Abstract:
BACKGROUND:Timely access to new medicines may be addressed through strengthening of registration efficiencies and timelines by establishing and refining value-added registration processes, resources, and systems. The aims of this study were to evaluate the timelines of the milestones of the South African review process and the overall approval process for new active substances (NASs) in 2015-2018 and to provide recommendations for improved patients' access to new medicines through timely registration. METHODS:Data identifying the milestones and overall approval times for NASs registered by the South African Agency during 2015-2018 were collected and analyzed. RESULTS:The most NASs (42) were approved in 2017 and the least (15) in 2018. The shortest median approval time (1218 calendar days) was achieved in 2015 and the longest (2124 days), in 2018. All applications were reviewed using the full review process, and 16/99 (16%) were assigned priority status and were reviewed and approved through the fast track review. CONCLUSIONS:While the extensive delays in NASs approvals in South Africa may be attributed to inefficient operational processes, resource constraints, and as an increased number of applications for registration, the newly established South African Heath Products Regulatory Agency has re-engineered and streamlined its regulatory review process, which has been piloted and will be enhanced prior to final implementation. Among recommendations for improvement, SAHPRA should consider measurement and monitoring of milestones, facilitated regulatory pathways, implementing a reliance strategy, and a quality management system.
journal_name
Ther Innov Regul Scijournal_title
Therapeutic innovation & regulatory scienceauthors
Keyter A,Salek S,Gouws J,Banoo S,Walker Sdoi
10.1007/s43441-019-00013-5subject
Has Abstractpub_date
2020-07-01 00:00:00pages
878-887issue
4eissn
2168-4790issn
2168-4804pii
10.1007/s43441-019-00013-5journal_volume
54pub_type
杂志文章abstract::The regulatory agencies all over the world have defined the pathway and regulations for the approval of simple small-molecule generics. In addition, the agencies are striving to form perspicuous regulatory frameworks for the approval of biosimilars. In this view, there are no defined regulations for the approval of co...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
doi:10.1007/s43441-020-00114-6
更新日期:2020-09-01 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
pub_type: 杂志文章
doi:10.1177/2168479018769292
更新日期:2019-01-01 00:00:00
abstract:BACKGROUND:In the breakthrough therapy designation (BTD) and Sakigake designation programs, rolling submission and close communication between applicants and regulatory authorities enable the timely access of patients to innovative medicines. However, challenges in the quality development, including chemistry, manufact...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-019-00019-z
更新日期:2020-07-01 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
pub_type: 杂志文章
doi:10.1177/2168479014563549
更新日期:2015-05-01 00:00:00
abstract::Much has changed in a relatively short period of time. There is a raging debate over the level of evidence expected to first introduce a treatment to patients based on smaller, more adaptive data sets. Some argue for less data followed by postapproval follow-up, others for more adaptive clinical trial designs and end-...
journal_title:Therapeutic innovation & regulatory science
pub_type: 社论
doi:10.1177/2168479018766887
更新日期:2018-07-01 00:00:00
abstract::After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Althoug...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479015618697
更新日期:2016-05-01 00:00:00
abstract:OBJECTIVE:To identify, evaluate, and characterize the variety, quality, and intent of the health economics and outcomes research studies being conducted in SAARC (South Asian Association for Regional Cooperation) nations. METHODS:Studies published in English language between 1990 and 2015 were retrieved from Medline d...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章,评审
doi:10.1177/2168479017731583
更新日期:2018-05-01 00:00:00
abstract::Attention deficit hyperactivity disorder (ADHD) is the most common neurobiological disorder in children, with a prevalence of ~6-7%1,2 that has remained stable for decades2. The social and economic burden associated with patients3, families, and broader systems (healthcare/educational) is substantial, with the annual ...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479015599811
更新日期:2015-09-01 00:00:00
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 pre...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00244-x
更新日期:2020-11-23 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...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479014546336
更新日期:2014-09-01 00:00:00
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
pub_type: 杂志文章
doi:10.1177/2168479013487541
更新日期:2013-07-01 00:00:00
abstract:BACKGROUND:(1) A growing number of pharmaceutical and biotechnology organizations are engaging patients, their support networks, and clinical trial site staff at various touchpoints along the clinical research development spectrum to solicit feedback on how to reduce the burden of clinical trial participation and admin...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479019849875
更新日期:2019-05-22 00:00:00
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
pub_type: 杂志文章
doi:10.1177/2168479018793130
更新日期:2019-03-29 00:00:00
