scholarly journals Access to innovation through the national early access program and clinical trials for patients with malignant melanoma

Cancer ◽  
2021 ◽  
Author(s):  
Claire Christen ◽  
Laetitia Belgodère ◽  
Bernard Guillot ◽  
Céline Jumeau ◽  
Annie Lorence ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Malignant Melanoma ◽  
Early Access ◽  
Access Program

Access to innovation through the French Expanded-Access Program and clinical trials in patients with malignant melanoma.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e22030-e22030
Author(s):  
Claire Christen ◽  
Laetitia Belgodere ◽  
Bernard Guillot ◽  
Ghania Kerouani-Lafaye ◽  
Liora Brunel ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Malignant Melanoma ◽  
Early Stage ◽  
Objective Response ◽  
Time Data ◽  
National Agency ◽  
Innovative Drug ◽  
Expanded Access ◽  
Expanded Access Program ◽  
Access Program

e22030 Access to innovation through the French Expanded-Access Program and clinical trials in patients with malignant melanoma Background: The arrival of immunotherapy (IT) and targeted therapy (TT) has constituted a revolution in the treatment of metastatic melanoma (MM) since the 2010s. The major challenge was to allow these new treatments to make them available as soon as possible. France has effective tools such as clinical trials (CT) and an expanded-access program (EPA) (through temporary use authorizations (ATU) and temporary use recommendations (RTU)) allowing the use of innovative drug without marketing authorization (MA) in case of therapeutic “dead-end”. Methods: Real-time data collection of nominal and cohort ATU, RTU (between 01/09/2009 and 01/09/2019), and CT authorizations (from 01/12/2017 to 01/09/2019) filed and evaluated by the French national agency for medicines and health products safety (ANSM) in the MM from internal databases. Clinical data of the cATU come from the summary reports produced by the laboratory. Results: 45 CT were authorized in MM, including 51% in early phases and 44% in phase II/III, mainly in the metastatic stage (86%) and with an industrial promoter (73%). IT and TT (63% and 24% respectively) are the most used in experimental arms, mostly in combination. Through the EPA, 3 RTUs were authorized in the adjuvant treatment of MM, 14 drugs benefited of a nATU and 5 obtained a cATU. This has treated 6538 patients (28% of nATU and 72% of cATU). The anti-PD1 and combination therapy of BRAF and MEK inhibitors are the most used, mainly in combination. 80% of the indications are from the second line. The duration of availability of the ATU is 9,85 months before obtaining the MA. Efficacy data in patients receiving cATU of vemurafenib and combination of cobimetinib and vemurafenib showed respectively the following objective response rate (ORR): at 8 weeks of treatment, ORR = 57,7% and 54,7%; at 20 weeks, ORR = 36,7% and 54,7% (IR criteria). For pembrolizumab, at 12 weeks, ORR = 29,7% and at 24 weeks, ORR = 40% (irRC criteria). All of these drugs obtained a MA and an inscription on the reimbursement list after the health technology assessment. Conclusions: Thanks to CT and the French EPA, patients were able to benefit from innovative treatments at an early stage of MM.

Real-world experience of generalized and partial lipodystrophy patients enrolled in the metreleptin early access program

2019 ◽  
Author(s):  
Keziah Cook ◽  
Anna Stears ◽  
David Araujo-Vilar ◽  
Ferruccio Santini ◽  
Stephen O'Rahilly ◽  
...  
Keyword(s):  
Real World ◽  
Partial Lipodystrophy ◽  
Early Access ◽  
Access Program

scholarly journals Lorlatinib in pretreated ALK- or ROS1-positive lung cancer and impact of TP53 co-mutations: results from the German early access program

2021 ◽  
Vol 13 ◽  
pp. 175883592098055 ◽  
Author(s):  
Nikolaj Frost ◽  
Petros Christopoulos ◽  
Diego Kauffmann-Guerrero ◽  
Jan Stratmann ◽  
Richard Riedel ◽  
...  
Keyword(s):  
Treatment Failure ◽  
Survival Rates ◽  
Real Life ◽  
Progression Free Survival ◽  
Leptomeningeal Carcinomatosis ◽  
Resistance Mutations ◽  
Duration Of Treatment ◽  
Tp53 Mutations ◽  
Early Access ◽  
Access Program

