Prime – The European approach to expedited pathways

By: Steffen Thirstrup, Director, NDA Advisory Board & Eva Lilienberg, Principal Consultant 


Steffen Thirstrup, Director, NDA Advisory Board

A timely market introduction is a critical component of any drug development strategy – not only from a commercial stand point, where an early introduction can mean beating competition to market or simply significantly increased revenue, but also to patients awaiting better or alternative treatment options.

The debate around how regulators can facilitate this process on both sides of the Atlantic has resulted in new pathways for new medicines of major public interest. The FDA have the Breakthrough/RMAT pathways, and in the EU, EMA has put the PRIME process in place.

In this white paper, Steffen and Eva discuss the different tools available to European regulators and the experiences so far with the PRIME pathway.

To read the full article download the PDF

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How we can help?

NDA Group supports life science companies all over the world with the aim to streamline the global development and commercialization process in order to accelerate patient  access to important medical therapies.

Whatever regulatory hurdle you’re facing, we can help you optimize every regulatory interaction and shape the dialogue about your product to create a more direct path to approval.

To learn more about our services and how we can help click here

Or contact us at meet-us@ndareg.com to talk to one of our experts.

European Regulatory Meetings – how best to prepare and perform

By: Steffen Thirstrup, Director, NDA Advisory Board 

In this article, NDA’s Steffen Thirstrup discusses three important factors to help you prepare for high stakes meetings with EU Regulators.


Steffen Thirstrup, Director, NDA Advisory Board

Having the possibility to engage face-to-face
with EU regulators is the ideal situation to help with authorisation of your product. However, most interactions in the EU are in writing and very few sponsors have the opportunity for a meeting with the EMA.

The most critical meetings are those where the scientific committees of the EMA would like to question the sponsor to fully understand the data and the sponsor’s position.

Such ‘high-stakes’ meetings like CHMP oral explanations are often the final chance for a pharmaceutical sponsor to convince the EU regulators that the benefit/risk balance of their product is positive.

We recommend that you apply the pertinent Benjamin Franklin quote,throughout your planning for a successful EU ‘high-stakes’ meeting.

“By failing to prepare, you are preparing to fail”

1.  Be prepared

Being well-prepared and performing professionally is the key to success. The sooner you can establish your team the better. Having your process and timelines mapped out will help prepare your team and maximise the chances of success. Plan for rehearsal sessions where you can work on the presentation, back-up slides and train comprehensively for questions and answers. If time allows, the team can be further coached by bringing in a ‘challenge panel’ of external experts who can act as a test-panel for your presentation and Q&A performance.

 

2. Know your data

EU regulators expect meetings like an oral explanation to be a scientific discussion based on data. To ensure this is the case, you should demonstrate intimate knowledge of your data to meet the regulators’ concerns as well as being able to present your position in a confident manner. To get to this stage takes meticulous preparation of key messages, slides and the script for the presentation and for potential questions.

One pitfall is to make use of already prepared slides as they will have been prepared with another aim in mind. Starting with a blank piece of paper, writing down the key messages that you believe the regulators need to hear gives you the best basis for creating slides that convey your key messages.

It is very important to make your slides as simple as possible, omitting any unnecessary information and this can be a challenge. Complicated slides distract the audience from your presentation; remember that the slides are there as an ‘aide memoire’ and not for the regulators to read verbatim as they will then not be listening to you!

In addition, keep graphics as simple as possible. A rule-of-thumb is a maximum of six points per slide with no more than six words in each.

 

3. Know your team

In many cases it makes sense to create sub-teams to address the individual topics, agree on key messages and create the slides. Group sessions can then be used to merge the slides into one presentation, optimise the script and ensure all information is in keeping with the key messages.

Additionally, there may be a need to coach key opinion leaders and/or patient representatives to speak or respond to questions on your behalf.

Finally, determining potential questions and being able to respond clearly and unequivocally to these are equally important to increase your chances of success. One way of doing this is to create a Q&A grid where all members of your team add likely questions, and questions they find difficult to answer. The team can prepare top-line responses, a more detailed explanation and any supporting slides for these potential questions and, of course, determine who is going to respond if the question is raised.

 

How we can help?

