Why should you engage early with regulators and HTA bodies?

By: Dr Mira Pavlovic-Ganascia & Claes Buxfeldt, NDA Group

In this white paper NDA’s Dr Mira Pavlovic-Ganascia and Claes Buxfeldt provide insight into the reasons to seek scientific advice and elaborate on what the expected outcomes of constructively engaging with regulators and HTA bodies could be.


Dr Mira Pavlovic-Ganascia
Claes Buxfeldt

In Europe there are many routes to gain scientific advice directly from official authorities. National regulatory advice is a routine practice of most regulatory agencies, as well as of the European Medicines Agency (EMA).

Scientific advice in the HTA arena is more recent. In this context, national HTA advice is provided against the backdrop of country or region-specific policies and legal requirements. Early dialogue involving multiple HTA bodies is also rather recent, provisioned in the context of the European Network of Health Technology Assessment (EUnetHTA) Joint Action 2 as well as specific actions financed by the European Commission (SEED)(ref) and involving both EMA and EUnetHTA. Indeed, in recent years, EMA and EUnetHTA have led several combined regulatory-HTA pilots to get experience both on the process and the content of such an exercise (1,2,3).

Since the creation of our NDA Joint Advice service offering in 2011, the NDA Advisory Board has also conducted numerous such projects for clients, focusing on ensuring a thorough understanding of the varying regulatory and HTA requirements and bridging the relative efficacy – relative effectiveness gap.

The means to gain scientific advice are many and diverse, but so are the reasons companies strive to get it.

Why seek scientific advice?

The combined scientific advice/early dialogue processes allow a company to engage relatively early in discussions with regulators and HTA bodies. The label on the tin indicates that the main interest of such an engagement would be to gain scientific input into the development program of a product to be able to steer it away from regulator, HTA and payer pitfalls and towards rapid patient access.

The scientific input usually covers the proposed study design(s), comparators, endpoints, target study population and inclusion criteria’s, study length and other key aspects important both for regulatory and HTA bodies. More in general, this early discussion will allow a company to check if the evidence to be generated for EMA is relevant for HTA submissions.

Asking for a combined regulatory and HTA advice is even more important in cases where treatment guidelines are weak or inexistent and/or there is no relevant HTA decision available in the field of interest.

Reality, however, is even more diverse than this and the reasons and rationales for companies to go for scientific advice therefore vary greatly.

1. Stake holder engagement

Engaging early with regulators and HTA bodies can be a crucial motivation for engaging in the scientific advice process, as it provides a unique opportunity to introduce these two key stake holders to the science and circumstances behind the company’s product. This can benefit the process in two ways:

  1. In any future engagements the regulators and HTA bodies will know the product/technology and targeted patient population better. This will potentially enhance future interactions and remove communication hurdles.
  2. By engaging with the right individuals in the right agencies, interest in the product can be sparked which can lead to constructive and positive input along the product’s development path. Building rapport with assessors are again an important vehicle to friction less communication.

 

2. Demonstrating progress

Engaging with external experts in a structured manner is also an excellent way to demonstrate to boards and stake holders that the product is progressing through the development process. Constructive feedback from regulators and HTA bodies can help steer the product development but can also be a value driver for the company.

If this is a main driver for expert engagement or seeking scientific advice, other reasons should also be carefully considered to optimize the value of the interaction. Seeking endorsement for the sake of it is rarely the optimal use of the time spent by external experts or regulatory agencies and HTA bodies.

 

3. Compliance

Many multi-product pipeline companies have highly controlled processes for how to progress products through the development process. This may well include the consideration and execution of a formal scientific advice procedure.

As this reason is not a value driver in itself, the reasons that the company put this requirement in place in the first instance should be carefully considered when running through the motions. Teams following a check list risk missing the underlying strategic reasons for why the process is necessary and may therefore not consider alternatives or options that might fit better or add more value.

 

4. Internal alignment

Although not its primary purpose, internal alignment is an incredibly valuable potential outcome of any scientific advice/early dialogue process. Teams working towards a clear goal along a clear timeline tend to glue together and more easily visualize the ultimate outcome. At NDA Group we’ve seen many occasions where the formal process has created strong composite teams. These teams are primed to progress the product through development with a determination and shared purpose that would not have been possible without the structure and clear goal that the scientific advice process offers.

