Arzneimittel für neuartige Therapien umfassend bewerten

Thanner, Mirjam; Nagel, Eckhard

doi:10.1007/s00103-011-1300-7

Cited by 6 publications

(2 citation statements)

References 21 publications

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“…ATMPs hold great potential for reshaping the progression or the disability associated with multiple diseases such as Alzheimer's disease, Parkinson's disease, cancer, muscular dystrophy, and so on ( 13 , 14 ), including an option for curing or reversing diseases known as untreatable today or that are just subject to symptomatic treatments. Maciulaitis et al ( 15 ) identified 318 clinical trials for ATMPs registered in the EU Drug Regulating Authorities Clinical Trials (EudraCT) database between 2004 and 2010.…”

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confidence: 99%

Advanced therapy medicinal products: current and future perspectives

Hanna

Rémuzat

Auquier

et al. 2016

Journal of Market Access & Health Policy

113

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BackgroundAdvanced therapy medicinal products (ATMPs) are innovative therapies that encompass gene therapy, somatic cell therapy, and tissue-engineered products. These therapies are expected to bring important health benefits, but also to substantially impact the pharmaceuticals budget.ObjectiveThe aim of this study was to characterise the ATMPs in development and discuss future implications in terms of market access.MethodsClinical trials were searched in the following databases: EudraCT (EU Drug Regulating Authorities Clinical Trials), ClinicalTrials.gov, and ICTRP (International Clinical Trials Registry Platform of the World Health Organization). Trials were classified by category of ATMP as defined by European regulation EC No. 1394/2007, as well as by development phase and disease area.ResultsThe database search identified 939 clinical trials investigating ATMPs (85% ongoing, 15% completed). The majority of trials were in the early stages (Phase I, I/II: 64.3%, Phase II, II/III: 27.9%, Phase 3: 6.9%). Per category of ATMP, we identified 53.6% of trials for somatic cell therapies, 22.8% for tissue-engineered products, 22.4% for gene therapies, and 1.2% for combined products (incorporating a medical device). Disease areas included cancer (24.8%), cardiovascular diseases (19.4%), musculoskeletal (10.5%), immune system and inflammation (11.5%), neurology (9.1%), and others. Of the trials, 47.2% enrolled fewer than 25 patients. Due to the complexity and specificity of ATMPs, new clinical trial methodologies are being considered (e.g., small sample size, non-randomised trials, single-arm trials, surrogate endpoints, integrated protocols, and adaptive designs). Evidence generation post-launch will become unavoidable to address payers’ expectations.ConclusionATMPs represent a fast-growing field of interest. Although most of the products are in an early development phase, the combined trial phase and the potential to cure severe chronic conditions suggest that ATMPs may reach the market earlier than standard therapies. Targeted therapies have opened the way for new trial methodologies, from which ATMPs could benefit to get early access. ATMPs may be the next source of major impact on payers’ drug budgets.

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confidence: 99%

Advanced therapy medicinal products: current and future perspectives

Hanna

Rémuzat

Auquier

et al. 2016

Journal of Market Access & Health Policy

113

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“…Although various autologous chondrocyte products have recently been approved as “advanced therapy medicinal products (ATMPs)” and therefore underlie regulation comparable to that of medicinal products [40], the minimum effective and the optimum cell/product doses have not yet been determined in a clinical trial. This seems remarkable, as preclinical studies appear to suggest an influence of the cell dose on morphological aspects of cartilage regeneration following cell‐based procedures [12, 25, 26, 28], while in some clinical studies a significant influence of cell viability and quality upon clinical outcome has been reported [33, 34].…”

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confidence: 99%

Safety and efficacy of matrix-associated autologous chondrocyte implantation with spheroid technology is independent of spheroid dose after 4 years

Niemeyer

Laute²,

Zinser³

et al. 2020

Knee Surg Sports Traumatol Arthrosc

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Purpose The aim of this study was to investigate the effect of product dose in autologous chondrocyte implantation (ACI) for the treatment of full‐thickness cartilage defects of the knee and to assess its influence on clinical and morphological mid‐term outcome. Methods Seventy‐five patients were included in this single‐blind, randomised, prospective, controlled clinical trial. Patients were assigned randomly to three different dose groups [low (3–7 spheroids/cm2), medium (10–30 spheroids/cm2), or high (40–70 spheroids/cm2)] and assessed using standardised clinical and morphological scoring systems (KOOS, IKDC, MOCART) for 4 years following the intervention. Results The analysis population comprised 75 patients (22 women, 53 men) aged 34 ± 9 years. Defect sizes ranged from 2 to 10 cm2 following intraoperative debridement. The assessment of the primary variable ‘overall KOOS’ showed a statistically significant improvement, compared with baseline, for each dose group, i.e., at baseline the mean ‘overall KOOS’ scores were 60.4 ± 13.6, 59.6 ± 15.4, and 51.1 ± 15.4 for the low‐, medium‐, and high‐dose groups, respectively, and 57.0 ± 15.2 for ‘all patients’. After 48 months those values improved to 80.0 ± 14.7, 84.0 ± 14.9, and 66.9 ± 21.5 in the respective dose groups and 77.1 ± 18.6 for ‘all patients’. Pairwise comparisons of these dose groups did not reveal any statistically significant differences. Likewise, assessment of the subjective IKDC score revealed no statistically significant differences between the three dose groups up to the 48‐month visit. However, between 12 and 48 months there was a low, but steady, improvement in the low‐dose group and a substantial amelioration in the medium‐dose group. The mean MOCART total scores 3 months after treatment were 59.8 ± 10.9, 64.5 ± 10.3, and 64.7 ± 9.4 for the low‐, medium‐, and high‐dose groups, and 62.9 ± 10.3 for ‘all patients’; 48 months after treatment these were 73.9 ± 13.1, 78.0 ± 12.4, and 74.3 ± 14.0 for the respective dose groups and 75.5 ± 13.1 for ‘all patients’. Conclusions Results of this study confirm the efficacy and safety of the applied “advanced therapy medicinal product”; no dose dependence was found either for the incidence or for the severity of any adverse reactions. All doses applied in the present study led to significant clinical improvement over time and can therefore be regarded as effective doses. The influence of product doses in the range investigated seems to be low and can be neglected. Thus, the authorised dose range of 10–70 spheroids/cm2 confirmed by this clinical trial offers a broad therapeutic window for the surgeon applying the product, thereby reducing the risk of over‐ or underdosing. Level of evidence I.

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