Meet the NDA Group at DIA

Meet the NDA Group at DIA in San Diego 23-27 June

Optimising the development of innovative medicines

NDA is a world leading drug development consultancy helping clients achieve regulatory and market access success in the US and EU.

Our goal is to streamline the global development and commercialisation process in order to accelerate patient access to important medical therapies.

Over the last 5 years, NDA Group has supported the approval of over 40% of new medicinal products in the EU and among our FDA advisory committee clients 80% have achieved product approval. We work across a range of disease areas including oncology, hematology, infectious disease, metabolic and immunologic disorders and CNS and have supported the development of over 50 rare disease programs.


Lisa Peluso
Director, Coaching and Client Engagement
Kaia Agarwal
Strategic Advisor and Global Regulatory Affairs Consultant

(Pre-Conference Short Course)
Short Course 3 | Sun, 23rd June- 09:00-12:30
Preparing for a US FDA Advisory Committee Meeting

More information

Lisa Peluso
Director, Coaching and Client Engagement
Steffen Thirstrup
Director NDA Advisory Board

(Pre-Conference Short Course)
Short Course 3 | Sun, 23rd – 13:30-17:00
European Regulatory Meetings: How Best to Prepare and Perform

More Information

Brian Edwards
Principal Consultant, Pharmacovigilance & Drug Safety

Session #106 | Mon, 24th June- 11:00-12:00
Moving forward in EU Pharmacovigilance

Session #155 | Mon, 24th June- 15:30-16:30
Incorporating Systems: Theory and Human factors into the Investigations of Serious Harm in Clinical Research

Session #403 | Thur, 27th June- 09:00-10-15
Successes and Challenges in Pharmacovigilance for Biologics and Biosimilars

NDA supported over 40% of the new medicinal products that were approved in the EU during the last five years.

Do you want your product to reach the world’s two largest markets with minimum delay?

Arrange to meet us at the DIA USA now.


Abraham Lincoln’s Jury Lawyering: Lessons for Sponsors Before FDA Advisory Committees

By John Ellis, Communications Coach

Recently, while reading “Lincoln’s Last Trial” by Dan Abrams and David Fisher, I was struck by strong parallels between Lincoln’s famously successful jury lawyering and the ways successful sponsors communicate with panelists at FDA advisory committee meetings:

  • First, Lincoln kept a laser-like focus on those one or two issues most likely to determine the jurors’ votes.
  • Second, he did everything in his power to keep the jurors’ minds and hearts open; he built a relationship with them as colleagues in a common enterprise; he didn’t pick needless fights; he treated everyone—from witnesses to prosecutors to judges—with respect.
  • Third, he marshaled the facts into a clear and compact narrative that embodied his interpretation of those facts in a way the jurors could easily follow and remember.

Here are some excerpts from the book (in red). While some of the aspects of what Lincoln did in the courtroom and what a sponsor needs to do at White Oak are not completely interchangeable, their similarity in spirit is striking.