Achieving internal alignment should therefore always be considered a potential beneficial outcome and should be planned for accordingly.

 

Read the full white paper

Don’t miss the opportunity to hear to Dr Mira Pavlovic-Ganascia and Claes Buxfeldt speak about HTA and Europe at our complimentary seminar:  Optimizing Value – Regulatory and Market Access Considerations in Stockholm on the 21st May.

Read more and register
 

The state of the Swedish orphan pipeline

In a short piece on the rare and orphan disease pipeline in Sweden, Business Sweden, together with SwedenBIO, have outlined the current state of Swedish orphan development. The report describes a vibrant drug development landscape that is reflective of the global move towards orphan development and the strong emphasis on oncology.

Over the last 19 years Swedish companies have been granted 60 orphan designations in the EU and 47 in the US. Unsurprisingly, oncology and neurology are the leading therapeutic areas representing 38% and 11% of the Swedish orphan pipeline respectively.

In total, Swedish companies are developing orphan drugs in 16 different therapeutic areas and it is interesting to note that transplantation comes in as the third largest area of interest in terms of number of compounds.

If the number of products in the pipeline and the activity of the life science networks and communities are any measures of future success – the Swedes and Danes are in for a very exciting ride!

In addition to the findings outlined in the report, we have looked at the geographic origin of the companies contributing to this space and two areas clearly dominate this development; Stockholm/Uppsala and Medicon Valley in the Malmö/Lund/Copenhagen region.

If we were to include Danish companies in the Medicon Valley area, we are sure the picture would change, but the two regions are undeniable hot-spots when it comes to pursuing orphan targets in the Nordics.

The Swedish biotech stage is thriving and expanding – at last count the industry organisation, SwedenBIO, had 265 member companies and it keeps growing. Medicon Valley Alliance is a network of life science organisations, from academia and local health care bodies, to life science companies and service providers, that span the Copenhagen/Malmö/Lund region. They have also grown at a steady pace and comprises over 250 organisations today.

If the number of products in the pipeline and the activity of the life science networks and communities are any measures of future success – the Swedes and Danes are in for a very exciting ride!

You can read the original report at over at Business Sweden’s website.

Optimizing Value – Regulatory and Market Access Considerations

Welcome to NDA’s seminar on 21st May 08:30-13:00 on Optimizing Value – Regulatory and Market Access Considerations.

NDA would like to invite you to join Professor Steffen Thirstrup and Professor Mira Pavlovich-Ganascia share their experiences and provide their insights and considerations to optimize the value of your development program.

The market access environment is getting increasingly challenging. The ability to develop plans and strategies for access, for today as well as tomorrow, is critical to bring new medicines to patients.


Agenda:
08:30 Registration and light breakfast
09:00 Welcome – Claes Buxfeldt, Director NDA HTA Advisory Board
09:30 Session one: Challenges on development programs for new drugs – Professor Steffen Thirstrup
10:00 Coffee and mingle
10:30 Session 2: HTA and Europe – where are we heading? – Professor Mira Pavlovich – Ganascia
11:00 Question and answer session
11:30 Concluding remarks and take home messages

12:00 – 13:00 Ask the Experts – Book a 20 min slot to speak directly with our experts, email: frukostseminarium@ndareg.com


About the speakers

Professor Steffen Thirstrup,
Director NDA Advisory Board, Former Head of Division, Medicines Assessment and Clinical Trials, Danish Health and Medicines Authority, and CHMP member. Steffen is an expert in clinical development and regulatory strategies.

 

Professor Mira Pavlovich-Ganascia,
NDA HTA Advisory Board member, practicing physician and former Deputy Director for Health Technology Assessment at the Hauté Autorité de Santé (HAS), France. Mira is an expert in HTA activities related to early dialogues with developers, disease-specific guidelines and methodology of assessment for reimbursement purposes.

