scholarly journals Regulatory landscapes for biomarkers and diagnostic tests: Qualification, approval, and role in clinical practice

2017 ◽  
Vol 243 (3) ◽  
pp. 256-261 ◽  
Author(s):  
William B. Mattes ◽  
Federico Goodsaid
Keyword(s):  
Product Development ◽  
Diagnostic Tests ◽  
New Technologies ◽  
Biomarker Discovery ◽  
Medical Product ◽  
The Road ◽  
Regulatory Decision ◽  
The Us ◽  
Recent Developments ◽  
Regulatory Decisions

While the term ‘biomarker’ is relatively new, the concept is millennia old. However, with the introduction of new technologies to discover potential biomarkers comes the need to assess their utility and veracity for any given use. This is particularly true for the use of biomarkers to support regulatory decisions in medical product development. Hence the US Food and Drug Administration has developed processes for the qualification of biomarkers and other medical product development tools, processes that are underscored by recent legislation (i.e. the 21st Century Cures Act). In addition to these qualification processes, diagnostic tests that measure a biomarker may follow a process for regulatory decision through the processes that evaluate companion diagnostics. This mini-review provides an overview of these processes and their role in pharmaceutical development and clinical use. Impact statement This work summarizes very recent developments in the US FDA’s biomarker qualification program. Furthermore, it contrasts biomarker qualification with companion diagnostic evaluation. As such, it will be highly informative for researches considering taking a biomarker discovery farther along the road to validation.

scholarly journals Emerging technologies and their impact on regulatory science

2021 ◽  
pp. 153537022110522
Author(s):  
Elke Anklam ◽  
Martin Iain Bahl ◽  
Robert Ball ◽  
Richard D Beger ◽  
Jonathan Cohen ◽  
...  
Keyword(s):  
New Technologies ◽  
Emerging Technologies ◽  
Cost Effective ◽  
Assessment Tools ◽  
Regulatory Science ◽  
Science Field ◽  
Regulatory Decision ◽  
Technical Advances ◽  
Novel Approaches ◽  
Regulatory Decisions

There is an evolution and increasing need for the utilization of emerging cellular, molecular and in silico technologies and novel approaches for safety assessment of food, drugs, and personal care products. Convergence of these emerging technologies is also enabling rapid advances and approaches that may impact regulatory decisions and approvals. Although the development of emerging technologies may allow rapid advances in regulatory decision making, there is concern that these new technologies have not been thoroughly evaluated to determine if they are ready for regulatory application, singularly or in combinations. The magnitude of these combined technical advances may outpace the ability to assess fit for purpose and to allow routine application of these new methods for regulatory purposes. There is a need to develop strategies to evaluate the new technologies to determine which ones are ready for regulatory use. The opportunity to apply these potentially faster, more accurate, and cost-effective approaches remains an important goal to facilitate their incorporation into regulatory use. However, without a clear strategy to evaluate emerging technologies rapidly and appropriately, the value of these efforts may go unrecognized or may take longer. It is important for the regulatory science field to keep up with the research in these technically advanced areas and to understand the science behind these new approaches. The regulatory field must understand the critical quality attributes of these novel approaches and learn from each other's experience so that workforces can be trained to prepare for emerging global regulatory challenges. Moreover, it is essential that the regulatory community must work with the technology developers to harness collective capabilities towards developing a strategy for evaluation of these new and novel assessment tools.

Technology Assessment in the EU Institutions

2011 ◽  
Vol 6 (5) ◽  
pp. 522-527
Author(s):  
Michael D. Rogers ◽  
Keyword(s):  
Technology Assessment ◽  
New Technologies ◽  
Decision Makers ◽  
Regulatory Decision ◽  
Advisory Groups ◽  
Formal Requirement ◽  
Regulatory Impact ◽  
The Right ◽  
Regulatory Decisions ◽  
The Eu

Technology Assessment (TA) is the study and evaluation of new technologies with the objective of understanding the likely impacts (costs and benefits) of these technologies on society and the environment – with the explicit aim of improving regulatory decision making concerning these technologies. This is a prospective exercise helping to ensure that “better” regulatory decisions are made by decision makers. TA and “TA like” activities are embedded within the main EU institutions. The Commission carries out Regulatory Impact Assessments on every significant regulatory proposal. It also has at its disposal a range of advisory groups which includes the European Group on Ethics in Science and New Technologies and the Institute for Prospective Technological Studies. The European Parliament has its own TA unit – the Science and Technology Options Assessment unit. The institutions are committed to quality, transparency and effectiveness in their use of expert groups and all such assessments are published on the internet. Occasionally full citizens’ consultations are carried out but this is not a formal requirement. Recent changes in the regulatory development process have emphasised the concept of “smarter” regulations. This concept is concerned not just with prospective analyses in advance of new regulations but also with the retrospective evaluation of existing regulations asking the question“Did they meet the need that was the raison d’être for enacting the regulation under consideration.”The distinction highlighted by Churchill [2] that experts should advise but not decide is intended to ensure that regulators take account of aspects other than the expert view. Nevertheless, it is essential that expert groups have the right to introduce advice thatmight otherwise be overlooked by the regulators, as is the case in a number of expert groups in the EU institutions.