  • [Just before his summation]: “Abraham Lincoln spent several seconds straightening the papers on the table in front of him, took a moment to fix his stock [tie], then stood…and faced the jury. And smiled…When [he] approached the jury box and leaned over close, he was just talking to some friends.”
    • Lincoln gave the jury a moment so they could give him their full attention. For any of us to have a chance of persuading people, they must first be giving us their full attention. 
    • Also, it’s remarkable how quickly all of us can forge feelings of friendly respect with others when we’re bound together in a common purpose.
  • “…weaving facts and emotions into a plausible tale….”
    • When creating a persuasive argument, facts and emotions are not either/or. The trick, as Aristotle well knew, is to make facts and emotions reinforce each other.
  • “…people simply liked him.”
    • A gift? Yes. But it was also a reflection of the attitude he radiated.
  • “He showed jurors a different way of looking at the same facts….”
    • This, of course, is the nub of it.
  • “[M]ost cases, he knew, turned on one significant point. He willingly conceded those points he couldn’t win or that made little difference, building up goodwill with the judge and jury that might pay off when he pounced on the salient issue.”
    • What to say when becomes much clearer when you’re focused on the verdict or the vote.
  • [Lincoln once gave this advice to his partner, Herndon:] ‘Billy, don’t shoot too high; aim lower and the common people will understand you. They are the ones you want to reach. The educated and refined people will understand you, anyway. If you aim too high, your idea will go over the head of the masses and hit only those who need no hitting.’
    • While there aren’t, in Lincoln’s sense, ‘common people’ among FDA staffers and panelists, there are always salient differences in educational and professional experiences whenever an Advisory Committee is convened. So, speak in a way that includes everyone–clear, concise, and compelling, using easily grasped comparisons for highly technical points.
  • “Lincoln rarely attacked a witness, believing the damage done to his relationship with the jury by that action might be more harmful than any gain from disputing facts. These were all friends and neighbors, good people, and it was taken for granted they were telling the truth. But even good people tended to remember things different, ‘specially when there was a whole hullabaloo going on around them.’”
    • For “friends and neighbors,” read the panelists and FDA staffers (whether physicians, statisticians, etc.). For “telling the truth,” read interpreting and conveying the data the way they understand it. For ‘hullabaloo,’ read a highly complex data set.
  • “…in his summation, Lincoln’s real focus was on those few issues that made all the difference in the outcome…Slowly he wound his way into the core of the case, moving step-by-step away from the facts into his interpretation of them.”
    •  In a similar way, no therapy, or its clinical development program, is perfect. You keep minds open by acknowledging that. At the same time, you know that what influences votes is not so much the facts as what committee members think the facts mean.
  • “[As the summation proceeded] his voice grew louder and was colored by emotion. His words cut through the calm he had created. Lincoln had laid the groundwork, he had done the legal job, now he was going for the heart….[Yet] this wasn’t a performance; this wasn’t an attempt to manipulate emotions. Lincoln meant every word he said.”
    • May it be so for all of us.

Europe vs USA: new drug product approvals in 2018

By Anna Leitgeb, Consultant, NDA GroupB

2018 was another exceptional year for the life science industry with a total of 103 new therapeutic drugs (NTDs) approved in EU and US.

Every year NDA reviews the NTD approvals in EU and US from previous year to spot trends and assess the year that has passed. The data is taken from the FDA and EMA websites on the new approved products during 2018 (i). In this review we include NTD product approvals with new active substance (chemical, biological, biotechnology or radiopharmaceutical substance), new biological entity, new drug combinations, biosimilars, new active ingredients and vaccines, but excluded generic and duplicate applications.

The following summary provides an overview of the key findings and an analysis of what the data means for the industry. The data is visually represented in an infographic below.

This year the regulators have ruled positively on some high-profile and high-stakes project. Important new drugs for indications with unmet medical need, for neglected diseases or where exciting new technologies are explored have been approved within the area of neurology (Aimovig, Emgality, Ajovy), infectious disease (Xofluza, Trogarzo),and women health (Orilissa). Important orphan drugs were also approved within neurology (Namuscla, Epidiolex, Onpattro, Tegsedi) and hematology (Crysvita) and advancements within precision medicine have been achieved within oncology (Vitrakvi).

It’s interesting to note that the number of NTD approvals in the two markets has not changed markedly from 2017 to 2018. However, the landscape of approvals in the different markets, indications, and company features has moved somewhat over the last 12 months. Out of the 103 approvals 45 were solely FDA-approved and 9 approved only in EU, indicating an increase in dual market approvals compared to 2017.

There was a rise in the number of NTDs approved for both the US and EU markets from 36 in 2017 to 49 in 2018. This indicates that the joint application strategy was more popular than previous years.

Approvals for oncology and infectious disease products increased in 2018 whilst the number of approvals within hematology, neurology and immunology/rheumatology has decreased during the same time frame.


2018 was a significant year for approvals of novel drugs, i.e. treatments based on new active substances. Out of the 103 new drugs, 89 were based on new active substances. This number has increased since the year before when 56 approvals were for novel drugs. This high number of green lights from the agencies follows a few successful years for drug developers. The agencies involvement and support during drug development has increased which also contributes to improvements to strategy rather than to only secure compliance with existing regulations.