Claes Buxfeldt,
HTA Director at NDA Group, former Global Price & Reimbursement Director in Respiratory & Inflammation at AstraZeneca. Claes has 20 years’ experience in the market access and health economic areas and has extensive experience in developing the market access strategy/payer strategy/payer evidence generation for drug development programs from pre-                                            clinical to launch phase.


Learning aspects

  • How to secure value in your development program considering both regulatory and market access requirements
  • Learn about how, when and why it is critical to consider HTA and market access requirements in your development program
  • Understand how to mitigate differences in demands/requirements between regulatory and HTA bodies

When: Tuesday 21st May 2019

Time: 08:30 – 13:00 (opportunity to book 1-1 meetings from 12:00)

Venue: SciLifelabs, Tomtebodavägen 23a, 171 65 Solna, Sweden

The seminar will be an open and interactive workshop with the opportunity to ask our presenters questions.

Specific questions can also be sent in advance to frukostseminarium@ndareg.com. Indicate if you would like to discuss them openly during the meeting; otherwise we can book separate meetings to discuss them after the seminar.

Registration: RSVP by Friday 17th May 2019 to frukostseminarium@ndareg.com

Contact: Denise Strömquist, Marketing and Management Coordinator, +46 (0)8 590 778 00, or email frukostseminarium@ndareg.com

The seminar is free however if you are unable to attend, please advise us no later than two days before the seminar.

 

We look forward to seeing you there!

 

 

4 ways that HTA will change under the new European regulation

By: Johan Strömquist, CEO, NDA Group

In this white paper NDA’s CEO, Johan Strömquist, discusses four ways that Health Technology Assessments will change under the new proposed European regulation.


Johan Strömquist, CEO

Over many years the European Network for Health Technology Assessment (EUnetHTA) has been working to HTA bodies into the same room to harmonise and develop thinking around the assessment of medicines from a societal point of view. The approaches to HTA assessment across Europe vary significantly across member states causing confusion, challenges and increased costs for drug development so the work of this voluntary network has been greatly appreciated by industry, payers and politicians alike.

However, the network’s voluntary nature and a lack of a formalized framework for the continuation of the activities have caused issues to progress and harmonization throughout its existence. To stabilise the situation and institutionalise the continued efforts, the European Commission (EC) has therefore presented a new HTA regulation back in January 2018 that will be up for decision in the European Parliament in spring 2019.

The regulation does not put any restrictions on how health economic decisions should made or how pricing of products should be carried out

Should this new regulation be endorsed as it is it will have important impact on drug developing companies and the way that drugs will and should be developed if drug developers want to optimise their path to market.

The regulation addresses four things that every biotech CEO and industry leader should be aware of.

1.   Joint Clinical Assessments

Years of experience and significant respect for the subsidiarity principle enshrined in the Maastricht Treaty has enabled a pragmatic approach in the draft regulation. The regulation does not put any restrictions on how health economic decisions should made or how pricing of products should be carried out. Instead it focuses on the area of commonality that has been agreed and pushed with EUnetHTA (with varying support from different member states). This area is clinical assessment or the assessment of relative effectiveness of the product.

Should the regulation pass as it is this means that all novel products (defined as any medicinal product passing through the centralised procedure or any medical device or IVD that receives an opinion under the new Medical Device Regulation) will be assessed under a new centralised clinical assessment scheme. This would in theory replace national clinical assessments and could straighten a product’s road to market.

Once a product has been assessed for its clinical benefits, national authorities would still have to assess its value in the local market but one crucial step in this process would have been eliminated and the outcome would be harmonised across the EU.

At NDA we will be ready to support clients managing this process when / if the regulation kicks in. Through our extensive experience in the market access area and by support of collaboration partners   we are ideally placed to work with companies pulling their HTA dossier together.

 

2. Joint Scientific Consultations

The practice of scientific consultations has become increasingly well-established yet is a relatively under-utilised mechanism for companies to improve their understanding of the different stake holder requirements. One reason why this is the case is because the process is time consuming. Another is because the procedure is under resourced and to a great extent based on voluntary contributions from the member agencies.

