience. Not for Distribution. The CT data were analysed using the predetermined methods of analysis. In all reviewed RCTs, in both intervention and placebo groups the size of the number of participants with missing outcome data and reasons for this were similar. 2011–004761-33, was designed after extensive study of the published CF RCTs [14]. One of the limitations of the current review is that only a small number of RCTs were identified in our search. The COS for peripheral arterial disease was developed in 2018 [18]. The relevant data were extracted once the database was locked and the researchers were unblinded [14]. According to the Global Gene Therapy Clinical Trials Registry [23], 2686 CTs were conducted in a shorter time period than the current systematic review (1 January 1995–31 December 2018), which is a far greater number than that found in the current search. Review DMD Gene Therapy Presentation at ASGCT. To learn about our use of cookies and how you can manage your cookie settings, please see our Cookie Policy. The use of a statistical tool would be beneficial for drawing empirical evidence for how the presence of bias in different domains influences the estimation of the intervention effect. 2006–001246-13, the primary efficacy and safety analyses were done in the ITT population comprising all 732 patients enrolled in the CT. In two of the RCTs (study nos. In study no. 2004–002508-13, the methods of outcome measurement were very specific and the same measurement methods and thresholds in both the intervention and placebo groups were used at comparable time points. 2004–002508-13 were similar, which suggest low risk of bias. US KOLs interviewed by GlobalData consider the time to market a deciding factor for the success of a product and agree that a first-in-market drug can achieve higher market shares than its contenders. Scott Gottlieb, the former FDA commissioner, predicted that by the year 2025, the US will be approving between 10 and 20 different gene therapies every year. Gene therapies are expected to capture 7.5% of the market in the US and 18.6% of the market in the 5EU by 2028. Bias due to the choice of reported results this domain use of and. A reason for dropout of patients [ 19–21 ] happy with it the... To apply a random patient distribution sequence was not described and changes in the CT protocol on! 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