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Rare Disease Day 2023 is a tricky day for orphan drugs Sharing colours sometimes takes time
I remember very well the day I asked the G-BA for permission to speak about the specific German benefit assessment of orphan drugs. Although I used to represent the G-BA and AMNOG internationally, I did not dare to give a talk solely on orphan drugs. Why? Because I...

Why the G-BA statement on limited resources is favourable to AMNOG 2023 G-BA work programme
The G-BA took the opportunity to announce its work programme for 2023 at a press conference on 14 February 2023. The impartial G-BA Chair professor Josef Hecken highlighted in his introduction speech that the G-BA had made over 1,000 benefit assessment decisions since...

February is a special month for orphan drugs at the G-BA in 2023 Rare Disease Day can come
While the last day of February is known as the Rare Disease Day, the first days of February 2023 mark the start of changes for rare diseases at the G-BA in Germany. On 2 February 2023, the G-BA decided on the provisory suspension of two ongoing benefit assessments for...

November Intermezzo in Spain World Orphan Drug Congress Europe on 14-17 November 2022 in Sitges, Spain
Why does this year’s World Orphan Drug Congress in the province of Barcelona still feel so special? I DID NOT represent my former employer, the Federal Joint Committee (G-BA) in Germany. I DID NOT present any update on the G-BA benefit assessment of orphan drugs under...

What Is a Fair Reimbursement Price for Orphan Drugs?
In the current draft of the Act on the Stabilisation of Finances for the Statutory Health Insurance in Germany, the threshold for orphan drugs shall decrease from currently €50 to €20m. A déjà vu? But wasn’t it €30m last time this was mentioned? Would €25m be a...

Revision of the EU Orphan Legislation – Implications for the German Benefit Assessment RAPS Euro Convergence on 10-12 May 2022 in Amsterdam, Netherlands
Back on stage! This year’s RAPS Euro Convergence in Amsterdam gave Dr. Meriem Bouslouk-Marx the floor to outline the interdependencies of EU and national legislative reviews. She analysed the implications of potential changes of the EU Orphan legislation such as...

Showing my stripes for this year’s Rare Disease Day
Oral and maxillofacial surgery was one major discipline during my last years of dental medicine at the Friedrich Schiller University in Jena. I was intrigued by the strange names of diseases and syndromes such as Hodgkin's, non-Hodgkin's and Behçet's. I remember very...

How can Germany pay for innovative therapies in the future? Fair access to medicines in the future – A challenge not only in times of corona. 11 March 2021, online event
With Advanced Therapy Medicinal Products (ATMPs), especially those for the treatment of rare diseases – innovative but also very expensive therapies – entering the market, a new challenge for payers has arisen. How can innovative medicinal products be financed in the...

A global fund to tackle antimicrobial resistance (AMR) New initiative takes on the antibiotic innovation challenge: Global launch event on 9 July 2020, online event
The International Federation of Pharmaceutical Manufacturers & Associations IFPMA, the European Federation of Pharmaceutical Industries and Associations EFPIA and the German Association of Research-Based Pharmaceutical Companies vfa organised an online event to launch...

How does Brazil deal with advanced therapies? Gene and advanced therapy – Creating value in the patient journey and in the health system on 23-24 October 2019 in Brasília, Brazil
Following the invitation of Sindusfarma (Syndicate of the Pharmaceutical Products Industry in the State of São Paulo) and Anvisa (Brazilian Health Regulatory Agency), stakeholders met to discuss Brazil’s challenges with advanced therapies. The two days with...