Dr. Pieter Neels Talks Cervical Cancer Vaccines

NDA Advisory Board member, Dr Pieter Neels, is presenting at the ‘Perspective on benefit-risk decision-making in vaccinology’ event being held on 23 – 25 June 2014, at Les Pensières, Annecy (France). The event is being run by Fondation Mérieux, with the aim to bring together experts and interested individuals from various disciplines to:

  1. Explore through examples, the specificities of benefit-risk evaluation of vaccines according to different perspectives
  2. To discuss data needed and methodologies for analysis of benefit-risk in vaccine development and in post-marketing surveillance of vaccination programs in terms of respective contributions, complementarities, limitations
  3. From the two previous aspects, recommend ways to maximize B/R of vaccine and vaccination program


Dr. Neels will present a case study on Human papilloma virus Vaccination, a Benefit Risk balance evaluation. He will discuss vaccines for cervical cancer, its cause and outlook with new vaccines.







Dr. Pieter Neels, NDA Group Clinical Expert and Advisory Board member

Key considerations to achieve market access in Europe

By Dr. Thomas Lönngren

EU-quote-markerRegulatory agencies are no longer at the forefront of deciding if patients get access to new medicines. A multitude of national agencies and organisations have risen over the past years with the purpose of evaluating the value of new medicines.
These systems are now a fact in the EU where different organisations are making individual decisions on whether pharmaceutical products will be allowed to pass through the public health care system or not in the region where they are responsible. Today it is ultimately these bodies that decide if a product will reach the patient.

Uncertainty demands more intelligence sooner

What consequences does this have on drug development and how should we act to ensure we take these changes into account in the development process?

The development of new medicines is now regulated by requirements from regulatory authorities as well as from HTA (Health Technology Assessment) bodies. The shift to increased payer power and the increased rate of change on the requirements affecting drug development because of this shift are making the process increasingly unpredictable. Therefore, there is a need for sound intelligence on how to optimize the development program to satisfy both the regulatory and the HTA requirements. This intelligence is hard to build up and maintain within companies, particularly for smaller and mid-sized companies. Consequently, companies experience a great need to seek external advice from both HTA/regulatory agencies, in the form of formal scientific advice, as well as from third party providers specialized in this field like NDA.When the business opportunity and the patient need is so clear in Europe, the critical question is: How do you best ensure regulatory success to reach patients in the EU?

The successful companies are gradually building the combined intelligence into their decision making process, integrating it as early as possible. This is done with the understanding that if it is left for too long, until after design of the phase II and III studies, it could lead to a total rejection from HTA bodies with the medicine standing without reimbursement or with a price set at the level of a generic.




Meet NDA at DIA 50th Annual Meeting, San Diego Convention Center







NDA will have a strong representation at this year’s DIA US, which celebrates its 50th Annual Meeting, taking place on 15-19 June in San Diego.

Located on Booth 1500, NDA will have leading experts on hand to discuss any EU regulatory, pharmacovigilance or HTA requirements, including the former head of the European Medicines Agency, ex MHRA and gene therapy experts.

Meet the Experts

Here are just some of the experts that will be in attendance and available for meetings:

  • • Thomas Lönngren – former head of the EMA and NDA strategic advisor
    • Lars-Helge Strömquist – CEO of NDA US
    • Shelley Gandhi – Pharmacovigilance expert, former MHRA
    • Dr. Gopalan Narayanan – Gene Therapy expert and NDA Consultant


Whether you are interested in a meeting or an informal chat, please email Anna Perrin: anna.perrin@ndareg.com

Hear the Experts

We also have two speakers during the five day event discussing hot issues around pharmacovigilance and gene therapy:


Shelley_GandhiShelley Gandhi
Title: The Pharmacovigilance System Master File: Business as Usual?
Day: Tuesday, June 17
Time: 3:30PM – 5:00PM (Pacific Standard Time)




Gopalan_NarayananGopalan Narayanan
Track 04 – Nonclinical and Translational Development/Early Phase Clinical Development
Title: Gene Therapy Symposium
Day: Tuesday, June 17
Time: 3:30PM – 5:00PM (Pacific Standard Time)



Track 17 – Rare/Orphan Diseases
Title: Optimizing Orphan Drug Development and Using Appropriate Methodology: Key Tips for Success
Day: Wednesday, June 18
Time: 1:30PM – 3:00 PM (Pacific Standard Time)

Meet us at the Euro meeting

To book a meeting please email Anna Perrin: anna.perrin@ndareg.com or visit us at Booth No. 1500.
We look forward to hearing back from you about a meeting and to seeing you at the event!