After decades of work on migraine prevention drugs finally an antibody-based approach has been approved. Aimovig (Amgen and Novartis) was first approved, and short thereafter came Emgality (Eli Lilly) and Ajovy (Teva). These are self-injected molecules and they all belong to a new class of drugs called calcitonin gene-related peptide receptor (CGRP-R) antagonists. They offer patients treatments that can reduce the number of days with migraine.

Other standouts include new drugs to treat infectious diseases. Xofluza (Roche), a polymerase acidic endonuclease inhibitor, is the first novel flu drug to reach the market in 20 years. This antiviral flu drug is the first that inhibit virus replication. Trogarzo(TaiMed) is a first in class antiretroviral monoclonal antibody approved for the treatment of HIV-1 infection in patients who are multidrug resistant to available treatments. Trogarzo may be able to improve patients’ outcomes when other options have run out.

Women health is historically a neglected field and has been a highly underserved market. However the field has received more attention in recent years. This year, the first new pill, Orilissa (Abbvie) for treatment of moderate to severe pain associated with endometriosis was approved. Orilissa lowers the amounts of estrogens which are expected to decrease the moderate and severe symptoms of endometriosis. It was more than 10 years since the last treatment for endometriosis was approved and there is still a lack of treatment options for this potentially debilitating condition.


One of the highlights of the year was the approval of the first drug that acts by RNA interference (RNAi), Onpattro (Alnylam). The research that lead to the 2006 Nobel Prize in Physiology or Medicine on RNAi was published in 1998 (ii) and has now, 20 years later, successfully been translated into a novel therapy for treatment of a neurology disorder. The transfer of RNAi technology into drug development has been a scientific triumph with great potential to generate treatments for many more indications in the future. Onpattro treat nerve damage caused by hereditary transthyretin (hATTR) amyloidosis and was designated an ‘orphan medicine’.

Last year Tegsedi (Akcea and Ionis), also an antisense oligonucleotide therapy developed for the same disorder, similarly won approvals by FDA and EMA. And more will come, at least six other RNAi therapeutics are in phase III clinical trials for other indications (iii).


In 2018, small and medium sized enterprises (SMEs) contributed with 56% of the approved NTD. We commented on the trend that more and more SMEs are able to take their products through to regulatory approval by themselves last year, and 2018’s figures only strengthen this trend (iv). One driver for this development is the great expansion of the orphan field that provides opportunities to run much smaller late stage trials, thereby limiting the cost of development in a way that suits SMEs. The orphan market accelerated significantly during this year as compared to the previous year.

In 2018 the number of approved new drugs designated orphan status almost doubled in both EU and US, as compared to 2017. Twenty and 42 new orphan drugs were approved in EU and US, respectively, during 2018 (in 2017 12 in EU and 24 in US). Interestingly, nearly 70% of all approved orphan drugs were sponsored by SMEs. This marks great progress of options for patients living with rare diseases, and proves that the drug development companies and the agencies have continued to speed up promising drugs to markets even if the patient groups are limited.

Some of the outstanding contributions to significantly benefit patients living with rare diseases include Epidiolex (GW Research) which is approved by FDA for seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. Its attention is also due to that it is the first FDA-approved drug that contains a purified drug substance derived from Cannabis sativa plant (marijuana). Another interesting new therapy is Namuscla (Lupin).

In EU, Namuscla is the first approved treatment for symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders, a group of inherited muscle disorders where muscles are slow to relax after contraction. These disorders are chronic life-long debilitating conditions characterized by long lasting pain.

Crysvita (Ultragenyx) is an additional exciting new treatment approved in US for patients with X-linked hypophosphatemia (XLH), a rare, inherited form of rickets. Crysvita is the first and only therapy that addresses the underlying cause of X-linked hypophosphatemia.


The ideas of precision medicine are not new, but recent advances in science and technology have helped speed up the pace of this area of research, and major efforts are being invested in the fields.

A notable new oncology drug is Vitrakvi (Loxo and Bayer), a kinase inhibitor for solid tumors in various sites of the body. Vitrakvi became the second cancer therapy to be approved by FDA treating adult and pediatric patients whose cancers have a specific genetic feature, rather than a specific location of the tumor. This approval is a continuation of the new paradigm in the development of cancer drugs that are “tissue agnostic” set by Merck’s Keytruda in 2017.


