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  • Misconceptions about translation — defined as the process of turning observations in the laboratory, clinic and community into interventions that improve the health of individuals — hinder efforts to close gaps and address challenges related to the translational process. This article highlights some misconceptions with the aim of improving understanding and advancing solutions.

    • Christopher P. Austin
  • Providing support for medicine development by academics and clinical researchers is a priority for the European Medicines Agency. The recent launch of free scientific advice to academia and non-profit research organizations developing orphan medicines can help align research with regulatory requirements early during medicine development and enhance the chances of success.

    • Rosa Gonzalez-Quevedo
    • Constantinos Ziogas
    • Anthony Humphreys
  • Greater investment is needed in antibiotic R&D, and more must be done to maximize the impact of such investments. More widespread data sharing, such as the recent joint data contribution from Merck and Kyorin to the Pew Charitable Trusts’ SPARK platform, has a key role.

    • Wes Kim
    • Kevin Krause
    • Kevin Outterson
  • 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.

    • Rod MacKenzie
    • Peter Honig
    • Marie-Pierre Hellio
  • 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.

    • Ruthie Davi
    • Nirosha Mahendraratnam
    • Rachel Sherman
  • 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.

    • Andrew S. Robertson
    • Alexis Reisin Miller
    • Felipe Dolz
  • 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.

    • Francesca Cerreta
    • Armin Ritzhaupt
    • Spiros Vamvakas
  • 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.

    • Peter B. Simpson
    • Graeme F. Wilkinson
  • 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.

    • Giulio Superti-Furga
    • Daniel Lackner
    • Claire M. Steppan
  • 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.

    • Lorna Ewart
    • Adrian Roth
  • 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.

    • Guangdi Li
    • Erik De Clercq
  • 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.

    • Seth Ness
    • Gahan Pandina
    • Husseini K. Manji
  • 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.

    • Kathryn R. Wagner
    • Annamaria De Luca
    • Volker Straub
  • 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.

    • Andrew S. Robertson
    • Hilary Malone
    • Max Wegner
  • 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.

    • Melissa Haendel
    • Nicole Vasilevsky
    • Tudor I. Oprea
  • 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.

    • Maria Mavris
    • Andrea Furia Helms
    • Nathalie Bere