Dr. Steffen Thirstrup speaks at EULAR 2014 congress

Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board, is speaking at EULAR (European League Against Rheumatism) 2014 congress in Paris on 12th June.

During the session, sponsored by Sandoz, and titled “Expanding treatment horizons for patients with rheumatic diseases”, Steffen will present on Biosimilars: regulatory perspectives and requirements.








Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board


Dr. Thirstrup speaks at 19th Congress of the European Haematology Association

Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board, is speaking at the 19th Congress of the European Haematology Association in Milan on 11th June 2014.

Steffen will present on Regulatory Perspectives on the Approval of Biosimilars during the Sandoz sponsored programme on “Biosimilars in hematology: increasing choice, expanding access”

According to the organisers: “The sessions aim to offer medical education in a new format where pharmaceutical companies in cooperation with academic or scientific institutes can invite speakers to present on the latest updates and information.”

During the meeting, they will discuss: “the development of biosimilars, with particular focus on biosimilar rituximab. We will detail biosimilar development from a regulatory perspective, as well as provide clinical perspectives on the current and future use of biosimilars. We will also discuss the role of biosimilars in helping reduce healthcare costs and expand patient access.”







Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board


How should PV legislation impact your development plans for new medicines?

By Dr. Brian Edwards

The idea that pharmacovigilance only concerns the reporting of suspected adverse reactions to regulatory agencies has long gone. As stated in the Implementing Regulation (EU) No 520/2012 of 19 June 2012, pharmacovigilance activities cover the whole life-cycle management of medicinal products in relation to safety. But in reality what does that mean and how should a pharmaceutical company respond?

Put simply, what it means is that the good pharmacovigilance practice modules apply to the safety system for a medicine as soon as it is put into man. Of course, these modules need to be adapted for an investigational medicine and the needs of good clinical practice. Indeed, we believe these modules may well help with a risk-based approach to GCP. This means pharmacovigilance professionals must have a more active role in the design of development programmes and clinical trials helping MAHs move towards proactive benefit risk evaluation throughout the product lifecycle.




Dr. Brian Edwards presents at Risk meeting at UMC, Uppsala, Sweden

Dr. Edwards is talking about systematic approaches to personalised risk management and is on a discussion panel concerning latest approaches to managing risk from medicines and what improvements are needed at Uppsala Monitoring Centre Research Conference 2014 May 22-23, Uppsala Concert and Congress Center, Uppsala, Sweden.

This symposium will showcase some of the latest methods and fields of enquiry in safeguarding patients.

The topics to be debated will include:

  • • Patient groups at risk – genotypes and phenotypes
    • Structure analysis of medicinal products
    • Individualized benefit-risk assessment
    • Effective communication to health professionals and patients in clinical settings


This programme will intrigue and excite all those with an interest in patient safety, the risks and benefits of medicines, and the practice of pharmacovigilance.








Dr Brian Edwards Principal Consultant, Pharmacovigilance & Drug Safety, NDA Group

Dr. Steffen Thirstrup chairs panel debate at University of Copenhagen

Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board, is chairing a panel debate at the Faculty of Health and Medical Sciences, University of Copenhagen on 19th May.

The overall event is on New trends in Regulatory Science – trends, options and ideas.
Steffen will chair a debate on ‘Regulatory Science – future opportunities’, alongside Merete Schmiegelow, EU Regulatory Advocacy, Regulatory Policies & Intelligence, Novo Nordisk A/S.

In addition, NDA’s Strategic Advisor and former head of the EMA, Dr. Thomas Lönngren, will also speak at the same event on the subject of the ‘EU Perspective’.








