Abstract:
BACKGROUND:The FDA Patient-Focused Drug Development Initiative was launched to ensure the incorporation of the patient voice into drug development and evaluation. Since 2017, the FDA must publish a statement outlining patient experience data (PED) considered in the approval of new drugs. This study investigated the presence and role of PED in drug approval and translation into product label claims. METHODS:PED reported in approval packages of the 48 drugs approved by FDA's Center for Drug Evaluation and Research in 2019 was identified and categorized. PED in the form of clinical outcome assessments (COAs) was characterized by endpoint positioning and outcome. The product labels were analyzed for PED-related claims. RESULTS:PED was reported as relevant for 39 of 48 (81.3%) drugs approved in 2019. COAs were the predominant PED type; other PED was identified for only 9 (18.8%) drugs, and none included qualitative or patient preference studies. COAs were the only type of PED for which associated claims were identified in the product labels. 27 out of 48 (56.3%) labels contained one or more efficacy claims based on COAs; of these, patient-reported outcomes were the most prevalent with claims identified in 19 labels (39.6%). CONCLUSION:There are ample opportunities for incorporating PED beyond COAs to inform drug development and facilitate availability of medicines tailored to patient needs. A higher level of transparency on the role of PED in regulatory decision-making and a clear path to PED-based label claims could incentivize sponsors and enable patient empowerment in treatment decisions.
journal_name
Ther Innov Regul Scijournal_title
Therapeutic innovation & regulatory scienceauthors
Schultz-Knudsen K,Sabaliauskaite U,Hellsten J,Lassen AB,Morant AVdoi
10.1007/s43441-020-00244-xsubject
Has Abstractpub_date
2020-11-23 00:00:00eissn
2168-4790issn
2168-4804pii
10.1007/s43441-020-00244-xpub_type
杂志文章abstract:BACKGROUND:Increasing generic drug price competition by facilitating abbreviated new drug applications (ANDA) submission may help patients have access to affordable care. This study examined factors associated with first ANDA submission for the brand drug to be copied [the "reference listed drug" (RLD)]. METHODS:This ...
journal_title:Therapeutic innovation & regulatory science
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doi:10.1007/s43441-020-00163-x
更新日期:2020-11-01 00:00:00
abstract::The volume and diversity of data collected to support each clinical study has increased dramatically in response to the rising scope and complexity of global drug development programs. The Tufts Center for the Study of Drug Development conducted an online survey of 257 unique global companies-77% drug development spon...
journal_title:Therapeutic innovation & regulatory science
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abstract::In general, similar to FDA and other health authorities, the PMDA requires clinical efficacy study(ies) to evaluate equivalence between a reference biological product and a Biosimilar product for new drug applications. Even if an identical clinical efficacy study is included in both of PMDA and FDA submissions, the co...
journal_title:Therapeutic innovation & regulatory science
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journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
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abstract:BACKGROUND:The US Food and Drug Administration (FDA) put out a call for comments on new draft guidance for industry "Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA." This call for comments elicited 7 submissions from various organization...
journal_title:Therapeutic innovation & regulatory science
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abstract:BACKGROUND:Communicating the clinical impact of immunogenicity in labeling is important for safe and effective use of certain prescription products. Current U.S. Food and Drug Administration (FDA) guidance does not provide comprehensive recommendations on the communication of clinical impact of immunogenicity in labeli...
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abstract:BACKGROUND:With the pharmaceutical industry's increased attention on enhancing patient experiences during their participation in clinical trials, the use of mobile nurse (MN) services to support the conduct of clinical trial assessments in the home (or in alternative locations other than in investigational sites) has b...
journal_title:Therapeutic innovation & regulatory science
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abstract::The Food and Drug Administration Amendments Act of 2007 gave the FDA the authority to require drug sponsors to submit a risk evaluation and mitigation strategies (REMS) program for those medicines with serious risks such that failure to effectively manage these risks would tip the benefit-risk balance. As of August 8,...
journal_title:Therapeutic innovation & regulatory science
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abstract::There is a long history of discussions about what is a minimal clinically important difference (MCID) and how this term applies to clinical research. This paper deals with a practical framework for MCID and its applicability to clinical trials. A literature review on the topic confirmed the fundamental role of MCID fo...
journal_title:Therapeutic innovation & regulatory science
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abstract:BACKGROUND:The influence of patient advocates on FDA regulatory decision making has increased. Despite enhanced engagement with FDA, there remain challenges to achieving the regulatory goals of patients within FDA's regulatory framework. Gaps exist between patient advocates' knowledge of the agency's processes and FDA'...
