Comment

Digital health technologies have the potential to help address some of the challenges in the clinical development of drugs for central nervous system disorders. This article discusses strategies for the development of such tools in the context of the European regulatory environment.

The response to the COVID-19 pandemic has shown that exceptional efforts can dramatically accelerate the clinical development of vaccines. We propose that it is time to also take immediate actions to improve clinical trials in other areas to better serve all patients.

Randomized controlled trials are the accepted standard for evaluating investigational therapies, but such trials are sometimes not an option for reasons of ethics or feasibility. Here, we discuss opportunities to address evidence gaps by using historical clinical trial data and real-world data in external control arms for single-arm trials, as well as the associated challenges.

Drug developers are increasingly applying data-driven analysis of the actions of regulatory agencies to gain insights into their expectations and applications of regulatory policy, but such strategies can be limited by the availability and quality of regulatory datasets. Here, we discuss how establishing a single, robust, accessible database of FDA regulatory actions could help address this limitation.

Regulatory agencies can provide advice to support developers of digital technologies for medicines use, but what are the best strategies to maximize the chance of a successful regulatory interaction? Here, EMA and industry representatives comment on the experience so far.

Consortia are enabling drug discovery in areas that individual organizations are unable to support alone because of the high risk or the need to pool information. This article discusses desirable features that can underpin the success of such consortia.

The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework.

The Innovative Medicines Initiative Consortium RESOLUTE has started to develop tools and produce data sets to de-orphanize transporters in the solute carrier protein (SLC) superfamily, thereby lowering the barrier for the scientific community to explore SLCs as an attractive drug target class.

After 3 years of co-design with stakeholders and EU regulatory partners, the European Medicines Agency’s Regulatory Science to 2025 strategy has been finalized. Here, we highlight the core recommendations that stakeholders deem most significant to advance evidence generation.

Using human-relevant, translational in vitro models is widely considered to reduce attrition during drug discovery and development. Despite this, the adoption of models based on microphysiological systems — organs-on-chips or organoids — by pharma companies is moderate at best, and realizing the full potential of these models will need greater collaboration between stakeholders.

Therapeutic options in response to the 2019-nCoV outbreak are urgently needed. Here, we discuss the potential for repurposing existing antiviral agents to treat 2019-nCoV infection (now known as COVID-19), some of which are already moving into clinical trials.

Numerous potential therapeutic targets are being investigated in autism spectrum disorder (ASD). Here, we discuss a platform trial approach for designing proof-of-concept (POC) clinical studies of ASD — via the Autism Spectrum POC Initiative (ASPI) — that can be conducted through a public–private partnership with the aim of finding effective treatments in the most expeditious manner.

This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.

Cloud-based systems for data submitted by sponsors to drug regulatory agencies have the potential to accelerate drug development, streamline regulatory review and enhance regulatory decision-making. Here, we provide a vision for integrating cloud-enabled practices in drug regulation globally.

A lack of robust knowledge of the number of rare diseases and the number of people affected by them limits the development of approaches to ameliorate the substantial cumulative burden of rare diseases. Here, we call for coordinated efforts to more precisely define rare diseases.

The European Medicines Agency and the US Food and Drug Administration have committed to engaging patients in their regulatory processes to promote patient-focused medicinal product development, as well as improve transparency and trust in the regulatory system. Here, we highlight exchanges of experience between the agencies and some impacts on patient engagement.

Lack of financial and human capital continues to hinder the development of successful biotechnology hubs. Venture philanthropy could be an important part of the solution.

Real-world evidence is increasingly valued by regulators and payers. Central to this evidence base is patient-reported outcome data describing the impact of drugs on quality of life, daily activities and symptoms. Here, we highlight key challenges with current real-world, patient-reported outcome data and describe collaborative next steps for international stakeholders to overcome these issues.

It is increasingly important that regulatory agencies catalyse the translation of scientific and technological innovation into improved patient-centred health care. Here, we highlight the strategic goals of the European Medicines Agency to advance regulatory science and optimize the opportunities arising in areas such as cell and gene therapies, big data and artificial intelligence.

One aspect of the ongoing debate about drug pricing is the added therapeutic benefit of new drugs compared with existing — and potentially cheaper — therapies. Here, we discuss the merits and pitfalls of proposals that are being discussed with regard to the role of regulatory agencies in establishing added therapeutic benefit.