New opportunities of real-world data from clinical routine settings in life-cycle management of drugs: example of an integrative approach in multiple sclerosis

ObjectiveTo evaluate the impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany.DesignThis study employs real-world data captured in-time during clinical visits in 67 German neurology outpatient offices of the NeuroTransData (NTD) multiple sclerosis (MS) registry between 1 January 2010 and 30 June 2019, including 237 976 visits of 17 553 patients with RRMS. Adherence and clinical effectiveness parameters were analysed by descriptive statistics, time-to-event analysis overall and by disease-modifying therapies (DMTs) stratified by administration modes (injectable, oral and infusion). Three time periods were compared: 2010–2012, 2013–2015 and 2016–2018.ResultsBetween 2010 and 2018, an increasing proportion of patients with RRMS were treated with DMTs and treatment was initiated sooner after diagnosis of MS. Introduction of oral DMT temporarily induced higher readiness to switch. Comparing the three index periods, there was a continuous decrease of annualised relapse rates, less frequent Expanded Disability Status Scale (EDSS) progression and increasing periods without relapse, EDSS worsening and with stability of no-evidence-of-disease-activity 2 and 3 criteria, lower conversion rates to secondary progressive MS on oral and on injectable DMTs.ConclusionSparked by the availability of new mainly oral DMTs, RRMS treatment effectiveness improved clinically meaningful between 2010 and 2018. As similar effects were seen for injectable and oral DMTs more than for infusions, a better personalised treatment allocation in many patients is likely. These results indicate that there is an overall beneficial effect for the whole patient with MS population as a result of the greater selection of available DMTs, a benefit beyond the head-to-head comparative efficacy, resulting from an increased probability and readiness to individualise MS therapy.

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Italian multiple sclerosis register as the basis for post-authorization safety studies

European Journal of Public Health ◽

10.1093/eurpub/ckaa166.1255 ◽

2020 ◽

Vol 30 (Supplement_5) ◽

Author(s):

M Ponzio ◽

M Trojano ◽

M Capobianco ◽

M Pugliatti ◽

M Ulivelli ◽

...

Keyword(s):

Multiple Sclerosis ◽

Pharmaceutical Industry ◽

Real World ◽

Treatment Effectiveness ◽

Patient Registries ◽

Real World Data ◽

World Data ◽

Disease Modifying Therapy ◽

Disease Modifying

Abstract While the safety and efficacy of Disease Modifying Therapy (DMT) in multiple sclerosis (MS) are assessed in clinical trials, these are of relatively short duration and always confined to highly selected patient groups. The evaluation of real-world data such as patient registries, is vital as it offers long-term data collection and is patient rather than product-focused during the lifetime of MS, and allows to document a patient's treatment history throughout the disease course. Patient registries can play an important role in monitoring the safety of drus. Regulators and the pharmaceutical industry have shown interest in complementing or even replacing phase 4 clinical studies with data from MS registries. The Italian MS Register (IMSR), in collaboration with the Big MS Data initiative, that also includes the national MS registries of Denmark, France, and Sweden and the international database network MSBase, came together with industry to conduct studies on post-authorization safety (PASS) and treatment effectiveness. The IMSR includes the clinical history of approximately 55,000 patients, or approximatively 45% of the estimated cases of MS in Italy. More than 10,000 patients have a follow-up duration of over 10 years. A Core Protocol outlining principles of PASS projects was created in which aggregated results made available to sponsors and health authorities. The Core Protocol specifies variables, emphasizes improved capture of adverse events, in particular cancer, non-melanoma skin cancers and immunosuppression-related infections, all MedDRA-coded. EUROCAT codes for pregnancy outcomes are also documented. Regulators, the pharmaceutical industry and national-level registries have jointly identified a format of collaboration on PASS for DMT in MS to benefit patients and the larger society. In this way, we hope to contribute to a framework that will include emerging and existing registries with the common goal of contributing to the advancement of knowledge in MS. Key messages The real-world data can contribute to understanding of the impact of disease-modifying therapy on long term. A format of collaboration among clinical research, regulators and pharmaceutical industry could be a winning framework to improve the knowledge on safety and treatment effectiveness in MS.

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Leveraging real-world data to investigate multiple sclerosis disease behavior, prognosis, and treatment

Multiple Sclerosis Journal ◽

10.1177/1352458519892555 ◽

2019 ◽

Vol 26 (1) ◽

pp. 23-37 ◽

Cited By ~ 7

Author(s):

Jeffrey A Cohen ◽

Maria Trojano ◽

Ellen M Mowry ◽

Bernard MJ Uitdehaag ◽

Stephen C Reingold ◽

...

Keyword(s):

Multiple Sclerosis ◽

Outcome Measures ◽

Real World ◽

Observational Studies ◽

Patient Reported Outcome Measures ◽

Medical Community ◽

Real World Data ◽

World Data ◽

Patient Reported ◽

Disease Behavior

Randomized controlled clinical trials and real-world observational studies provide complementary information but with different validity. Some clinical questions (disease behavior, prognosis, validation of outcome measures, comparative effectiveness, and long-term safety of therapies) are often better addressed using real-world data reflecting larger, more representative populations. Integration of disease history, clinician-reported outcomes, performance tests, and patient-reported outcome measures during patient encounters; imaging and biospecimen analyses; and data from wearable devices increase dataset utility. However, observational studies utilizing these data are susceptible to many potential sources of bias, creating barriers to acceptance by regulatory agencies and the medical community. Therefore, data standardization and validation within datasets, harmonization across datasets, and application of appropriate analysis methods are important considerations. We review approaches to improve the scope, quality, and analyses of real-world data to advance understanding of multiple sclerosis and its treatment, as an example of opportunities to better support patient care and research.

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