How to Keep up With The Fast-Evolving World of ATMPs

Join us on Thursday 19th November 2020, 15:00 GMT | 16:00 CET | 10:00 EST, when Dr Paula Salmikangas and Professor Steffen Thirstrup discuss how to keep up with the Fast-Evolving World of ATMPs.

The development of novel advanced therapy medicinal products (ATMPs) is moving fast, as witnessed by the growing number of products appearing in clinical trials. Investments and ATMP developers are also increasing worldwide. Innovative technologies like gene editing have enhanced the design of products to better target the indications – for example, by allowing the move from autologous products to allogeneic, off-the-shelf products.

Advances in virus vector production systems have greatly improved the safety profiles of current gene therapy products. However, the design and engineering of ATMPs are becoming more and more complex, which brings new and possibly unknown risks and regulatory concerns. The availability of non-clinical models for safety testing is limited for these products and it may be that some risks are identified only through human exposure; this requires careful planning of the clinical studies and robust strategies for risk mitigation and long-term pharmacovigilance.

The challenges of persistency and immunogenicity for some products, such as adeno-associated viruses (AAVs), are currently hampering their long-term effectiveness and the overall clinical outcomes. Outstanding efficacy results for approved products like Kymriah®, Yescarta®, and Zolgensma® are strongly encouraging the field towards curative treatments, but the risks related to novel technologies should remain manageable and well-controlled to ensure real benefits for patients.

The key discussion points of this webinar will include:

  • An overview of the current status of approved ATMPs and those in clinical trials worldwide
  • Recognition of novel manufacturing technologies and their perceived risks
  • Understanding the pros and cons of non-clinical studies, including risk identification and mitigation strategies
  • An update on recent clinical results and new trial designs, including histology agnostic trials


Click to book your place today

If you require further information, please do not hesitate to contact us.


We are expecting this webinar to generate a lot of audience interest, so please send in your questions now to ensure the speakers have time to answer them in the Q&A: email our moderator on If your question is for a specific presenter, please include this in your email.


Dr Paula Salmikangas, Director of Biopharmaceuticals and ATMPs, NDA Group

Paula is the Director of Biopharmaceuticals and ATMPs and has been at NDA since 2017. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and the CMC aspects of biopharmaceuticals. As a former EU regulator, Paula was actively involved in establishing the regulatory guidelines for ATMPs and applies her unique experience to provide companies with technical & strategic advice for their drug development programs.


Professor Steffen Thirstrup, Director NDA Advisory Board, NDA Group

Steffen has been with NDA since 2013 and excels at advising companies on their development strategies to meet the expectations of regulatory agencies around the world. He applies his skills to a broad range of therapeutic areas and has successfully helped numerous clients interact with regulatory agencies throughout the stages of development and during regulatory review.


Moderated by Conor Kavanagh, Journalist and Editorial Assistant, Pharmafocus


Expedited Reviews: What you need to know to be Successful

A Case Study

By: Judith Plon, Principal Consultant, NDA Group

In the September issue of Pharmafocus, Frank Casty, MD, Senior Clinical Regulatory Advisor at NDA discussed “Expedited regulatory pathways and what you need to know to be successful.” This month I am following up with a case study of Bavencio (avelumab) injection, a Biological License Application (BLA) that was able to come to market quickly by utilising the benefit of multiple expedited review pathways.

Bavencio is a drug indicated to treat an aggressive neuroendocrine tumour of the skin for which there are no other treatments. With fewer than 20,000 cases per year in the US, the drug qualified for Orphan Status and, like other rare diseases, the primary review for the treatment effect was based on a small patient population, in this case less than 88 patients.1

As the sponsor had a well-planned regulatory strategy and utilised all the tools available to them during the drug development process, they were able to obtain both the Fast Track and Breakthrough Therapy Designation (BTD). Based on BTD, a preliminary advice meeting was held with the FDA to discuss an Accelerated Approval approach. The sponsor benefitted from several multi-disciplinary meetings with FDA, an important advantage of requesting and receiving this designation early in the programme.

The BLA also utilised a Rolling Review Process with the nonclinical sections of the application submitted ahead of the clinical data. Finally, the FDA granted the company a Priority Review status which means the application was reviewed in a six-month time frame. All of this was accomplished without the need for an FDA Advisory Committee Meeting!