scholarly journals An Evolved Approach to Advisory Boards in Rare Disease Drug Development: 5-Step Model to Finding and Engaging Patient Advisors

2020 ◽  
Vol 7 (6) ◽  
pp. 978-981
Author(s):  
Carlotta Dillon ◽  
Joyce Knapp ◽  
Mark Stinson
Keyword(s):  
Drug Development ◽  
Product Development ◽  
New Product Development ◽  
Medical Product ◽  
Full Potential ◽  
Regulatory Decision ◽  
Advisory Boards ◽  
New Product Development Teams ◽  
Step Model ◽  
Disease Advocacy

Nearly all new product development teams at pharmaceutical companies will routinely conduct patient advisory boards. These board meetings will help collect and document the experience of patients and caregivers for medical product development and regulatory decision-making. Recently, in June 2020, The US Food and Drug Administration (FDA) published a final guidance on methodological patient-focused drug development (PFDD) to address, in a stepwise manner, how stakeholders (patients, researchers, medical product developers, and others) can successfully use these patient forums. In the process of developing this guidance, the FDA acknowledged that leading its own PFDD meetings, especially when limited to organized disease advocacy groups, cannot address the gaps in information on the patient perspective. So, it has expressed support for advancing the science and utilization of patient input other means. Because traditional methods of conducting patient advisory boards often do not achieve the full potential of patient centricity, the authors of this article share an approach to consider when selecting patient advisors, in order to gain the most actionable input to a product development team.

Recent Developments of Ambulatory Assessment Methods

2014 ◽  
Vol 25 (4) ◽  
pp. 279-287 ◽  
Author(s):  
Stefan Hey ◽  
Panagiota Anastasopoulou ◽  
André Bideaux ◽  
Wilhelm Stork
Keyword(s):  
Real Time ◽  
Information And Communication Technologies ◽  
New Technologies ◽  
Psychological Research ◽  
Ambulatory Assessment ◽  
Emotional States ◽  
Recent Developments ◽  
Potential Applications ◽  
Information And Communication ◽  
Physiological Sensors

Ambulatory assessment of emotional states as well as psychophysiological, cognitive and behavioral reactions constitutes an approach, which is increasingly being used in psychological research. Due to new developments in the field of information and communication technologies and an improved application of mobile physiological sensors, various new systems have been introduced. Methods of experience sampling allow to assess dynamic changes of subjective evaluations in real time and new sensor technologies permit a measurement of physiological responses. In addition, new technologies facilitate the interactive assessment of subjective, physiological, and behavioral data in real-time. Here, we describe these recent developments from the perspective of engineering science and discuss potential applications in the field of neuropsychology.

Regulatory decisions from the US FDA

2001 ◽  
Vol &NA; (1272) ◽  
pp. 22
Author(s):  
&NA;
Keyword(s):  
The Us ◽  
Us Fda ◽  
Regulatory Decisions

Green Synthesized Nanomaterials as Theranostic Platforms for Cancer Treatment: Principles, Challenges and the Road Ahead

2019 ◽  
Vol 26 (8) ◽  
pp. 1311-1327 ◽  
Author(s):  
Pala Rajasekharreddy ◽  
Chao Huang ◽  
Siddhardha Busi ◽  
Jobina Rajkumari ◽  
Ming-Hong Tai ◽  
...  
Keyword(s):  
Nanoparticle Synthesis ◽  
Research Field ◽  
Synthesis Methods ◽  
Synthetic Approach ◽  
Future Directions ◽  
The Road ◽  
New Methods ◽  
Recent Developments ◽  
Theranostic Applications ◽  
Insight Into

With the emergence of nanotechnology, new methods have been developed for engineering various nanoparticles for biomedical applications. Nanotheranostics is a burgeoning research field with tremendous prospects for the improvement of diagnosis and treatment of various cancers. However, the development of biocompatible and efficient drug/gene delivery theranostic systems still remains a challenge. Green synthetic approach of nanoparticles with low capital and operating expenses, reduced environmental pollution and better biocompatibility and stability is a latest and novel field, which is advantageous over chemical or physical nanoparticle synthesis methods. In this article, we summarize the recent research progresses related to green synthesized nanoparticles for cancer theranostic applications, and we also conclude with a look at the current challenges and insight into the future directions based on recent developments in these areas.