It is obvious that the agencies are working hard to increase the patient access of important medicines where there is huge unmet medical need. In US as many as 53 NTDs were approved through fast track, breakthrough, accelerated approval or priority review approval. In EU only two expedited approvals of NTDs were granted by conditional approval last year.

The trend from 2017 remains during 2018 with more expedited approvals in US than in EU. This might be because of the eligibility to use the expedited pathways is much more limiting in the EU than in the US or that the alternatives in EU for expedited approvals are not as well established with the industry as they are in the US.


NDA had a strong presence in the EU regulatory arena and supported over 40% of the new products approved from 2013 to 2018.

To read the statistics of new drug product approvals from last year click here.


i. The data was gathered from the EMA and FDA official websites, as reported on the FDA and the EMA official websites on January 2019.
ii. Fire A., et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391:806-811.
iii. Mullard A. FDA approves landmark RNAi drug, Nature Reviews Drug Discovery 2018;17:613

Free Webinar – Be Heard: The Patient Voice at FDA Advisory Committee Meetings

In recent years, the global trend in drug development has included more active and influential patient activity. Patient and caregiver voices have become a key part of the regulatory process, and FDA Advisory Committee Meetings are no exception. At the Open Public Hearing (OPH) part of these meetings, patients have an opportunity to educate the committee and the Agency, impact regulatory decision-making and even help guide the future of drug development. This is particularly important for rare disorders. 

Join us on September 27th, 15:00 UK / 16:00 CEST / 10:00 EDT, for this engaging webinar where NDAs Lisa Peluso, Director of Coaching and Client Engagement, will cover:

• Quick guide to FDA Advisory Committees
• The Open Public Hearing and what it means
• Registering, Preparing, Delivering
• Tips on making sure your voice is heard

The webinar will be followed by a Q&A session for you to get direct feedback on key areas of uncertainty.

Click here to book your place today.

Lisa Peluso is Director of Coaching and Client Engagement at PharmApprove/NDA, where she helps development teams, doctors, patients and advocates to create and deliver clear, consistent, and convincing messages to regulators in the US and Europe. Lisa has coached hundreds of individuals and provided presentation/Q&A training and workshops to teams in pharma and biotech, and has helped over 45 clinical development teams prepare to present at FDA Advisory Committee meetings and EMA Oral Explanations. Her experience and familiarity with high-stakes regulatory meetings allows her to get to the heart of the challenge, and she excels at giving presenters and responders clear and actionable feedback, to instill confidence and help them communicate with clarity and impact. Prior to joining PharmApprove, Lisa worked at Kyowa Pharmaceuticals, where she supported both commercial and clinical teams and served as the principal liaison between the Tokyo, UK and US offices. Lisa spent 12 years in Japan, where she worked in communications in the legal, financial and hospitality industries.




Meet NDA at the DIA Annual Meeting in Boston






We will be attending this year’s DIA Annual Meeting, June 24-28, at the Boston Convention & Exhibition Center.

If you are planning to be there, we would really like to meet up with you!

Over the last 5 years, NDA Group has supported the approval of over 40% of new medicinal products in the EU and among our FDA advisory committee clients 80% have achieved product approval. We work across a range of disease areas including oncology, hematology, infectious disease, metabolic and immunologic disorders and CNS and have supported the development of over 50 rare disease programs.

Do you want your product to reach the world’s two largest markets with minimum delay? Arrange to meet us at DIA USA 2018 now.




Shelley Gandhi – Strategic Advisor, Pharmacovigilance & Drug Safety

Monday, June 25 | 15:00-16:15
#140: How Inspection Ready is your Organization?

Tuesday, June 26 | 16:15-17:30
#276: Patient Engagement in Pharmacovigilance


Dr Brian Edwards – Principal Consultant, Pharmacovigilance & Drug Safety

Wednesday, June 27 | 12:45-13:15
#351: Moving Forward with the EU Vigil: The Patient Contact in Pharmacovigilance


Dr Edwards will also be involved in the following DIA Community Round Table Discussions taking place at the DIA Community Zone, North East Lobby, BCEC.