This case study is an excellent example of how a well-planned and meticulously executed regulatory strategy can lead to a collaborative and rapid review, approval and, most importantly, availability of a new treatment for patients in an area with high unmet medical need.

At NDA, we support many companies in both the US and Europe seeking multiple expedited review procedures.

The full article was published in September 2020 in UK News – Pharmafocus.

Click to the full article

1. (Bavencio)

Optimizing Global Submissions in the Age of Rapidly Advancing Innovation and Rare Disease

Panel discussion moderated By: Laurie Smaldone MD

At the upcoming Chief Medical Officer Summit 360°, NDA’s CMO/CSO Laurie Smaldone MD will moderate the panel  “The Challenges of Global Development: Optimizing Global Submissions in the Age of Rapidly Advancing Innovation and Rare Disease.”

To kick off the discussion and  explore their thoughts on the subject the panel met (via telephone ) earlier in the year. In this summary we capture the main points raised during the call.

Panel members:

Laurie Smaldone, MD CMO/CSO, NDA Group, 

Matt Ros, Chief Strategy & Business Officer, Epizyme,  

Adrian Senderowicz, MD, SVP, CMO, Constellation Pharmaceuticals,  

Christine Swenson, SVP Regulatory Affairs, Beam Therapeutics, 

Haishan Xiong, PhD, MBA, SVP Business Development, Fountain Medical Development 

The panel discussion highlighted the major challenges in global drug development, in particular country specific differences around the definition of acceptable study endpoints, in healthcare philosophy and standard-of-care, and also the impact of small, but well advocated patient populations.

“Anyone who is in the development space should pay attention to the patient voice” [Matt Ros, Chief Strategy & Business Officer, Epizyme ]

Working in rare diseases comes down to understanding the insights from the patient’s experience as they are the true experts of their condition. If performed properly, the clinical development program becomes meaningful, does not create tremendous burden and will most likely help patients already in the process.

One of the biggest challenges in developing treatments for rare diseases globally is the definition of an acceptable endpoint and path to approval across countries. “Accelerated approval strategies in the United States are very common, whereas conditional approval strategies in Europe are unique and limited, suggesting differences in these regulatory pathways” [Matt Ros, Chief Strategy & Business Officer, Epizyme ]

To make the matter even more complex, global development can impact studies from their initiation. In China, the healthcare trial resources are currently highly concentrated in big cities, but this is opening up, making trial participation more accessible for the patients.

The trial set-up impacts both timing and recruitment and is challenged by the small patient populations and the gold standard of randomized controlled trials (RCT). “In rare diseases there are often situations where you are not going to be able to do that,” [Christine Swenson, SVP Regulatory Affairs, Beam Therapeutics]

Additional complications may include regulatory constraints in the sense that different regulatory bodies may offer different scientific advice or have alternative requirements. How physicians treat, patients may vary, as well as the standard-of-care in different countries. If you don’t plan with that in mind, timelines are much likely to extend, while you wait for approval for registration trials.

To read the full discussion click here

The panelists will continue their discussion at the  Chief Medical Officer Summit 360°, October 26-27 , make sure to be there for additional insights. For more information and full agenda click here.

Leveraging Expedited Regulatory Pathways to Optimize Drug Development

A Pharmafocus webinar

Join us on Thursday 24th September 2020, 15:00 BST | 16:00 CEST | 10:00 EDT, when Dr Frank Casty, Judith Plon and Professor Steffen Thirstrup will guide you through the expedited regulatory pathways available in the EU and US and discuss how to effectively leverage these to accelerate and optimize your drug development program.

A timely market introduction is a critical component of any drug development strategy – not only from a commercial standpoint, where an earlier market entry can lead to a competitive advantage, but also for patients awaiting better treatment options.

Regulators worldwide have developed specialized pathways to expedite review and approval of new drugs. Navigating these pathways can be a daunting task for sponsors.

Partnering with experienced regulators both in the US and EU is critical to achieving expedited reviews since getting the strategy right, developing effective documentation, and preparing for regulatory interactions can make the difference between a standard regulatory review and a successful expedited review

Our panel will discuss:

  • Which pathways are available
  • When to apply for specific designations
  • What data are required and what to expect if the designation is granted
  • Best practice to create a successful strategy

Click here to book your place today!