3D Printing in Pharmaceuticals: Regulatory Perspective

2019 ◽  
Vol 24 (42) ◽  
pp. 5081-5083 ◽  
Author(s):  
Mohd. A. Mirza ◽  
Zeenat Iqbal
Keyword(s):  
New Technologies ◽  
Pharmaceutical Product ◽  
Regulatory Agencies ◽  
Major Research ◽  
Data Bases ◽  
Drug Products ◽  
Regulatory Perspective ◽  
Regulatory Guidelines ◽  
Recent Developments ◽  
Technical Issues

Background: The last few decades have witnessed enormous advancements in the field of Pharmaceutical drug, design and delivery. One of the recent developments is the advent of 3DP technology. It has earlier been successfully employed in fields like aerospace, architecture, tissue engineering, biomedical research, medical device and others, has recently forayed into the pharmaceutical industry.Commonly understood as an additive manufacturing technology, 3DP aims at delivering customized drug products and is the most acceptable form of“personalized medicine”. Methods: Data bases and search engines of regulatory agencies like USFDA and EMA have been searched thoroughly for relevant guidelines and approved products. Other portals like PubMed and Google Scholar were also ferreted for any relevant repository of publications are referred to wherever required. Results: So far only one pharmaceutical product has been approved in this category by USFDA and stringent regulatory agencies are working over the drafting of guidelines and technical issues. Major research of this category belongs to the academic domain. Conclusion: It is also implicit to such new technologies that there would be numerous challenges and doubts before these are accepted as safe and efficacious. The situation demands concerted and cautious efforts to bring in foolproof regulatory guidelines which would ultimately lead to the success of this revolutionary technology.

Mass Spectrometry for Proteomics and Recent Developments in ESI, MALDI and other Ionization Methodologies

2019 ◽  
Vol 16 (4) ◽  
pp. 267-276
Author(s):  
Qurat ul Ain Farooq ◽  
Noor ul Haq ◽  
Abdul Aziz ◽  
Sara Aimen ◽  
Muhammad Inam ul Haq
Keyword(s):  
Mass Spectrometry ◽  
Electrospray Ionization ◽  
New Technologies ◽  
Protein Complexes ◽  
Imaging Techniques ◽  
Desorption Electrospray Ionization ◽  
High Throughput Analysis ◽  
Enhanced Sensitivity ◽  
Recent Developments ◽  
The Way

Background: Mass spectrometry is a tool used in analytical chemistry to identify components in a chemical compound and it is of tremendous importance in the field of biology for high throughput analysis of biomolecules, among which protein is of great interest. Objective: Advancement in proteomics based on mass spectrometry has led the way to quantify multiple protein complexes, and proteins interactions with DNA/RNA or other chemical compounds which is a breakthrough in the field of bioinformatics. Methods: Many new technologies have been introduced in electrospray ionization (ESI) and Matrixassisted Laser Desorption/Ionization (MALDI) techniques which have enhanced sensitivity, resolution and many other key features for the characterization of proteins. Results: The advent of ambient mass spectrometry and its different versions like Desorption Electrospray Ionization (DESI), DART and ELDI has brought a huge revolution in proteomics research. Different imaging techniques are also introduced in MS to map proteins and other significant biomolecules. These drastic developments have paved the way to analyze large proteins of >200kDa easily. Conclusion: Here, we discuss the recent advancement in mass spectrometry, which is of great importance and it could lead us to further deep analysis of the molecules from different perspectives and further advancement in these techniques will enable us to find better ways for prediction of molecules and their behavioral properties.

The Dilemma between Aligned Expectations and Diversity in Innovation

2018 ◽  
Author(s):  
Timur Ergen
Keyword(s):  
New Technologies ◽  
Historical Case ◽  
History Of Capitalism ◽  
Synthetic Fuel ◽  
The Us ◽  
History Of ◽  
The One ◽  
Technology Policies ◽  
Lock In ◽  
Innovation Processes

This chapter brings together arguments from economics, sociology, and political economy to show that innovation processes are characterized by a dilemma between the advantages of aligned expectations—including greater coordination and investment—and those of diversity, including superior openness to new technological possibilities. To illustrate the argument, the chapter discusses a historical case involving one of the largest coordinated peace-time attempts to hasten technological innovation in the history of capitalism, namely the US energy technology policies of the 1970s and 1980s. Close examination of the commercialization of photovoltaics and synthetic fuel initiatives illustrates both sides of the dilemma between shared versus diverse expectations in innovation: coordination but possible premature lock-in on the one hand, and openness but possible stagnation on the other. The chapter shows that even the exploration and interpretation of new technologies may be as much a product of focused investment as of trial-and-error search.

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