Under the new regulation all scientific consultations would be managed centrally, including the parallel consultations with the EMA.

This is most likely going to have relatively little impact on the use and performance of consultations to begin with, but by establishing a long-term platform for these activities there will be greater room to expand resources, improve the process and harmonise the contributions from the member states.

NDA’s Joint Advice services addresses several of the weaknesses of parallel consultations. By relying on the experts that built the European regulatory systems we deliver high quality, high speed advice. This is used by many companies as a proxy for formal consultation when time is tight, or as a way to prepare for the formal process to maximise the value that can be attained from engaging early with HTA bodies.

 

3. Identification of emerging health technology

Horizon scanning as it’s popularly called is a resource intensive activity when it is performed at the member state level. Consolidation of this to a central European function will allow more efficient use of resources, but it will also result in an overall higher quality of the output and material that will be used for training and intelligence at the member state level.

In the long run biotechs and pharma companies can expect their HTA counterparts to be more up to speed with emerging health technologies and that the playing field will become gradually more level across countries thanks to this.

 

4. Voluntary Cooperation

A clause that is easy to dismiss in the new regulation is where it speaks of voluntary cooperation. However, this means that the current cooperation, which has been moving slowly but has left important marks in the way assessments are carried out, has a formal home. This is important as it gives legitimacy and encourages the continuation of EUnetHTA’s activities in a new and more official format.

Expect this to lead to increased cooperation between HTA bodies in Europe and an increased exchange of scientific and methodological ideas across the member states.

NDA continually monitors the regulatory development to cover any relevant regulatory changes. As many changes are not publicised through regulation, but are a matter of practice in the agencies, our exposure to on-going procedures and 25% of our staff having experience working at a regulatory body are essential in staying on top of the change. Over the last five years NDA has been involved in more than 40% of the new medicinal products approved in the EU.

 

Will it lead to harmonisation?

There are still big differences in the legal traditions, pricing and reimbursement systems and the socio-economic circumstances across the member states of the EU. We will not see these things change overnight, hence it will take substantial time for these collaborative efforts to bear fruits across the range of the spectrum. Since health care systems and financing is a country matter, appraisals of value and decisions if a new medicine should be granted access is still a country matter. This includes all decisions around pricing as well.

We have however already seen how collaboration, exchange of ideas and development of joint therapeutic area specific methodologies are spreading. This does lead to harmonisation in a few crucial areas around clinical assessment and this is not just progress, but also important for drug developers across the world, as it has the potential to make HTA more transparent and therefore predictable.

The new HTA regulation builds on these important steps to increase the transparency and predictability even further. Despite the challenges that remain, this is progress.

As a partner NDA is ideally placed to support drug development companies navigate through the changes that this new regulation entails. By tracking and reflecting the current thinking and practices of the agencies and by providing tailor made, actionable advice and the help to operationalise this we are looking forward to seeing how this new regulation can help increase predictability and improve the speed with which important therapies reach patients in need, all across the EU.

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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

 

 

 

Webinar – Is an Early Development Strategy Really Needed?

Due to the high costs for product development, it is difficult for small and medium-sized pharmaceutical companies to take their product to the market on their own. By integrating project-specific development strategies and deliverables in the overall product development objectives can help develop a strategic roadmap to add value during product development which in turn can optimise exit strategies.

Join us on 16th May at 15:00 BST, 16:00 CEST, 10:00 EDT for this engaging webinar where Dr Niamh Kinsella, Biologics Expert, VP, Early Stage Development, NDA Group will cover the following strategies:

  • See how to implement a stage-gate approach for the early development of your product
  • See how a global regulatory strategy can be implemented
  • Determine your strategy for agency interactions.

This webinar will enable you to reflect on how well your own plans are progressing and what more needs to be done.

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


Click here to book your place today!


Dr Niamh Kinsella, Biologics Expert, VP, Early Stage Development, NDA Group.
Niamh has worked in regulatory affairs for over 15 years and has 5 years’ experience in biopharma industry prior to entering regulatory affairs. Niamh joined NDA in January 2010 and specialises in CMC / quality aspects of biological products, including recombinant proteins, monoclonal antibodies, vaccines and advanced therapy medicinal products. Niamh has strategic experience in CMC/quality and regulatory development throughout the product lifecycle and also has experience in regulatory operations in the preparation of documentation for regulatory submissions (Agency meetings, IMDS/INDs and MAAs/BLAs).