abstract:BACKGROUND:Predictions of drug sales play an important role in setting drug prices in Japan, where drug prices are highly regulated. One of 2 primary Japanese drug-pricing methodologies-the cost calculation method- refers extensively to the prediction of drug sales in establishing drug prices. Deviations from predicted...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479019860123
更新日期:2019-07-16 00:00:00
abstract::Large electronic health care databases can be used to evaluate the effectiveness of regulatory action. Designing and interpreting such studies can be challenging. We demonstrate using case studies on modafinil, benzodiazepines, dosulepin, and piroxicam that measuring the impact of regulatory action is possible and can...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479015570333
更新日期:2015-07-01 00:00:00
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
pub_type: 杂志文章
doi:10.1177/2168479015587362
更新日期:2015-11-01 00:00:00
abstract::Meta-analyses are increasingly influencing clinical practice, but significant methodological flaws have been reported. The purpose of this study was to evaluate the quality of search strategies utilized by anti-infective meta-analyses. The Embase database was searched for meta-analyses evaluating anti-infective drug t...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479013516774
更新日期:2014-05-01 00:00:00
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...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479018778528
更新日期:2019-03-01 00:00:00
abstract::The product pipeline for diseases that disproportionately affect the developing world has considerably expanded over the last decade. Indeed, there are about 134 products for these diseases in the pipeline, including vaccines, drugs, diagnostics, microbicides, and vector control tools, and dozens of these products are...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479013478952
更新日期:2013-05-01 00:00:00
abstract:BACKGROUND:Access and use of historical control data was identified as a top stakeholder concern across organizations according to results of a survey of needs and challenges related to nonclinical data conducted by the FDA/PhUSE Nonclinical Working Group in 2011. There is a perception there may be additional ways to c...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479015600203
更新日期:2016-03-01 00:00:00
abstract:BACKGROUND:(1) To assess the differing viewpoints of actual and potential adult clinical trial participants in 5 age cohorts, (2) identify misperceptions and opportunities to reduce unnecessary burdens on volunteers, and (3) provide age-specific feedback to study sponsors on their current engagement efforts. METHODS:B...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-019-00033-1
更新日期:2020-01-01 00:00:00
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
pub_type: 杂志文章
doi:10.1007/s43441-019-00055-9
更新日期:2020-03-01 00:00:00
abstract:BACKGROUND:Pediatric regulations enacted in both Europe and the USA have disrupted the pharmaceutical industry, challenging business and drug development processes, and organizational structures. Over the last decade, with science and innovation evolving, industry has moved from a reactive to a proactive mode, investin...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00116-4
更新日期:2020-09-01 00:00:00
abstract:BACKGROUND:The design and sample size of a phase III study for new medical technologies were historically determined within the framework of frequentist hypothesis testing. Recently, drug development using predictive biomarkers, which can predict efficacy based on the status of biomarkers, has attracted attention, and ...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00119-1
更新日期:2020-09-01 00:00:00
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
pub_type: 杂志文章
doi:10.1007/s43441-019-00103-4
更新日期:2020-05-01 00:00:00
abstract:BACKGROUND:A data monitoring committee (DMC) has special responsibilities for protecting the safety of clinical trial participants. Few guidance documents are available that address the operations and mechanics of establishing, serving on, or reporting to a DMC. This article provides a practical guide to sponsors, inst...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479016646812
更新日期:2016-09-01 00:00:00
abstract:BACKGROUND:Contending with a continuously expanding volume and variety of clinical data poses challenges and opportunities for the industry and clinical data management organizations. METHODS:Tufts CSDD conducted an online survey aimed at further quantifying and understanding the magnitude and impact that expanded dat...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1007/s43441-020-00213-4
更新日期:2020-09-14 00:00:00
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
pub_type: 杂志文章,评审
doi:10.1177/2168479018775659
更新日期:2019-03-01 00:00:00
abstract::Adaptive designs are increasingly used in clinical trials. The Drug Information Association's Adaptive Design Scientific Working Group (ADSWG) works to foster collaboration among regulatory agencies, academia, and pharmaceutical and biotech companies to further the science of adaptive clinical development. The ADSWG S...
journal_title:Therapeutic innovation & regulatory science
pub_type: 杂志文章
doi:10.1177/2168479014522468
更新日期:2014-07-01 00:00:00
abstract::A universal framework for the evaluation of the benefit-risk assessment of medicines during development by pharmaceutical companies and in the regulatory review by regulatory authorities is considered of value, as it would result in the systematic structured approach to support transparency in decision making. Several...
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