Editorial: The use of real world data for regulatory purposes in the rare diseases setting

por Giannuzzi, Viviana, Stoyanova-Beninska, Violeta, Hivert, Virginie
Publicado 2022

Editorial: Reviews in: regulatory science

por Kerpel-Fronius, Sandor, Stoyanova-Beninska, Violeta, Giannuzzi, Viviana, Sun, Zhonghua
Publicado 2023

Regulatory Standards in Orphan Medicinal Product Designation in the EU

por Tsigkos, Stelios, Mariz, Segundo, Sheean, Maria Elzbieta, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta
Publicado 2021

Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective

por Tomeo, Francesca, Mariz, Segundo, Brunetta, Angelo Loris, Stoyanova‐Beninska, Violeta, Penttila, Karri, Magrelli, Armando
Publicado 2021

Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe

por Farkas, Andreas M., Mariz, Segundo, Stoyanova-Beninska, Violeta, Celis, Patrick, Vamvakas, Spiros, Larsson, Kristina, Sepodes, Bruno
Publicado 2017

Dose‐Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis

por Schuller, Yvonne, Gispen‐de Wied, Christine, Hollak, Carla E. M., Leufkens, Hubertus G. M., Stoyanova‐Beninska, Violeta
Publicado 2018

Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

por Schuller, Yvonne, Biegstraaten, Marieke, Hollak, Carla E. M., Klümpen, Heinz-Josef, Gispen-de Wied, Christine C., Stoyanova-Beninska, Violeta
Publicado 2018

Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation

por Mulder, Jorn, van Rossum, Tobias, Mariz, Segundo, Magrelli, Armando, de Boer, Anthonius, Pasmooij, Anna M. G., Stoyanova-Beninska, Violeta
Publicado 2021

A Comparison of Post-marketing Measures Imposed by Regulatory Agencies to Confirm the Tissue-Agnostic Approach

por Mulder, Jorn, van Stuijvenberg, Odile C., van Hennik, Paula B., Voest, Emile E., Pasmooij, Anna M. G., Stoyanova-Beninska, Violeta, de Boer, Anthonius
Publicado 2022

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

por Aartsma-Rus, Annemieke, Straub, Volker, Hemmings, Robert, Haas, Manuel, Schlosser-Weber, Gabriele, Stoyanova-Beninska, Violeta, Mercuri, Eugenio, Muntoni, Francesco, Sepodes, Bruno, Vroom, Elizabeth, Balabanov, Pavel
Publicado 2017

Extension of Indication for Authorised Oncology Products in the European Union: A Joint Effort of Multiple Stakeholders

por Mulder, Jorn, Verjans, Robin, Verbaanderd, Ciska, Pean, Elias, Weemers, Just, Leufkens, Hubert G. M., Pignatti, Francesco, de Boer, Anthonius, Voest, Emile E., Stoyanova-Beninska, Violeta V., Pasmooij, Anna M. G.
Publicado 2021

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

por Sheean, Maria E., Naumann-Winter, Frauke, Capovilla, Giuseppe, Kalland, Maria Elisabeth, Malikova, Eva, Mariz, Segundo, Matusevicius, Darius, Nistico, Robert, Schwarzer-Daum, Brigitte, Tsigkos, Stelios, Tzogani, Kyriaki, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta
Publicado 2021