Authors’ Reply to Ravi Jandhyala’s Comment on “Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments”

McGettigan, Patricia; Olmo, Carla Alonso; Plueschke, Kelly; Castillon, Mireia; Zondag, Daniel Nogueras; Bahri, Priya; Kurz, Xavier; Mol, Peter G. M.

doi:10.1007/s40264-019-00863-w

Cited by 18 publications

(26 citation statements)

References 8 publications

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“…Real-world data provide information about long-term safety and effectiveness of treatments outside of clinical trials; the value of such data is increasingly recognized as real-world analysis methodologies become more rigorous [1]. Disease registries are a valuable source of real-world data and are useful to evaluate benefits and risks of medicines [2]. In cystic fibrosis (CF), a lifeshortening, rare genetic disease, researchers have the unique opportunity to evaluate data from the majority of patients in their respective regions collected by national CF registries, with the US Cystic Fibrosis Foundation Patient Registry (US CFFPR) and the UK Cystic Fibrosis Registry (UK CFR) being the largest [3][4][5][6].…”

Section: Introductionmentioning

confidence: 99%

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

et al. 2020

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Introduction: In this long-term, postapproval, observational study, data from the US Cystic Fibrosis Foundation Patient Registry and the UK Cystic Fibrosis Registry were used to evaluate the impact of ivacaftor treatment on cystic fibrosis (CF) by comparing outcomes in ivacaftor-treated patients with those in matched untreated comparator patients. Registry data from up to 5 years of ivacaftor availability in the US and up to 4 years of availability in the UK were evaluated. Methods: Starting in the first year of ivacaftor availability, ivacaftor-treated patients in each registry were matched 1:5 to comparator patients who never received ivacaftor. Clinical endpoints were evaluated in annual cross-sectional safety analyses. The key endpoints were death, organ transplants, pulmonary exacerbation, and hospitalization. Relative risks and 95% CIs were calculated to compare the ivacaftor and comparator cohorts in each registry. Results: Here, we report the complete and final results of the annual cross-sectional safety analyses across the duration of the study, with up to 5 years of follow-up. Data show a pattern of lower risk of death, transplant, pulmonary exacerbation, and hospitalization among ivacaftor-treated patients in both registries. Conclusions: Ivacaftor-treated patients had consistently favorable clinical outcomes relative to untreated comparators, and no new safety concerns were identified. While general limitations of observational research apply, these findings support disease modification by CF transmembrane conductance regulator (CFTR) modulator therapy with ivacaftor. Future research of novel CFTR modulators will need to explore alternative methods for comparator selection for evaluation of clinical data given the evolving landscape of CF treatment.

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Section: Introductionmentioning

confidence: 99%

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

et al. 2020

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“…There are additional opportunities for HTA bodies and regulatory agencies engagement around optimization of real-world data (RWD) generation such as the use of patient registries ( 98 ) or the opportunity to share periodic benefit–risk assessment reports and therapeutic value reassessments. This would include alignment on key areas such as outlining the definition of data to be collected (i.e., minimum dataset) in registries ( 19 ).…”

Section: Resultsmentioning

confidence: 99%

Improving Interactions Between Health Technology Assessment Bodies and Regulatory Agencies: A Systematic Review and Cross-Sectional Survey on Processes, Progress, Outcomes, and Challenges

2020

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The need to optimize drug development and facilitate faster access for patients has ignited discussions around the importance of improving interactions between health technology assessment (HTA) bodies and regulatory agencies. In this study, we conducted a systematic review to examine processes, progress, outcomes, and challenges of harmonization/interaction initiatives between HTA bodies and regulatory agencies. MEDLINE, EMBASE, and the International Pharmaceutical Abstracts database were searched up to 21 October 2019. Searches for gray literature (working papers, commissioned reports, policy documents, etc.) were performed via Google scholar and several institutional websites. An online cross-sectional survey was also conducted among HTA (n = 22) and regulatory agencies (n = 6) across Europe to supplement the systematic review. Overall, we found that while there are areas of divergence, there has been progress over time in narrowing the gap in evidentiary requirements for HTA bodies and regulatory agencies. Most regulatory agencies (4/6; 67%) and half (11/22, 50%) of the HTA bodies reported having a formal link for "collaborating" with the other. Several mechanisms such as early tripartite dialogues, parallel submissions (reviews), adaptive licensing pathways, and postauthorization data generation have been explored as avenues for improving collaboration. A number of pilot initiatives have shown positive effects of these models to reduce the time between regulatory and HTA decisions, which may translate into faster access for patients to life-saving therapies. Thus, future approaches aimed at improving harmonization/interaction between HTA bodies and regulatory agencies should build on these existing models/mechanisms while Ofori-Asenso et al. Improving HTA-Regulatory Interactions examining their long-term impacts. Several barriers including legal, organizational, and resource-related factors were also identified, and these need to be addressed to achieve greater alignment in the current regulatory and reimbursement landscape.

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“…On a much smaller scale, the current EMA Patient Disease Registry Initiative provides an example of how incorporating the needs of all relevant stakeholders informs the development of minimal quality standards and data elements in order to facilitate downstream data harmonization and maximize the utility of the data. 3 Facilitating broad engagement and seeking agreement will support and drive adoption.…”

Section: Data Characteristicsmentioning

confidence: 99%

Big Data – How to Realize the Promise

Cave

Brun

Sweeney

et al. 2020

Clin Pharma and Therapeutics

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The increasing volume and complexity of data now being captured across multiple settings and devices offers the opportunity to deliver a better characterization of diseases, treatments, and the performance of medicinal products in individual healthcare systems. Such data sources, commonly labeled as big data, are generally large, accumulating rapidly, and incorporate multiple data types and forms. Determining the acceptability of these data to support regulatory decisions demands an understanding of data provenance and quality in addition to confirming the validity of new approaches and methods for processing and analyzing these data. The Heads of Agencies and the European Medicines Agency Joint Big Data Taskforce was established to consider these issues from the regulatory perspective. This review reflects the thinking from its first phase and describes the big data landscape from a regulatory perspective and the challenges to be addressed in order that regulators can know when and how to have confidence in the evidence generated from big datasets.

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Authors’ Reply to Ravi Jandhyala’s Comment on “Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments”

Cited by 18 publications

References 8 publications

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

Improving Interactions Between Health Technology Assessment Bodies and Regulatory Agencies: A Systematic Review and Cross-Sectional Survey on Processes, Progress, Outcomes, and Challenges

Big Data – How to Realize the Promise

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