Dr. Steffen Thirstrup, Director of NDA’s Regulatory Advisory Board

Paul Chamberlain presents at SFPT

Biopharmaceuticals Expert on NDA’s Regulatory Advisory Board, Paul Chamberlain, is presenting at the scientific meeting of the French Society of Toxicologic Pathology (SFPT) in Lyon on 16th May. The event is on “Biopharmaceutical products and alternative therapeutics – Update and perspectives”.

Paul will be giving a presentation entitled: “Role of pre-clinical data for the immunogenicity risk assessment of therapeutic proteins.” The aim of this presentation is to ‘illustrate the value of pre-clinical data for assessing immunogenicity-related risks of investigational biopharmaceutical products, in emphasizing mechanistic aspects that could be relevant for overall clinical benefit vs. risk’

The “Société Française de Pathologie Toxicologique” (French Society of toxicologic pathology) is a non-profit association. The members of the SFPT are scientists working in the field of toxicologic pathology.








Paul Chamberlain, NDA Group Biopharmaceuticals Expert and Advisory Board Member


Adaptive licensing – How are regulators supporting innovation?

By Johan Strömquist Published in Mednous May 2014

This commentary by NDA CEO Johan Strömquist on the development of novel regulatory pathways such as the FDA breakthrough designation and the EMA adaptive licensing concept, was published in the May issue of MedNous.

Drug development has become an increasingly timeconsuming and costly exercise. In fact, the cost of new medicines is continuing to escalate just as the public is becoming more aware of the need to have new agents to treat serious diseases. The pressure is therefore on to find new ways to deliver innovation – and to deliver it efficiently.

The drug regulator stands in a pivotal position in this debate. Regulators have a legal responsibility to protect the public health by making sure that new medicines meet rigorous standards of safety and efficacy both before and after market launch. Recently, both the European Medicines Agency and the Food and Drug Administration have been employing some vision to try and facilitate innovation as well. Thus far, the US has been the most pro-active.

In July 2012 the FDA introduced a new regulatory pathway called the ‘breakthrough therapy designation’ that builds on a pre-existing pathway called the ‘fast track designation.’ Both pathways allow the faster review of products with a potential for treating serious or life-threatening conditions. Of the two, the breakthrough designation is clearly targeted at innovation.

In order to obtain breakthrough status, a company must provide preliminary clinical evidence that its drug may demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoint, such as showing substantial effects of treatment during clinical development. Similarly fast track designation is intended for drugs that would fill an unmet medical need.

However fast track drugs need only show some advantage over available therapies – not the substantial improvement required for breakthrough designation. This is a subtle but important difference.

Relevant links





Dr. Pieter Neels speaks at the 15th ADVAC, 12-23 May

Dr. Pieter Neels speaks at the 15th Annual ADVAC (Advanced Course in Vaccinology) between 12-23 May. This is the most prestigious course in vaccinology, inviting just 65 people worldwide to take part in this annual event.

Dr. Neels is invited during the first week (12-17/5) of the training course in Advanced Vaccinology to support the teaching team with regulatory expertise and to give critical advices and questions to help people to understand the regulatory process and clinical development of vaccines.

During the five day event there are several workshops being run, including an exercise in developing a new vaccine. Pieter will also take part in panel discussions on Regulatory Considerations and ethical considerations (placebo vs comparator vaccine in clinical trials).








Dr. Pieter Neels, NDA Group Clinical Expert and Advisory Board member

Thomas Lönngren speaks at four biotech events in USA in May and June

Thomas Lönngren is speaking at four biotech events in USA in May and June. He started out at the Chief Medical Officer East Conference in Boston this week (6th May), followed by the R&D Summit in New York on 8th May, and finishes this week at the McKinsey Regulatory Panel on 8-9th May in Boston. Then again in June, Thomas will be presenting at the Innovation & Strategy Summit at the GLG Institute in San Francisco on 19-20June, shortly after participating in the 50th Annual DIA Meeting in San Diego.