 

 

Observations of a market access expert

An insightful interview with NDA’s Claes Buxfeldt, HTA Director, entitled “Observations of a market access expert” featured in Aprils edition of Pharmafocus.

Reimbursement is the fourth hurdle of pharmaceutical development. Payers and Health Technology Assessment (HTA) bodies demand compelling evidence of how new interventions improve the standard of care and public health.

In the article Claes reflects on his experiences, challenges & opportunities and highlights the key trends and drivers for changes that will affect the Life Science industry in the HTA and market access space.

 

 

 

 

Interactions with Agencies During Drug Development

Welcome to NDA’s free Lunch seminar on Tuesday 30th April 12:00- 14:00 on Interactions with Agencies During Drug Development

There are many opportunities for bringing your message across to regulators, by interacting with the right EU Agencies at the right time, depending on the type of product, applicant, procedure and stage of development. Find out whether you’re making the most of all these opportunities to facilitate your drug development program.


About the speakers

Professor Steffen Thirstrup

Director NDA Advisory Board, Former Head of Division, Medicines Assessment and Clinical Trials, Danish Health and Medicines Authority, and CHMP member. Steffen is an expert in clinical development and regulatory strategies.

 

Dr Rosalind Cox
Principal Consultant NDA UK, formerly Divisional VP with Abbot. Roz specialises in European Regulatory Strategy and Global Development.

 

 


LEARNING ASPECTS:

  • Get an overview of the opportunities for interacting with National Regulatory Agencies and EMA throughout development and how to optimise your interactions
  • Learn about procedures and product type specific interactions with special consideration for SMEs
  • “By failing to prepare, you are preparing to fail”Benjamin Franklin An oral explanation is your ultimate chance to engage with EU regulators in getting your product approved

When: Tuesday 30th April 2019

Time: 12:00 – 14:00 (opportunity to book 1-1 meeting afterwards)

Venue: Queen Edith’s Room, The Cambridge Building, Babraham Research Campus, Cambridge, UK

The lunch seminar will be an open and interactive workshop with the opportunity to ask our presenters questions.

Specific questions can also be sent in advance to ndaseminar@ndareg.com. Indicate if you would like to discuss them openly during the meeting; otherwise we can book separate meetings to discuss them after the seminar.

Registration: RSVP by Friday 26th April 2019 to ndaseminar@ndareg.com.

Contact: Anna Perrin, Marketing Assistant, +44 (0) 1372  860 623, or email ndaseminar@ndareg.com.

The lunch seminar is free. If you are unable to attend, please advise us no later than two days before the seminar.

 

We look forward to seeing you there!

 

 

 

Meet the NDA experts in March

March is a busy month for our NDA experts and there are plenty of opportunities to meet them and discuss.

This month we are participating in several events across the globe. If you would like to book a time to speak with one of our experts, contact us!

Brian Edwards
Principal Consultant, Pharmacovigilance & Drug Safety is speaking at: 

GCC Pharma Regulatory Summit

Where: Oberoi Hotel, Dubai, UAE

When: 13th – 14th March

Session: Pharmacovigilance Training

Lisa Peluso
Director, Coaching and Client Engagement is speaking at: 

DIA Medical Affairs and Scientific Communications Forum

Where: Loews Sapphire Falls Resort, Orlando, Florida, USA

When: 18th – 20th March

Steffen Thirstrup
Director, NDA Advisory Board is speaking at: 

3rd Annual Nordic Precision Medicine Forum

Where: Hotel Birger Jarl, Stockholm, Sweden

When: 18th – 19th March

Session: Examining the Influence of Current Regulatory Frameworks in Facilitating the Progress & Implementation of Precision Medicine

Steffen Thirstrup
Director, NDA Advisory Board is also key speaker at NDA’s breakfast seminar: 

