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Welcome to our FiresideSTATS Podcast Episode 2. Today we are joined by our Statistical experts, Niccolo and Emily.
Welcome to our FiresideSTATS Podcast Episode 2. Today we are joined by our Statistical experts, Niccolo and Emily.
In 1992, the Food and Drug Administration (FDA) introduced four distinct and successful approaches known as Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track approval of the investigational drug. Under the ‘accelerated approval’ regulation, surrogate endpoints for serious conditions that filled an unmet medical need are reviewed by FDA, allowing faster approval of drugs. A two-tiered system of review times – Standard Review and Priority Review were introduced by FDA under the Prescription Drug User Act (PDUFA). FDA reviews ‘Priority Review’ designated drugs within 6 months (compared to 10 months under standard review).[i]
The leading cause of death in patients with coronavirus disease 2019 (COVID-19), caused by the virus ‘severe acute respiratory syndrome coronavirus 2’ (SARS‑CoV‑2), is respiratory failure from acute respiratory distress syndrome.[1] Patients who required invasive mechanical ventilation had an 88% mortality rate in one study in New York, and a 53% mortality rate in one study in the UK.[2][3] While an alarming proportion of patients with respiratory failure are dying, for the those who recover there is an urgent need to consider, and subsequently manage, their longer-term care.[4] Patients who have survived the COVID‑19 infection continue to experience feelings of fatigue, shortness of breath and reduced exercise tolerance. There is currently limited information regarding the best route to full recovery for post-coronavirus infection patients. Care will likely be multidisciplinary in nature and include a respiratory review along with physiotherapy, nutritional advice, psychiatric support, and potentially other disciplinary involvement.
There are two main areas in medical writing, medical communications and regulatory writing. This blog focuses on regulatory writing, which involves the preparation of clinical study and regulatory submission documentation.
This article has been updated in May 2020 to reflect the recent guidance by European Medicines Agency and other regulatory bodies on remote monitoring due to the recent coronavirus disease (COVID-19) pandemic.
Within the last few decades the number and complexity of clinical trials has increased considerably, not only across the industry but also within individual companies. With this increase comes the enhanced pressure of effectively monitoring these trials.
The Clinical Data Interchange Standards Consortium (CDISC) creates standards that is now mandatory for the regulatory submission to the FDA and PMDA. Study Data Tabulation Model (SDTM) is one of the standards which provides a standard for streamlined data in collection, management, analysis and reporting. If your data is not in SDTM standards then a pharmaceutical company or Clinical Research Organizations (CRO) will regard SDTM mapping to the latest version of SDTM standards for regulatory submission.
The most common design used for Phase II and Phase III clinical trials is the randomized controlled trial design. In randomized controlled trials, the probability of being assigned the study treatment or the control treatment is fixed throughout the trial and is normally 50%, so that each treatment is given to the same number of patients. This approach leads to a high‑chance of identifying if one treatment is significantly better than the other. The fact that 50% of the trial population is assigned to the control treatment is not a particular issue in common diseases such as cardiovascular disease. For example, of the 7 million people in the United Kingdom (UK) with cardiovascular disease, if 200 of them are involved a clinical trial, then 99.9% of the overall cardiovascular disease population will benefit from the results of the clinical trial[1].
The imminent application of Clinical Trial Regulation (EU) No. 536/2014, estimated to happen in 2020, will peak an emergent era of transparency in clinical development. The regulation applies to all interventional clinical trials (Phase 1 to 4) performed in the European Union (EU)/European Economic Area (EEA), plus additionally specified paediatric trials, and covers documents at the single clinical trial level irrespective of the drug’s marketing approval status. The regulation requires the disclosure of the study Protocol, Investigational Medicinal Product Dossier (IMPD; Sections S and E), Investigator’s Brochure (IB), Subject Information Sheet, Clinical Study Report (CSR; redacted) and a Plain Language Summary (PLS; also known as a Laypersons Summary) for each clinical trial within 12 months of the end of the trial (6 months for paediatric trials). Disclosure is readily welcomed by study participants who want to know what has been learned from their participation and is in accordance with the European Medicines Agency’s (EMA) goal of protecting and fostering public health by allowing better‑informed use of medicines. We will be able to learn from past successes as well as failures now that any biased under‑reporting of results will be stopped.
As the novel coronavirus pandemic sweeps across the globe, the media coverage of the COVID-19 disease is vast. There are articles being published continuously on all types of websites, and by journalists, across multiple disciplines. The aim of this blog is to pull together a list of trusted, valuable, statistically sound, and scientifically robust resources on COVID-19 for data scientists, statisticians, clinical programmers, medical writers and other individuals involved in drug development within the pharmaceutical and biotechnology industry or beyond.
With constantly evolving industry regulations, the increasing pressures to reduce the costs of drug development, and raising demand for new therapies, trends in clinical trial outsourcing strategies emerge.