Whilst at the Chief Medical Officer East Conference in Boston and the GLG Institute in San Francisco, Thomas is discussing the EU Landscape, specifically looking at what are the regulators looking for and what are the payers looking for in Europe. However, at the McKinsey Regulatory Panel, he will be part of a panel discussing ‘Global Regulatory Alignment: Impossible or a Possible Dream?’ Moderated by McKinsey, Thomas will sit alongside Vincent Ahonkhai, M.D., Bill and Melinda Gates Foundation, William Chin, M.D., Pharmaceutical Research and Manufacturers of America, Barry Gertz, M.D., Ph.D., Merck Research Laboratories; and Theresa Mullin, Ph.D., FDA.









Thomas Lönngren, Strategic Advisor NDA Group

4 points to ensure success in Europe

By Johan Strömquist

EU-quote-markerOver the years NDA Group has had the privilege of contributing to an increasing number of drug development success stories. We supported over 45% of the new products approved in the EU in 2013, with strategic input at critical points in the development plan aligned with full submission support. We’ve also contributed significantly to over 64 different major regulatory submissions over the past five years.

Through our exposure to and long experience with a large number (500+) of different life science companies from all over the world at NDA we’ve identified a number of critical points that have a significant impact on the success rate of any drug development program aiming for Europe. This experience is particularly relevant for US drug development companies interested in approaching the EU.

Why Europe?

The EU population comprises in excess of 500 million people compared to the US’ 318. Together, the EU countries form the second biggest market in terms of revenue for the pharmaceutical industry. In spite of the per capita health care expenditure in the US being significantly higher than that in the EU, the sheer number of treatable patients means that many products would double their revenue as soon as they’re introduced in the EU.

With a larger, and aging, population there will also naturally be more patients in need of good products. Most developing companies with a passion for improving the lives of patients will eventually have to take a global approach to product development and marketing. The first step for a US drug developer to go global is often spelt “EU”.

When the business opportunity and the patient need is so clear in Europe, the critical question is: How do you best ensure regulatory success to reach patients in the EU?

At NDA we’ve identified a large number of pit falls and challenges facing US based drug development companies as they start considering entering the EU market. These challenges vary between therapy areas, technology platforms and patient populations but some high level characteristics can be identified which are associated with companies who have successfully negotiated the complex EU framework, often with our support, and have planned and executed their program in order to be harmonised to meet both FDA and EMA requirements.





Dave Gilbert presents at Europe 101 – Opportunities for Collaboration & New Markets

DGDave Gilbert, member of the Advisory Board, has been invited to speak at MassBio forum Europe 101 – Opportunities for Collaboration & New Markets in Cambridge, MA 10 April.

Europe is the second largest pharmaceutical market after North America. With a strong academic base, a highly skilled workforce, and a number of government incentives, Europe continues to be an important territory in any biopharmaceutical company strategy as a venue for collaboration at all stages of drug development and as a market for products.
At this forum local and European experts share insights on important European business areas such as product approval, reimbursement and pricing, IP protection, entrepreneurship, financing, and commercialization policies.

To read more and to register click here

To find out more about Dave Gilbert and NDAs Advisory Services, please click here

Paul Chamberlain to present at FIP / PSWC

Paul Chamberlain, a member of NDAs Regulatory Advisory Board, will be presenting at this years symposium at 5th FIP Pharmaceutical Sciences World Congress on Tuesday 15 April, held in Melbourne, Australia. The event attracts leading pharmaceutical scientists from around the world and will focus on ‘Pharmaceutical Sciences beyond 2020 – The rise of a new era in healthcare’.

Paul is involved in two sessions at the event. He will present on the ‘Multidisciplinary approach to evaluating immunogenicity of biosimilars: Lessons learnt and open questions based on 10 years experience of EU regulatory pathway’, as well as chairing a roundtable on ’Interchangeability of biosimilars – Yes or no?’ The roundtable will involve four other panellists: Anita O’Connor, (USA); Paul Declerck, (Belgium); Simon Roger (Australia); and Freddy Faccin (USA).
Paul is a biopharmaceuticals expert for NDA’s Regulatory Advisory Board. He was formerly a senior director at MDS Pharma Services, head of quality control at Metris Therapeutics and EU regulatory manager at Amgen Europe. Paul is an expert in CMC and preclinical biopharmaceutical product development and immunogenicity risk assessment.

To find out more on the event, please click here

To find out more about Paul Chamberlain and NDAs Advisory Services, please click here