Host Community: Clinical Safety & Pharmacovigilance

Tuesday, 26 June | 15:15-16:15
01 7027101 – Risk Management: New Direction

Wednesday, 27 June | 08:00-09:00
01 7027113 – Pharmacovigilance: No Longer Going it Alone

Wednesday, 27 June | 13:00-14:00
01 7027109 – Patient Engagement in Pharmacovigilance

Click here to download the Final Programme.


To book a meeting contact Anna Perrin:
We look forward to hearing back from you about a meeting and to seeing you at the event!


CAR T-Cells for Cancer Immunotherapy – Moving Target for Industry?

NDA’s Paula SalmikangasNiamh Kinsella and Paul Chamberlain have co-authored the article “Chimeric Antigen Receptor T-Cells (CAR T-Cells) for Cancer Immunotherapy – Moving Target for Industry?

Read the full article written by Paula Salmikangas, Director for Biopharmaceuticals and ATMPS, NDA Advisory Board, Niamh Kinsella, Principal Consultant, VP Early Stage Development and Paul Chamberlain Biopharmaceutical and Immunogenicity Expert which discusses the product-and patient-related variables that may pose challenges for the industry and developers both from the scientific and regulatory perspective.

This will be featured in Volume 35, Issue 8, August 2018 – in the Topical Collection of Pharmaceutical Research and is currently available online.

Pharmaceutical Research is an Official Journal of the American Association of Pharmaceutical Scientists, which presents papers that describe innovative research spanning the entire spectrum of drug discovery, development, evaluation, and regulatory approval. Small drug molecules, biotechnology products including genes, peptides, proteins and vaccines, and genetically engineered cells are an integral part of papers published here.

Meet us at BIO International Convention in Boston

We look forward to attending the 2018 BIO International Convention at the Boston Convention and Exhibition Center June 4-7th.

Over the last 5 years, NDA Group has supported the approval of over 40% of new medicinal products in the EU and among our FDA advisory committee clients 80% have achieved product approval.

We work across a range of disease areas including oncology, hematology, infectious disease, metabolic and immunologic disorders and CNS and have supported the development of over 50 rare disease programs.

The following individuals will be representing NDA Group as we participate in a week of networking and education with 16,000+ attendees from 74 countries. If you would like to arrange a face-to-face meeting with the NDA Group Team, please contact Barbara Clendenen, who will be happy to assist


Barbara Clendenen, Director, Business Development






Eva Lilienberg, Service Area Lead, Global Development






Dr Laurie Smaldone Alsup, COO and CSO







Tony Baker, VP Strategic Development


To read more about the event click here.


Professor Carole Longson to join NDA as Scientific Adviser

Carole Longson MBE, former Executive Director and Board member at the National Institute for Health and Care Excellence (NICE), has joined the NDA Advisory Board. Carole will act as an adviser within NDA on matters relating to drug approval, scientific advice and Health Technology Assessments, areas in which she has had great impact in her previous role.

Carole brings tremendous research, academic and pharmaceutical industry experience to the role, including seven years as a Research Lead at GlaxoSmithKline R&D.

She has extensive expertise of directing multidisciplinary life sciences research programmes, managing large scientific departments and collaborating on multinational research grants.

Carole was a past member of the Scientific Advisory Committee for Innovative Medicines Initiative (IMI), the leading pan-European public- private partnership which funds health research and innovation. She also holds non-Executive Director and advisory roles in other scientific organisations in the UK and abroad. These positions give her a unique and extensive perspective on the changing face of industry.

Carole has unparalleled insight into the fast evolving medicines pipeline having spent the past 18 years at NICE where she has pioneered and led the organisation’s technology appraisal programme. Carole is highly respected as a leader in HTA & Market Access, both in the UK and internationally and was formerly President of Health Technology Assessment International.

Throughout her career and role at NICE, Carole has gained valuable understanding of how the public and private sector must continue to collaborate to meet the shared aims of ensuring medical innovation benefits patients in the UK.

Johan Strömquist, CEO NDA Group, commented on the appointment:
“I am delighted to see Carole join us. Not only will she bring a wealth of knowledge and experience to NDA that fits intimately with our strategic direction, her reputation in the industry and among her former Agency peers, also stems from her positive and collaborative personality.”