NDA Group supports life science companies all over the world with the single aim to streamline the global development and commercialization process in order to accelerate patient  access to important medical therapies.

In Europe, we assist with Scientific Advice Group meetings, Oral Explanations, PRAC meetings and preparation for intense pricing negotiations. In the US, our Principals and Communications Strategists are particularly experienced at preparing for FDA Advisory Committee meetings, in addition to pre-IND,  end of phase 2, pre-NDA/BLA, PMA, and mid- and late-cycle review meetings. We offer our clients an unparalleled breadth of global experience and expertise to drive efficient product development and health authority interactions across the US and Europe.

Whatever regulatory hurdle you’re facing,  we can help you optimize every regulatory interaction and shape the dialogue about your product to create a more direct path to approval.

Article originally published in the Jan/Feb 2019 issue of Pharmafocus

Click to original article

In memoriam – Dr Eric Abadie

We were all greatly saddened to learn of the recent passing of our colleague and friend Dr Eric Abadie.

Eric joined the NDA Advisory Board five years ago after a remarkable career as one of the world’s most influential regulators. He brought with him his amazing knowledge and experience, working diligently to support the development of novel and life changing medicines.

There are many ways to honour the memory of a colleague and we had planned to write about Eric’s career at the CHMP and EMA and of course the French Medicines Agency. However, when we reached out to those who have worked most closely with Eric over a long period of time, we were inundated with personal memories. Out of all of these we have picked this story from his long-standing colleague Dr Mira Pavlovic-Ganascia, which we feel encapsulates Eric’s personality. It exemplifies his profound knowledge and a passionate dedication to what mattered so much to him – the safety of European patients:

During one Epiphany celebration at the French Medicines Agency ten years ago, Eric Abadie found a small china figure in his piece of cake. With paper crown on his head, he turned to those next to him: “Being assessor and CHMP Chair is like being Henry Fonda in Sidney Lumet’s ‘Twelve angry men’”.

He explained for those who had not seen the film:

A jury of twelve men is appointed to decide the fate of an 18-year old black male, accused of having stabbed his father to death. If there is any reasonable doubt, they are to return a verdict of not guilty; if found guilty, he will receive the death sentence.

In a preliminary vote, and without any deliberation, 11 jurors vote “guilty” except one, juror 8 (Henry Fonda), who doubts. Juror 8 questions the reliability of the only two witnesses and the prosecution’s claim that the murder weapon is “rare”. Juror 8 argues that he cannot vote “guilty” because there is reasonable doubt.

For Eric, the film raised two unanswerable questions:

  1. Is it possible to make adequate decisions that do not depend on any subjective judgment?
  2. How can randomly chosen jurors, who have only a limited view of the facts, decide if a man is to die or not?

Eric continued:
“Regulatory decision making is all about reasonable doubt – the degree of uncertainty. Uncertainty in the knowledge of benefits, both short and long term, and uncertainty in the knowledge of risks. You should all be Henry Fonda, always be aware of the ‘reasonable doubt’, and never stop until your knowledge and understanding of a new drug’s effects, including any related uncertainties, is as complete as possible.

Thank you, Eric. Thank you for all your contributions, for your integrity, your passion and for setting an example for the rest of us to follow. You will be sorely missed.

Your friends at NDA

 

 

 

Prof. Steffen Thirstrup co-chairs biosimilars at EU DIA Biosimilars Workshop November 21-22 in Dublin, Ireland

Medical Advisor and NDA Advisory Board Member, Steffen Thirstrup (MD, PhD) is taking part in a DIA event titled “Biosimilars workshop – European experiences and challenges” in Dublin on 21-22 November. Steffen Thirstrup has been the chair of the organizing committee of this workshop.

This 1.5 day workshop will focus on the European experience with biosimilar medicinal products, giving an overview of the current regulatory and market-access situation, lessons learned, together with the challenges faced by two classes of products, Erythropoietin (EPO) and Granulocyte-Colony Stimulation Factor (G-CSF).