About the Panel

Dr Frank Casty: Senior Clinical Regulatory Advisor, NDA Group

Frank works with small and large companies across several therapeutic areas including cardiovascular, respiratory, inflammation, oncology and rare diseases.  He has led numerous FDA interactions, IND and NDA filings as well as presented at successful FDA Advisory Committee Meetings.

Before joining NDA, Frank worked for more than 25 years in small and large pharmaceutical companies leading Global Clinical Research and Medical Affairs organizations as well as serving as technical consultant for Business Development activities.

Judith Plon: Principal Consultant, NDA Group

Judy works with small and large companies across multiple therapeutic areas. She serves as a US Agent for clients leading the submission of their INDs, NDAs, and special designation requests (i.e. Orphan Drug Designations).

Prior to joining NDA Judy spent over 25 years in the Biopharma Industry, primarily working in the discipline of regulatory affairs. She held positions of increasing responsibilities and last served as a Vice President of Global Regulatory Affairs.

Professor Steffen Thirstrup: Director NDA Advisory Board, NDA Group

Steffen excels at advising companies on their development strategies to meet expectations of regulatory agencies around the world. He applies his skills to a broad range of therapeutic areas and have successfully helped numerous clients interact with regulatory agencies throughout the stages of development and during regulatory review.

Prior to joining NDA in 2013, Steffen was employed as the head of Division for Medicines Assessment and Clinical Trials at the Danish Health and Medicines Authority where he also acted as Danish member of the Committee for Human Medicinal Products (CHMP) at the EMA.



Expedited Reviews: What you need to know to be successful

By: Frank Casty, Senior Clinical Regulatory Advisor, NDA Group

In this great article published in the September issue of Pharmafocus, Frank Casty discusses “Expedited Reviews: What you need to know to be successful”

Speed to market is a critical component of any drug development strategy – not only from a commercial standpoint, but also for patients awaiting better treatment options. Throughout the drug development process there are many factors influencing speed to market but none more visible and critical than an expedited regulatory review.

The debate around how regulators can facilitate this process on both sides of the Atlantic has been top of mind for decades. During the early 1980s public outrage focused the FDA on faster reviews of HIV/AIDS treatments. In 2012, the FDA Safety and Innovation Act which introduced Breakthrough Therapy Designation, was followed by a final guidance in 2014 describing requirements for the four main expedited pathways, Fast Track(1), Breakthrough Therapy Designation (2), Accelerated Approval(3) and Priority Review(4).

In 2016, the Regenerative Medicines Advanced Therapy Designation, designed for expedited reviews of certain biologics, came about because of the 21st Century Cures Act. Recently, another expedited review process for antibacterial and antifungal agents, the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway) has been established.

Click to the full article

At NDA, we support many companies in both the US and Europe seeking multiple expedited review procedures. We will be discussing this in more detail in our upcoming webinar, where we will be ready to answer any questions you may have.


Potential Consequences of SARS-CoV-2 to ongoing clinical programs

The FDA Perspective

By: Laurie Smaldone, CMO/CSO, NDA Group 

In this white paper, NDA’s Laurie Smaldone discusses the potential impact of COVID-19 on on-going clinical trials.

Laurie Smaldone

The virus SARS-CoV-2 and the resulting COVID-19 disease have created devastating impacts around the globe on lives and livelihood, including major disruption of ongoing research and development activities for innovative therapies. From many perspectives the spread of SARS-CoV-2 has impacted and will continue to impact ongoing global registrational programs for a variety of disorders especially those not directly addressing the treatment or prevention of the SARS-CoV-2.

This disruption impacts all facets of the drug development process, from the Sponsor company, to the supply chain of investigational treatments to the productivity of study sites. The FDA has issued guidance to address the challenges that may arise from a range of disruptions that could impact the validity and integrity of clinical programs.

The FDA has noted that the spread of the virus may lead to GCP violations and protocol deviations, including impact on trial endpoints and safety collection.   FDA’s Center for Drug Evaluation and Research Director Janet Woodcock stated that “trials may be able to shift to tele-outcome assessments”, but others “may be damaged and may have to halt and not start up again until we can interact more freely”.

FDA’s recent guidance, “Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic”  addresses the potential impacts to ongoing trials.  The guidance provides stakeholders in the drug development and approval process with critical considerations for ongoing trials.