Werner Van den Eynde, Vice President NDA Advisory Services, commented:
“This appointment is a major step in our continuing aim to broaden our reach and expand HTA and market aspects into our drug development service offerings. It is also a testament to NDAs reputation amongst regulators and HTA bodies – NDA remains the undisputed prime destination for the greatest contributors to our regulatory and assessment systems.”

NDA media contact:

About NDA
NDA is an independent consulting group with offices in Stockholm, London, Zürich, Munich, Paris, Princeton, Boston and San Francisco. Its mission is to ensure that good medicines reach patients without unnecessary delay. It accomplishes this by providing the pharmaceutical industry with a comprehensive range of professional drug development services focusing on regulatory affairs, health technology assessment, pharmacovigilance and quality assurance. NDA’s team of more than 150 full time professionals is comprised of more than 25% ex-regulators from major EU Agencies, and a specialist Advisory Board consisting of Europe’s leading regulatory and HTA experts. The NDA Advisory Board provides scientific advice to pharmaceutical companies supporting them in gaining fast and constructive assessment of applications from regulatory agencies and HTA bodies.

Dr. Thomas Lönngren to Present at the Chief Medical Officer Summit, May 7th

Dr. Thomas Lönngren, Strategic Advisor to NDA Group and former Executive Director of the European Medicines Agency (EMA), has been invited to present at the Chief Medical Officer Summit, Omni Parker House, Boston, MA on 7th May. 

Thomas will present along with Jeffrey Sherman, CMO, EVP R&D, Horizon Pharmaceuticals, in the session ‘Taking a Strategy Approach to Global Regulatory and Market Access Challenges’, were they will discuss:

  • Is a global regulatory and market access strategy the way to go?
  • Why isn’t EMA approval the same as market access in Europe?
  • What is the difference between EMA and FDA in regulating and approving medicines?
  • When and how to generate evidence that will satisfy regulators and payers
  • What is the best strategy to interact with regulators and payers?
  • Early access schemes in EU and how to qualify
  • How will trends like Real World Evidence influence regulatory decision making?
  • Brexit and its consequences for the EU regulatory system and how will it affect pharmaceutical companies

As well as presenting at the event, NDA Group is ‘Associate Sponsor’ of the CMO Summits, demonstrating its commitment and support towards the CMO community.

Please come by booth #2 and meet us;

  • Dr. Thomas Lönngren, Strategic Advisor to NDA Group and former Executive Director of the EMA
  • Laurie Smaldone-Alsup, MD, COO/CSO
  • Barbara Clendenen, Director, Business Development

The CMO conference is an annual gathering for physicians and scientists in biotech to exchange best practices in the advancement of drug development and business management. Although the program is primarily designed for CMOs of emerging biotech companies, CMOs of large pharma and other R&D decision-makers will also benefit.

To find out more about the event click here.

Meet NDA Group at PEGS Boston

NDA Group will be attending PEGS Boston, that takes place in Seaport World Trade Center, Boston April 30 – May 4, 2018

NDA supported over 40% of the new medicinal products that were approved in the EU during 2013 -2017. In 2016 NDA acquired PharmApprove, the market leader in managing critical FDA interactions to help us achieve the same results in the US.

If you are planning to attend this year’s PEGS, we would really like to meet up with you. We will have a few of our experts available at the event at booth 437:

  • Dr Niamh Kinsella, Biologics Expert
  • Dr Jenny Sims, Non Clinical Expert
  • Dr Josefin-Beate Holz, Clinical Strategy Expert

If you would like to arrange a meeting, please contact Niamh Kinsella, who will be happy to assist,


Three of our experts will present at the event:


Jenny Sims: Monday 30th April 12.20 pm, Immunogenicity Case Studies and Clinical Management Track.

Title: Early Development Strategy for Bacterial- or Viral-Vectored Gene Therapies: 2 Case Studies

Josefin-Beate Holz
Monday 30th April and Tuesday 1st May, Poster Session A
Developing an Integrated Summary of Immunogenicity (ISI) to Effectively Manage Regulatory Risks in Product Development

Niamh Kinsella
Wednesday 2nd May and Thursday 3rd May, Poster Session B
Maximising Value in Early Development



Click here to find out more about the event.

We look forward to hearing back from you about a meeting and to seeing you at the event!