advanced therapy medicinal products - atmps - a case study in health economics: chondrocelect...
TRANSCRIPT
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Advanced Therapy Medicinal ProductsAdvanced Therapy Medicinal Products- ATMPs -- ATMPs -
A case study in health economics: ChondroCelect
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A representative sample of the slides that will be presented and discussed during the course is displayed hereafter.
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ATMP’s
ATMPs include:Gene therapy medicinal products
Somatic cell therapy medicinal products
Tissue-engineered products
Centralised procedure registration
Principles of existing legislation on medicines apply:Marketing authorisation
Demonstration of Quality, Safety and Efficacy
Post-authorisation vigilance
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Unique features in the ATMP regulation
Risk-based approach to determine level of data
Post-authorisation follow-up of safety and efficacy
Incentives and fee reductions
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ChondroCelectChondroCelect
First cell-based medicinal product
authorized under the European ATMP framework
(Regulation 1394/2007)
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ChondroCelect as medicinal product:
Autologous cell-based medicinal product (EU/1/09/563/001)
Characterised autologous cartilage cells expanded ex vivo expressing specific marker proteins
Repair of single symptomatic cartilage defects of the femoral condyle of the knee (ICRS grade III or IV) in adults.
Active substanceActive substance
IndicationIndication
Controlled manufacturingQuality control
ImplantationBiopsy
ProductProduct
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ChondroCelect – ATMP development
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Underlying research(ECFA-Assay and ChondroCelect molecular markers)
Underlying research(ECFA-Assay and ChondroCelect molecular markers)
Pre clinical testing & validationPre clinical testing & validation
Phase III Clinical trialPhase III Clinical trial
Compassionate use in 370 patientsCompassionate use in 370 patients
Marketing authorization application at EMA*
Marketing authorization application at EMA*
EC ApprovalEC Approval
Year
2001 20021994... 2003 2004 2005 2006 2007 2008 2009
Outcome of 15 years of R&D > 500 patients treated till dateOutcome of 15 years of R&D > 500 patients treated till date
2010...
Post MA Follow-upPost MA Follow-up
*EMA : European Medicines Agency
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CC clinical evidence:superior structural repair at 12 months
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ChondroCelect
Microfracture
The American Journal of Sports Medicine , Volume 36, No 2, February 2008
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CC clinical evidence:superior clinical benefit over MF over 36 months
9 The American Journal of Sports Medicine , Volume 37 – Suppl. 1, November 2009
KOOS P-value
Overall(1) 0.048
Pain 0.044
Symptoms 0.123
Activity (ADL) 0.064
Sports 0.123
Quality of Life 0.036
Longitudinal analysis: treatment effect at 36m
(mixed linear model)
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Model structure – from model entry to OA onset after 15 years
During the 3 years following the knee intervention (microfracture [MFX] or autologouschondrocytes implantation [ACI] using ChondroCelect [CC]), the symptoms can be reduced(‘Success’) or not; in case of continued symptoms, the patient can experience re-operation(‘Re-op’) or not within 5 years; in case of reoperation, the symptoms can be reduced(‘Re-op success’) or not; regardless of symptom reduction, patients will experience OA (‘long-term [LT] problem’) or not, which is evaluated 15 years after the initial intervention in the model.
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Cost-utility analysis of ChondroCelect (CC) vs microfracture (MFX),base-case and additional scenarios (healthcare payer perspective)
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Acceptability curve of CC vs. MFX, from probabilistic sensitivity analysis
The curve shows the proportion of cost-effective strategies dependingon the willingness-to-pay level. CC is cost-effective in 80% of Monte-Carlo simulations if the payer has a willingness-to-payof 22,000 EUR/QALY.
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