Potential COVID-19 impacts

Trials may be impacted in a variety of ways. Some sites have had to close down or have witnessed significant recruitment delays due to the inability of patient travel, illness, or redeployment of study personnel to address COVID-19 patients or trials.  These limitations can greatly impact study continuity and procedures to maintain study integrity.  In making decisions on trial continuity, Sponsors will need to do a robust   assessment of  trial conduct that may impact patient safety, GCP, drug storage and administration, and protocol procedures .


Click to read the full white paper

Mandatory use of eCTD in 2019 – Are you ready?

Mandatory use of eCTD for all submission types and procedures by 1 January 2019
From January 2019 all submissions in CP, DCP, MRP and NP, related to authorisation and regulatory maintenance (variations) of human medicinal products, must be submitted in eCTD format.

Baseline dossier
Submission of a baseline CTD Module 3 serving as a start of an eCTD is highly recommended by the agencies, as the baseline dossier facilitates the review and assessment of future applications. A baseline submission is a compiled submission of the current status of the dossier, i.e. resubmission of currently valid documents that have already been provided to an agency but in another format. The sections provided to make up a baseline can be defined by the applicant, but any omissions should not render the submitted content to be misleading. A baseline would typically consist of the Module 3 documents that tend to change over time during the lifecycle of the product.

NDA Service
NDA´s well experienced team may assist you with publishing of variations in eCTD format as well as with conversion of your current documentation to eCTD format.
For more information about eCTD publishing and NDA´s other services, please contact



Shelley Gandhi Chairs the 2nd Annual Biosimilars Europe Congress

Shelley Gandhi, Strategic Advisor Pharmacovigilance & Drug Safety NDA Group, has been invited to Chair the 2nd Annual Biosimilars Europe Congress Nov 23 – 24, 2017 at Hilton London Olympia, London.

The conference will bring together industry experts to explore the strategies to gain insight into new biosimilar development strategies, different characterization and analysis methods, clinical advancement and successful case studies.

This conference offers a rare opportunity to its participants to understand and learn from top experts in the biosimilars field and to share experiences. The conference will also provide a platform to discuss the current vital issues, regulatory issues, market assessment and commercialization and globalization.

Key themes:

  • Current status of Biosimliars market
  • Monoclonal antibody Biosimilars
  • Challenges faced when moving towards globalization
  • Opportunities in emerging markets
  • IP issues, naming and labelling issues related to biosimilars
  • Partnership, new investment and business models
  • Challenges and opportunities for biobetters and monoclonal antibody biosimilars
  • Clinical trials strategies and new guidelines for biosimilar clinical trials
  • Benefits and concerns of interchangeable and biosubstitutes and post authorization monitoring
  • Bringing biosimilars closer to patients and healthcare professionals
  • New solutions to demonstrate similarity and different characterization methods

To find out more about the event click here.







Shelley Gandhi, Strategic Advisor Pharmacovigilance & Drug Safety NDA Group

How do we value drugs? Reforms for better decisions and use.

Within the framework of SNS, Centre for Business and Policy Studies, and the research project Value of new drugs, a number of researchers at the SNS initiative examined a number of issues of great importance for health care. The final report presents overall conclusions and policy suggestions for how we can improve today’s decisions on the use of drugs, both for the drugs that are available today and the new drugs coming on the market.

Authors are NDA’s HTA Advisory Board Chairman Bengt Jönsson and Katarina Steen Carlsson. The report was presented at a conference in Stockholm, Sweden, 22 November 2013, and was discussed by leading decision makers in the Swedish health-care and pharmaceutical policy, for example Christina Åkerman Director General of the MPA and Anders Blanck CEO LIF, the Swedish life science industry organisation. NDA’s Thomas Lönngren, who also contributed to the report, participated in a couple of the panel discussions as well.


Professor Bengt Jönsson






Professor Bengt Jönsson
Chairman, HTA

Professor in Health Economics at Stockholm School of Economics. Founding director of the Centre for Health Technology Assessment (CMT) at Linköping University. Member of the Editorial Board of the European Journal of Health Economics and the International Journal of Health Technology Assessment in Health Care.  Former President of the international Health Economics Association. Advisor and consultant to WHO, OECD and the World Bank.

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.

Dr. Markku Toivonen talked at EURORDIS Summer School on Medical Research and Clinical Trial Methodology

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: