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Developing standards for breakthrough therapy designation in oncology.

ABSTRACT: In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.

SUBMITTER: Horning SJ

PROVIDER: S-EPMC3745545 | biostudies-literature | 2013 Aug

REPOSITORIES: biostudies-literature

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Developing standards for breakthrough therapy designation in oncology.

Horning Sandra J SJ Haber Daniel A DA Selig Wendy K D WK Ivy S Percy SP Roberts Samantha A SA Allen Jeff D JD Sigal Ellen V EV Sawyers Charles L CL

Clinical cancer research : an official journal of the American Association for Cancer Research 20130529 16

In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor wo ...[more]

PMID: 23719260

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Project description:BackgroundEffective standardisation of methodologies to analyse the microbiome is essential to the entire microbiome community. Despite the microbiome field being established for over a decade, there are no accredited or certified reference materials available to the wider community. In this study, we describe the development of the first reference reagents produced by the National Institute for Biological Standards and Control (NIBSC) for microbiome analysis by next-generation sequencing. These can act as global working standards and will be evaluated as candidate World Health Organization International Reference Reagents.ResultsWe developed the NIBSC DNA reference reagents Gut-Mix-RR and Gut-HiLo-RR and a four-measure framework for evaluation of bioinformatics tool and pipeline bias. Using these reagents and reporting system, we performed an independent evaluation of a variety of bioinformatics tools by analysing shotgun sequencing and 16S rRNA sequencing data generated from the Gut-Mix-RR and Gut-HiLo-RR. We demonstrate that key measures of microbiome health, such as diversity estimates, are largely inflated by the majority of bioinformatics tools. Across all tested tools, biases were present, with a clear trade-off occurring between sensitivity and the relative abundance of false positives in the final dataset. Using commercially available mock communities, we investigated how the composition of reference reagents may impact benchmarking studies. Reporting measures consistently changed when the same bioinformatics tools were used on different community compositions. This was influenced by both community complexity and taxonomy of species present. Both NIBSC reference reagents, which consisted of gut commensal species, proved to be the most challenging for the majority of bioinformatics tools tested. Going forward, we recommend the field uses site-specific reagents of a high complexity to ensure pipeline benchmarking is fit for purpose.ConclusionsIf a consensus of acceptable levels of error can be agreed on, widespread adoption of these reference reagents will standardise downstream gut microbiome analyses. We propose to do this through a large open-invite collaborative study for multiple laboratories in 2020. Video Abstract.

Project description:BACKGROUND: There is a lack of effective and affordable technologies to address health needs in the developing world. One way to address problems of innovation and affordability is to design global health technologies to follow agreed-upon standards. This Debate article argues that we can better develop standards for global health technologies if we learn lessons from other industries. DISCUSSION: The article's Background section begins by explaining why standards are needed in global health. For example, if global health technologies can be modularized into independent interfacing parts, these parts can then interact via well-defined standards in a "plug and play" fashion. This can avoid development of mutually incompatible solutions by different organizations, speed the pace of innovation, unlock health systems from single providers and approaches, and lower barriers to entry. The Background then gives a brief primer on standards and discusses incentives for health standards. The article's Discussion section begins with brief relevant cases of standards development from other industries, including electricity, container shipping, CD standards, Universal Serial Bus (USB), and the Internet. It then explores lessons from these and other industries that suggest how to develop standards for global health technologies. The remainder of the Discussion considers intellectual property and regulatory issues and standards-based global health business models, and ends with a checklist of considerations for health standards development leaders. (The associated Additional file discusses observations from standards development for cell phones and semiconductors, as well as challenges in the standards development process itself.) Throughout the article, point-of-care diagnostics are used as an illustrative example. An initiative is already underway to explore standardized diagnostics platforms. SUMMARY: This Debate article aims to convince the reader that standards can benefit global health technologies if we learn lessons from other industries. The article draws from historical examples and the authors' experiences to suggest principles, challenges, and opportunities in developing these standards. If implemented well, standardized platforms can lower barriers to entry, improve affordability, and create a vibrant ecosystem of innovative new global health technologies.

Project description:IntroductionDespite widespread availability of clinical practice guidelines (CPGs), considerable gaps continue between the care that is recommended ('appropriate care') and the care provided. Problems with current CPGs are commonly cited as barriers to providing 'appropriate care'.Our study aims to develop and test an alternative method to keep CPGs accessible and up to date. This method aims to mitigate existing problems by using a single process to develop clinical standards (embodied in clinical indicators) collaboratively with researchers, healthcare professionals, patients and consumers. A transparent and inclusive online curated (purpose-designed, custom-built, wiki-type) system will use an ongoing and iterative documentation process to facilitate synthesis of up-to-date information and make available its provenance. All participants are required to declare conflicts of interest. This protocol describes three phases: engagement of relevant stakeholders; design of a process to develop clinical standards (embodied in indicators) for 'appropriate care' for common medical conditions; and evaluation of our processes, products and feasibility.Methods and analysisA modified e-Delphi process will be used to gain consensus on 'appropriate care' for a range of common medical conditions. Clinical standards and indicators will be developed through searches of national and international guidelines, and formulated with explicit criteria for inclusion, exclusion, time frame and setting. Healthcare professionals and consumers will review the indicators via the wiki-based modified e-Delphi process. Reviewers will declare conflicts of interest which will be recorded and managed according to an established protocol. The provenance of all indicators and suggestions included or excluded will be logged from indicator inception to finalisation. A mixed-methods formative evaluation of our research methodology will be undertaken.Ethics and disseminationHuman Research Ethics Committee approval has been received from the University of South Australia. We will submit the results of the study to relevant journals and offer national and international presentations.

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Developing standards for breakthrough therapy designation in oncology.

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Developing standards for breakthrough therapy designation in oncology.

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