Introduction: We report on the results of the German early access program (EAP) with the third-generation ALK- and ROS1-inhibitor lorlatinib. Patients and Methods: Patients with documented treatment failure of all approved ALK/ROS1-specific therapies or with resistance mutations not covered by approved inhibitors or leptomeningeal carcinomatosis were enrolled and analyzed. Results: In total, 52 patients were included [median age 57 years (range 32–81), 54% female, 62% never smokers, 98% adenocarcinoma]; 71% and 29% were ALK- and ROS1-positive, respectively. G1202R and G2032R resistance mutations prior to treatment with lorlatinib were observed in 10 of 26 evaluable patients (39%), 11 of 39 patients showed TP53 mutations (28%). Thirty-six patients (69%) had active brain metastases (BM) and nine (17%) leptomeningeal carcinomatosis when entering the EAP. Median number of prior specific TKIs was 3 (range 1–4). Median duration of treatment, progression-free survival (PFS), response rate and time to treatment failure were 10.4 months, 8.0 months, 54% and 13.0 months. Calculated 12-, 18- and 24-months survival rates were 65, 54 and 47%, overall survival since primary diagnosis (OS2) reached 79.6 months. TP53 mutations were associated with a substantially reduced PFS (3.7 versus 10.8 month, HR 3.3, p = 0.003) and were also identified as a strong prognostic biomarker (HR for OS2 3.0 p = 0.02). Neither prior treatments with second-generation TKIs nor BM had a significant influence on PFS and OS. Conclusions: Our data from real-life practice demonstrate the efficacy of lorlatinib in mostly heavily pretreated patients, providing a clinically meaningful option for patients with resistance mutations not covered by other targeted therapies and those with BM or leptomeningeal carcinomatosis.

Booster-Effect with Velaglucerase Alfa in Patients with Gaucher Disease Switched from Long-Term Imiglucerase Therapy: Early Access Program Results from Jerusalem

2012 ◽  
Vol 105 (2) ◽  
pp. S28-S29
Author(s):  
Deborah Elstein ◽  
Gheona Altarescu ◽  
Hannah Maayan ◽  
Mici Phillips ◽  
Aya Abrahamov ◽  
...  
Keyword(s):  
Gaucher Disease ◽  
Early Access ◽  
Access Program ◽  
Booster Effect

Clinical Trials on Chemical Radiosensitization of Malignant Melanoma of the Choroid

1979 ◽  
Vol 178 (4) ◽  
pp. 194-197 ◽  
Author(s):  
H. Żygulska-Mach ◽  
Z. Maciejewski ◽  
S. Lukiewicz ◽  
B. Iwasiów ◽  
E. Link
Keyword(s):  
Clinical Trials ◽  
Malignant Melanoma

First experience of Nusinersen early access program in patients with spinal muscular atrophy type 1

2017 ◽  
Vol 27 ◽  
pp. S211-S212 ◽  
Author(s):  
E. Gargaun ◽  
K. Aragon-Gawinska ◽  
A. Seferian ◽  
T. Gidaro ◽  
S. Gilabert ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type ◽  
Early Access ◽  
Access Program

Booster-effect with velaglucerase alfa in patients with Gaucher disease switched from long-term imiglucerase therapy: Early Access Program results from Jerusalem

2012 ◽  
Vol 48 (1) ◽  
pp. 45-50 ◽  
Author(s):  
Deborah Elstein ◽  
Gheona Altarescu ◽  
Hannah Maayan ◽  
Mici Phillips ◽  
Aya Abrahamov ◽  
...  
Keyword(s):  
Gaucher Disease ◽  
Early Access ◽  
Access Program ◽  
Booster Effect
Sign in / Sign up

Export Citation Format

Share Document