Interacting with the Agencies during drug development

Where: SciLifeLab, Tomtebodavägen 23A, Stockholm, Sweden

When: 20th March

Paula Salmikangas
Director of Biopharmaceuticals & ATMPs, NDA Advisory Board is speaking at: 

EBMT – 45th Annual Meeting of the European Society for Blood and Marrow Transplantation

Where: Messe Frankfurt, Frankfurt, Germany

When: 25th – 27th March

Session: Regulatory issues of genetically modified cells

Thomas Lönngren, Andrea Aschenbrenner & Monika Eck-Schaupp
are attending: 

Bio Europe Spring

Where: Messe Wien Exhibition and Congress Center, Vienna, Austria

When: 25th – 27th March

Paul Chamberlain
NDA Advisory Board is speaking at: 

17th Biosimilar Conference, Medicines for Europe

Where: Hotel Okura, Amsterdam, The Netherlands

When: 28th – 29th March

Session: Biosimilarity demonstration & regulatory fitness-for- purpose towards a tailored approach to comparative clinical trials

 

If you are interested in booking one of our experts to speak at your event, contact us.


 

Interactions with Agencies During Drug Development

Welcome to NDA’s free Breakfast seminar on Wednesday 20th March 08:30- 10:00 on Interactions with Agencies During Drug Development

There are many opportunities for bringing your message across to regulators, by interacting with the right EU Agencies at the right time, dependent on the type of product, applicant, procedure and stage of development. Find out whether you’re making the most of all these opportunities to facilitate your drug development program.

Join us to hear Steffen Thirstrup, Director NDA Advisory Board member, formerly Division Head at the Danish Medicines Agency and CHMP member share his experiences and provide his insights into making the most of the opportunities to interact with EU Agencies during the drug development process.


LEARNING ASPECTS:

  • Get an overview of the opportunities for interacting with National Regulatory Agencies and EMA throughout development and how to optimise your interactions
  • Learn about procedures and product type specific interactions with special consideration for SMEs
  • “By failing to prepare, you are preparing to fail”Benjamin Franklin An oral explanation is your ultimate chance to engage with EU regulators in getting your product approved

When: Wednesday 20th March 2019

Time: 8:00 – 8:30 Breakfast, 8:30 – 10:00 Presentation, 10:00 – 12:00 meet with NDA experts

Venue: SciLifeLab (Air & Fire at ground floor) Tomtebodavägen 23A, Solna, Sweden

The breakfast seminar will be an open and interactive workshop with the opportunity to ask questions to Steffen. Specific questions can also be sent in advance to frukostseminarium@ndareg.com.

To book a meeting with our experts after the seminar please write 1-1 meeting and specify the topic and participants from your company in the registration email. You will recieve a confirmation email with the time slot for your meeting.

Registration: RSVP by Friday 15th March 2019 to frukostseminarium@ndareg.com

Contact: Denise Strömquist, Client relations Nordics, +46 (0)8 590 778 00, or email frukostseminarium@ndareg.com

The breakfast seminar is free. If you are unable to attend, please advise us no later than two days before the seminar.

 

We look forward to seeing you there!

 

 

 

Passionate about solutions for patients with rare diseases

 

1 in 20 people will live with rare disease at some time in their life*.

Johan Strömquist CEO NDA says: “There are more than 6 000 identified, untreated rare diseases in the world. A lot has been done in recent years to address this by regulators and innovators but for many it is still not enough.”

At NDA much of the work we do go into supporting companies working to address this. We care passionately about this work and about getting treatments to the patients affected. Rare Disease Day is our opportunity to show this to the world and to promote the vital work that is going on in this space by so many passionate patients, physicians and scientists.


What is Rare Disease Day?

Rare Disease Day was first launched by Eurordis and its Council of National Alliances in 2008 with the goal to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives. 1 in 20 people will live with rare disease at some time in their life but despite this there is no cure for the majority of rare diseases and many go undiagnosed.

For 20 years the NDA Group has supported pharmaceutical and biotech companies around the world with strategic regulatory guidance across a range of diseases. Increasingly more companies have been trying to solve the puzzles for a diversity of untreated rare diseases. NDA has been there every step of the way.

