Twenty-First Century Global ADR Management: A Need for Clarification, Redesign, and Coordinated Action.

Adverse drug reactions (ADRs) are estimated to be between the fourth and sixth most common cause of death worldwide, taking their place among other prevalent causes of mortality such as heart disease, cancer, and stroke. ADRs impact a broad range of populations across a wide variety of global geography and demographics, with significant mortality and morbidity burden in vulnerable groups such as older people, pediatric populations, and individuals in low-income settings. Too large a share of medicines risk management remains limited to signal detection in big ADR databases (USFDA, EMA, WHO, etc.

Patient Contribution to the Development and Safe Use of Medicines During the Covid-19 Pandemic.

COVID-19 has catapulted the issue of the patient voice in healthcare and healthcare policy to the front of the global agenda. The world population has been affected with varying government-required risk mitigation measures including social distancing, national, regional and local "lock down" quarantines, and the wearing of masks along with diligent handwashing. Clearly, not all of these measures are possible in every country due to a lack of resources and healthcare infrastructure, and it will surely be patients who will suffer the most as a result.

Impact of Real-World Data on Market Authorization, Reimbursement Decision & Price Negotiation.

Real-world data (RWD) and real-world evidence (RWE) appear now as complementary to traditional randomized controlled trials (RCT), that remain the gold standard of the evidence. This review aims to illustrate how health authorities in France, United States (USA) and United Kingdom (UK) can integrate RWD and RWE in market authorization discussions and in new pathways of price and reimbursement negotiations. We conducted a review from the literature, online investigations and interviews.

21st-Century Oncology Drug Safety via New-Age Regulatory Standards and Practices.

There is no such thing as a drug that is 100% safe or effective. Determining whether or not a new oncology treatment (or an additional indication for an existing medicine) should be approved by a regulatory licensing authority is, ultimately, as much regulatory science as public health art and nuance. There are many dynamic shifts in regulatory science (expedited review pathways, biomarker validation, use of real-world evidence, expanded off-label usage, etc) interpreted and expressed within the context of 21st-century oncology drug development, and these new tools and the learnings gleaned from them are helping to advance patient care.

Implications of CRISPR-Based Germline Engineering for Cancer Survivors.

Cancer survivors can carry germline mutations that will be transmitted to their progeny. Today, many of these mutations have been identified and can be tracked. With the recent development of genome-editing technologies and CRISPR (clustered regularly interspaced short palindromic repeats), the possibility of genetically modifying the human germline-gametes and embryos-has never been closer.

From the Valley of Death to the Crossroads of Opportunity: A Discussion of Evolving Benefit/Risk Evaluation Standards.

A series of recent US Food and Drug Administration (FDA) approvals (such as Sarepta's Exondys 51, Merck's Keytruda, and Portola's Bevyxxa) has generated significant interest within the drug development ecosystem. Facilitated regulatory pathways aimed toward expediting medicines to patients suffering from serious and life-threatening conditions are a good thing, even if it raises curiosity and introduces some degree of uncertainty. Over the last 20 years, two key words in drug development have been speed and innovation.

Advancing Drug Safety Through Prospective Pharmacovigilance.

Much has changed in a relatively short period of time. There is a raging debate over the level of evidence expected to first introduce a treatment to patients based on smaller, more adaptive data sets. Some argue for less data followed by postapproval follow-up, others for more adaptive clinical trial designs and end-point modification driven by patient-focused drug development and use of real-world evidence.

Advancing pharmaceuticals and patient safety in Saudi Arabia: A 2030 vision initiative.

Low-quality medicines deliver sub-optimal clinical outcomes and waste precious health resources. It is important to ensure that public funds are spent on healthcare technologies that meet national regulatory bodies such as the Saudi Food and Drug Authority (SFDA), quality standards for safety, efficacy, and quality. Medicines quality is a complicated combination of pre-market regulatory specifications, appropriate sourcing of ingredients (active pharmaceutical ingredient (API), excipients, etc.

Using Off-Label Communications to Responsibly Advance the Public Health.

The debate over off-label communications doesn't begin or end with the Caronia or Amarin decisions. It's a continuing dialogue between manufacturers and the FDA, between doctors and patients, between doctors and academics, between lawyers and judges, and between advocates on all sides. And the red thread that ties these conversations together is responsible off-label communications.

21st century pharmacovigilance: efforts, roles, and responsibilities.

In an era when the number of expedited and conditional review pathways for newly available brand-name drugs and biosimilar medicines to treat serious and life-threatening diseases is increasing, defining pharmacovigilance has never been more crucial. 21st century pharmacovigilance is not merely about uncovering, reporting, and addressing adverse events associated with already approved and marketed agents, but can be described as the systematic monitoring of the process of pre-market review and post-market surveillance, which includes the use of medicines in everyday practice. Pharmacovigilance identifies previously unrecognised adverse events or changes in the patterns of these effects, the quality and adequacy of drug supply, and should ensure effective communication with the public, health-care professionals, and patients about the optimum safety and effective use of medicines.