Why do we need noncommercial, investigator-initiated clinical trials?

“…In zahlreichen Publikationen wurde seither massive Kritik sowohl an der nationalen als auch an der europäischen Gesetzgebung geäußert [8,9,10,11,12,13,14,15,16,17,18]. Die EU-Kommission hat im Jahr 2011 einen öffentlichen Konsultationsprozess zur Novellierung der Direktive begonnen und in ihrer Einleitung erkennen lassen, dass die Hemmung der unabhängigen klinischen Forschung als Problem durchaus erkannt wurde [19].…”

Section: Konsequenzen Der European Clinical Trials Directive Und Der unclassified

Beantragung der Bewilligung von akademischen Multicenterstudien bei deutschen Ethikkommissionen

Gökbuget¹,

Naumann

Dgho³

2013

Oncol Res Treat

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Für klinische Studien muss vor Beginn ein positives Votum der zuständigen Ethikkommissionen eingeholt werden. In Deutschland gibt es fast 50 unabhängige Ethikkommissionen, die für die einzelnen Standorte in Multicenterstudien zuständig sein können. Da gerade bei Studien an seltenen Erkrankungen eine Beteiligung von vielen Zentren notwendig ist, stellt das Beantragungsverfahren bei akademischen Multicenterstudien für maligne hämatologische Erkrankungen häufig einen bürokratischen Albtraum dar. Einzelne Aspekte der Antragstellung sowie Ansätze für mögliche Verbesserungen werden dargestellt.

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“…Because many of these are investigator-initiated, strategy-based studies, the bureaucratic burden may hamper development. 46 In the meantime, it is important to recall the advice of a leader in the field, Irving Weissman, on his election to president of the International Society of Stem Cell Research 47 :…”

Section: The Futurementioning

confidence: 99%

Successes and Failures of Stem Cell Transplantation in Autoimmune Diseases

Tyndall

2011

Hematology

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Over the past 15 years, more than 1500 patients have received HSCT, mostly autologous, as treatment for a severe autoimmune disease (AD). More than 1000 of these have been registered in the European Group for Bone Marrow Transplantation (EBMT) and European League Against Rheumatism (EULAR) combined database. A recent retrospective analysis of 900 patients showed that the majority had multiple sclerosis (MS; n ϭ 345) followed by systemic sclerosis (SSc; n ϭ 175), systemic lupus erythematosus (SLE; n ϭ 85), rheumatoid arthritis (RA; n ϭ 89), juvenile idiopathic arthritis (JIA; n ϭ 65), and idiopathic cytopenic purpura (ITP; n ϭ 37). An overall 85% 5-year survival and 43% progression-free survival was seen, with 100-day transplantation-related mortality (TRM) ranging between 1% (RA) and 11% (SLE and JIA). Approximately 30% of patients in all disease subgroups had a complete response, often durable despite full immune reconstitution. In many patients, such as in those with SSc, morphological improvement such as reduction of skin collagen and normalization of microvasculature was documented beyond any predicted known effects of intense immunosuppression alone. The high TRM was in part related to conditioning intensity, comorbidity, and age, but until the results of the 3 prospective randomized trials are known, an evidence-based modification of the conditioning regimen will not be possible. 1 In recent years, multipotent mesenchymal stromal cells (MSCs) have been tested in various AD, exploiting their immune-modulating properties and apparent low acute toxicity. Despite encouraging small phase 1/2 studies, no positive data from randomized, prospective studies are as yet available in the peer-reviewed literature.

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Why do we need noncommercial, investigator-initiated clinical trials?

Cited by 15 publications

References 7 publications

Application of autologous stem cell transplantation in various adult and pediatric rheumatic diseases

Application of autologous stem cell transplantation in various adult and pediatric rheumatic diseases

Beantragung der Bewilligung von akademischen Multicenterstudien bei deutschen Ethikkommissionen

Successes and Failures of Stem Cell Transplantation in Autoimmune Diseases

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