Laurie Smaldone Alsup, MD, CSO and CMO of NDA says: “I believe we are still at the beginning of addressing rare diseases. With new scientific and technical advances every day we will be turning a corner where many more novel treatments will be discovered to address a range of difficult to treat disorders.”

She continues: “For me Rare disease day is a call to arms to create awareness of the continued need to address serious rare disorders to improve and extend patients’ lives.”

The theme of 2019 – Bridging health and social care

The theme for this 2019 Rare Disease Day is ‘Bridging health and social care’ For most people living with a rare disease, as well as their family members or carers, the reality of daily life can include any combination of the following: collecting and taking medicines, attending appointments, participating in physical therapy, using specialist equipment and accessing various social and community support services and respite care. Managing these care-related tasks alongside their usual daily activities such as work, school and leisure time can be challenging.

In support of patients with rare disease

2018 Rare Disease Day theme, ‘Patient Involvement in Research’, helped to underpin the importance of including patients in the drug development process.

Dr. Markku Toivonen, MD, Scientific Director at NDA, former CHMP member and Chairman of EMA’s Scientific Advice Working Party comments:
“The development of patient advocacy and the involvement of patients in the development and regulatory process has truly been game changing. At NDA we take great pride in helping to empower patient advocates through training in regulatory science and medical research with Eurordis, the European Organisation for Rare Diseases. ”

Markku continues: “Personally, it has been a privilege to participate in Eurordis Summer School for patient experts, advocates and researchers annually over the past 10 years as one of the trainers. Based on the candid feedback Eurordis has received over the years, the enthusiastic and highly motivated participants with varied backgrounds have benefited from the interactions with peers. They have returned home with new tools to help them in their endeavors and to navigate in the maze of science, medicines development, licensing and real-world challenges. It has been a pleasure to meet many of them again over the years, and to see how they have contributed at various levels from new medicinal product development to regulatory authority activities and overcoming the ultimate hurdle: access to treatment.”

* www.rarediseaseday.org

 

 

Immunogenicity for investigational biopharmaceutical products – Start with the goal in mind

Welcome to NDA’s free Breakfast seminar on Thursday 14th March 09:00- 10:30 on Immunogenicity for investigational biopharmaceutical products – Start with the goal in mind

All biopharmaceutical products are associated with an intrinsic potential to induce immune responses in treated subjects. Regulatory agencies expect sponsors to evaluate and mitigate these risks during product development, applying a strategy that addresses product- and patient-related factors. Overall, understanding and controlling immunogenicity-related risks are attainable objectives, and approvability should not be compromised if these risks are suitably evaluated from the earliest stages of development.

Join us to hear Paul Chamberlain, Biopharmaceuticals & Immunogenicity Expert and NDA Advisory Board member, explain how to anticipate and avoid issues for biopharmaceutical development. Grab this opportunity to benefit from his experiences and insights.


LEARNING ASPECTS:

  • How to apply immunogenicity risk assessment at the lead candidate selection stage
  • Minimizing incremental risks associated with manufacturing
  • Understanding what and how to evaluate, at different stages of clinical development
  • Integrated presentation of data to regulatory agencies

When: Thursday 14th March 2019, breakfast from 09:00, presentations start at 09:30

Where: Am Klopferspitz 19a, 82152 Martinsried/Planegg, BioM Seminarraum, Munich, Germany

The Breakfast seminar will be an open and interactive workshop with the opportunity to ask questions to Paul. Questions can also be sent in advance to ndaseminar@ndareg.com.  Please specify if you would like to ask them openly during the seminar or prefer to discuss them privately afterwards.

Registration: By Tuesday 12th March 2019 to: ndaseminar@ndareg.com

Contact: Nathalie Brodale-Breinbauer, +49 (0) 89 3585 4036 or email nathalie.brodale-breinbauer@ndareg.com

 

We look forward to seeing you there!