journal_title:Therapeutic innovation & regulatory science
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abstract::Social media presents new challenges to the biopharmaceutical industry for conducting pharmacovigilance activities. The authors reviewed worldwide regulatory guidance documents related to monitoring of adverse events posted on social media sites and identified gaps in current regulatory definitions for pharmacovigilan...
journal_title:Therapeutic innovation & regulatory science
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abstract::In the current pharmaceutical regulatory environment, patients continue to benefit from great advances in medical care. Sophisticated regulatory review systems have also evolved to ensure that safe and effective medicines are approved. However, these systems are not optimized in all countries. Gaps in individual regul...
journal_title:Therapeutic innovation & regulatory science
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abstract::The American Statistical Association and DIA have created an interdisciplinary working group of drug safety experts from academia, industry and regulatory backgrounds to explore the future direction for safety monitoring. This introduction to the series explains the background and rationale for this special section. ...
journal_title:Therapeutic innovation & regulatory science
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doi:10.1177/2168479018793130
更新日期:2019-03-29 00:00:00
abstract:BACKGROUND:The coherence between the relationship of QTc and drug plasma concentration (this relationship is measured through the slope) and ICH E14 findings based on hundreds of QT study reports was studied. RESULTS:Based on ICH E14 analysis, our findings indicate that if the slope was not positive, in most cases (86...
journal_title:Therapeutic innovation & regulatory science
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abstract::In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is v...
journal_title:Therapeutic innovation & regulatory science
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abstract::When does a single positive adequate and well-controlled study of a new drug meet the statutory requirement to demonstrate substantial evidence of effectiveness? The answer to this question, particularly with respect to new molecular entities, has been of considerable debate since 1962 when the requirement that new dr...
journal_title:Therapeutic innovation & regulatory science
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doi:10.1007/s43441-019-00103-4
更新日期:2020-05-01 00:00:00
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journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479014547421
更新日期:2015-01-01 00:00:00
abstract:BACKGROUND:The number of diabetes patients is steadily increasing worldwide. Consequently, the social burden of diabetes is huge, requiring urgent countermeasures. We performed an intensive survey of antidiabetic drugs approved in Japan, the United States, and the European Union. METHODS:Information about approved ant...
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更新日期:2019-07-01 00:00:00
abstract::TransCelerate BioPharma Inc developed a methodology based on the notion that shifting monitoring processes from an excessive concentration on source data verification to comprehensive risk-driven monitoring will increase efficiencies and enhance patient safety and data integrity while maintaining adherence to good cli...
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更新日期:2014-09-01 00:00:00
abstract:BACKGROUND:Package leaflets of medicines distributed within the European Union should use the template headings and standard texts created by the Working Group on the Quality Review of Documents (QRD). The following study investigated how the QRD template is implemented in this patient information. METHODS:All English...
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更新日期:2016-01-01 00:00:00
abstract::Although much information is already available publically from information-sharing initiatives such as ClinicalTrials.gov, information about clinical programs is unstructured, inconsistent, and incomplete. Clinical research within bioscience companies, health care, academia, and governmental agencies could benefit fro...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2015-01-01 00:00:00
abstract:BACKGROUND:A new incentive program, "Premium Rewards for the Promotion of Innovative Drug Discovery and Resolution of Off-Label Use, etc," was introduced in Japan in 2010 and subsequently applied in 2010, 2012, and 2014. In our previous study, we reported statistical evidence regarding the attributes of drugs for which...
journal_title:Therapeutic innovation & regulatory science
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更新日期:2016-01-01 00:00:00
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journal_title:Therapeutic innovation & regulatory science
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abstract::Data quality is critical for clinical trials to obtain robust conclusions about drug safety and efficacy evaluation. Effective data quality evaluation has been one of the major obstacles to new drug approvals in China, which hinders innovation in drug discovery and development ultimately. To improve the data quality s...
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更新日期:2021-01-01 00:00:00
abstract:BACKGROUND:Formal incorporation of patients' perspectives is becoming increasingly important in medical product development and decision making. This article shares practical advice regarding how patient advocacy organizations, the pharmaceutical industry, and academic experts in stated-preference research can effectiv...
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更新日期:2018-07-01 00:00:00
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更新日期:2014-03-01 00:00:00
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