Why We Need Rigorous Study Designs For Rare Disease Clinical Trials
Finally FDA approved Sarepta’s eteplirsen (Exondys 51), doing this, they actually welcomed the Duchenne muscular dystrophy (DMD0 community. Though it was shrouded with some controversies.
Eteplirsen is a gene therapy drug for administration in DMD patients whose dystrophin mutation is manageable by skipping exon 51.It was approved after a thorough study of 12 patients, 4 of whom, received placebo for 24 weeks before being re-randomized to one of two dosages.There were some brow raising questions regarding the use of historical data as a control in the absence of a concurrent placebo arm of the latter part of the study and the efficacy measures used. But finally, Eteplirsen was given approval by the FDA, and though entire the process was taken over by controversy, demonstrated the power for innovations in designing a clinical trial and analytical methods.
Producing High-Quality Data
After this recent approval, and the essentials for novel clinical research in rare drug development, it might be tempting for sponsors to push boundaries such that their drugs comes to market more fast and at cheaper price. The authorities of Food and Drugs says that owing to all controversies over Eteplirsen they must multiply their efforts to propagate the therapeutic development ecosystem to apply methods that would yield high-quality data from the beginning.
Requirements for FDA Approval
There are certain criteria that need to be fulfilled to get FDA approval. This needs evidence of efficacy and clinical utility of a candidate with well accepted standards and research trial design should portray those needs. Considerable flexibility in evidentiary standards of approval has been noted by other authors in the orphan disease space, suggesting a portfolio of design options that might be possible contingent upon the nature of the product and the therapeutic target. Like other therapeutic areas, endpoints must be clearly defined and time points should be clinically practical. Although the use of historical controls can be considered as a component of the review process (like in cancer drug development), patients must be exactly matched and clinical assessments must be standardized across all contributing trial centres. Statistics must be prospectively designed to account for all time points and dosages, and potential heterogeneity across reference trials. Studies should be planned such that the protocol is elaborate with the endpoints; in simple words, test what you have specified.
Bringing Innovation to Trial Designs
Many ways novel ideas can be introduced to planning trials while maintaining the standards of scientific excellence. When you have to design clinical trials for rare disease, this might include getting the most out of early phase studies. If your access to patients is limited, make sure that every patient enrolled in the study “counts” – that even proof of concept studies are well designed and properly optimized to help lend credence to later phase studies. Innovation can also be introduced in study design, mainly in early phase research, that mixes endpoints to reduce timelines and streamline outcomes while informing the next stages of drug development. A classic instance of this is the mixed SAD/MAD studies that includes algorithm or model based methods of dosage escalation for the purposes of defining an operational dose range for subsequent studies. To include, Worldwide Clinical Trials has been able to use creative designing to shorten the timelines by 4 months.
We conclude the discussion here.
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