For more information, please click here

Prof. Steffen Thirstrup speaks at DIA Biosimilars 2013 November 13-14 in Bethesda, Maryland, USA

Medical Advisor and NDA Advisory Board Member, Steffen Thirstrup (MD, PhD) is attending and speaking at the DIA Biosimilars Conference on 13-14 November in Bethesda, Maryland, USA. Steffen will run a session on ‘Market experience in EU’, which will address the EU’s approach to biosimilars and his experience of how this works in practice. Steffen was co-chair for the project group on Market Access for Biosimilars under EC’s Platform on Access to Medicines in Europe. To find out more, please click here.

Thomas Lönngren 2013 IPS Medal Winner

Thomas Lönngren, Strategy Advisor at NDA Group, has been elected as the 2013 IPS Medal Winner on behalf of the Executive Committee of the Industrial Pharmacy Section of FIP.
Announced on 4th September at the IPS Business meeting in Dublin, the medal was presented by its Chair, Linda Hakes.
Following the award, Thomas gave a presentation on his experience gained from working at the European Medicines Agency and his thoughts on the future.

ATMPs workshop at 2013 PDA Europe Conference on Advanced Therapy Medicinal Products

Professor Beatriz Silva Lima, PharmD, PhD, Non-Clinical Expert for NDA’s Regulatory Advisory Board, is contributing towards a workshop on ATMPs at 2013 PDA Europe Conference on Advanced Therapy Medicinal Products.

Beatriz will specifically talk about ‘iPS cells, can they replace (some) preclinical studies?’ as part of a broader session on Pre-Clinical Studies.
The PDA Advanced Therapy Medicinal Products Conference provides an opportunity to exchange information and thoughts between the industry and regulators of different areas with the aim to improve the understanding of ATMP development and the regulatory expectations in this new and emerging field of medicinal products.

The event takes place in Florence, Italy, on 25-26 June 2013. For further information, please see: https://europe.pda.org/index.php?n1=665&n2=689&id=911&content=Overview

Dr. Toivonen talked at EURORDIS Summer School

Dr. Markku Toivonen, Scientific Director at NDA Group, held a talk at this year’s EURORDIS Summer School on Medical Research and Clinical Trial Methodology. EURORDIS is committed to empowering people living with rare diseases and set up its annual Summer School to provide training and hands-on experience on clinical trials, drug development and the EU regulatory processes. Around 40 participants took part, selected dependent on their English language skills, experience in rare disease advocacy, and their willingness to be involved in drug development and regulatory affairs at the European level. During Markku’s session on clinical research, he discussed the need for greater evidence-based medicine, the lifecycle of drug development from pre-clinical (specifically of orphan medicinal products) and the stages of drug development. After, Markku followed with a session on clinical trials, specifically looking at the ‘Gold Standards’. This year’s school was held between 17-21 June 2013 in Barcelona. For more information, please see: http://www.eurordis.org/content/eurordis-summer-school-patient-advocates.

Biosimilars in the EU – what have we learned so far?

NDA Group Advisory Board member, Steffen Thirstrup, has written an article in Scrip Regulatory Affairs Magazine Sep 2013 on ‘Biosimilars in the EU – what have we learned so far?’

With the EU set to approve its first two biosimilar monoclonal antibodies, Steffen looks back over the past seven years since the first biosimilars were approved and examines the lessons learnt and the outlook in the EU.

 

Biosimilars by Steffen Thirstrup

This article was published in Scrip Regulatory Affairs September 2013

 

Dr Steffen Thirstrup

Dr Steffen Thirstrup, MD, PhD
Medical Advisor, NDA Group

Previously Head of Division, Medicines Assessment and Clinical Trials at the Danish Health and Medicines Authority. During his 9 years with the regulatory agency in Denmark, Steffen held several significant roles including 5 years as CHMP member, 1 year as a member of the Committee for Advanced Therapies (CAT), Chairman of the CHMP Respiratory Drafting Group and Co-Chair of the EC Working Group on Market Access of Biosimilars under the EU Platform on Market Access of Medicines in EU. Steffen is a medical doctor, has a PhD in pharmacology and has a broad clinical background in general internal medicine with a special interest in respiratory medicine combined with an in-depth knowledge of clinical pharmacology and therapeutics.

He is Board Certified Specialist in Clinical Pharmacology and Therapeutics with the National Board of Health in Denmark. He is also adjunct professor at the School of Pharmacy, Faculty of Health Sciences at the University of Copenhagen.