 

 

 

Effective presentation of immunogenicity related data in regulatory dossiers

NDAs Paul Chamberlain, Biopharmaceuticals & Immunogenicity Expert and NDA Advisory Board member, provides practical advice about how to present immunogenicity-related information in regulatory dossiers, with a particular focus on a model for an Integrated Summary of Immunogenicity to be submitted in the marketing authorization application for novel biopharmaceutical products in ICH regions (EU, USA and Japan) in the latest publication of Bioanalysis.

A format that links the analysis of potential risk factors to a justification of the methodology applied for risk evaluation and conclusions for riskmitigation is presented as a model that can be adapted according to the weight of evidence to be submitted in support of the assessment of impact on overall clinical benefit versus risk for the particular situation.

 

 

 

If you are interested in hearing more from Paul he will be presenting at the following events in March:

DDF Summit on; Biosimilars – Regulatory Primer for Formulation Scientists 12th March in Berlin. To learn more click here.

NDAs breakfast seminar; Immunogenicity for investigational biopharmaceutical products – Start with the goal in mind, 14th March in Munich. To learn more and to sign up for the event click here.

 

 

Seasoned drug development professional Kim Forbes-McKean PhD to lead NDA Group’s US expansion

Kim Forbes-McKean PhD, joins NDA Group from a distinguished career spanning more than 30 years of leading research and development activities for large pharma, as well as small biotech companies.


“Kim brings with her tremendous experience of the challenges facing development companies all over the world,” commented Johan Strömquist, CEO NDA Group.
“I am very happy and proud to have her join the NDA Group management team to further expand our activities in the US and I believe her expertise and experience will be of great value to our clients.”

Prior to joining NDA Kim was responsible for worldwide development of dermatology products at various companies including, Rhône-Poulenc Rorer, Aventis-Sanofi and Aqua Almirall, overseeing seven successful regulatory approvals and product launches.

She co-founded a dermatological development company and has been an executive member of management teams who have successfully raised over $90 million to support business development, licensing objectives and drug development activities which lead to partnering or acquisition opportunities.

Kim has built and led teams across all phases of development to advance global drug and device programs as well as for support and maintenance of commercial products. She has extensive experience and has served as a company representative directly interacting with the FDA throughout the development and approval process.

“In Kim, not only do we have an accomplished scientist and strategic regulatory thinker, but an experienced business professional who has been in our clients’ shoes,” commented Laurie Smaldone-Alsup, CSO/CMO NDA Group. “Kim understands the pressures and priorities of large pharma as well as small biotechs and the trade-offs facing CEOs and drug development executives on a daily basis. I am delighted to welcome her to our team. “


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. www.ndareg.com

NDA media contact:
anna.perrin@ndareg.com

 

 

 

Free Webinar – Brexit Impact: Are you ready?

The current political turmoil in the UK surrounding Brexit is causing confusion and uncertainty across the board. With so much uncertainty, how can the Pharmaceutical and Life Science sector prepare for the upcoming changes? With less than two months to go until the 29th of March, solutions and plans need to be put in place.

Join us on 14th February at 15:00 GMT, 16:00 CET, 10:00 EST for this engaging webinar where Thomas Lönngren, Strategic Advisor, and Dr Brian Edwards Principal Consultant, Pharmacovigilance & Drug Safety will discuss the following:

  • What are the changes that are relevant for the industry?
  • Which processes need to be examined for the necessary changes?
  • What solutions are available to facilitate these changes?

Although no one can pretend to have a crystal ball and predict the outcome of Brexit, there are practical arrangements that can be put in place to protect the legal status of your marketing authorisation and authority to operate in the EU!

This webinar will enable you to reflect on how well your own plans are progressing and what more needs to be done.

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


Click here to book your place today!


Thomas Lönngren, Strategic Advisor, NDA Group
Former Executive Director of the European Medicines Agency (EMA).

Dr Brian Edwards, Principal Consultant, Pharmacovigilance & Drug Safety, NDA Group,
GMC registered physician with previous experience in hospital, renal medicine and clinical research, as a pharmacovigilance assessor in the UK regulatory authority (MHRA), clinical trials and post-marketing pharmacovigilance in a global CRO, deputy QP for pharmacovigilance at Johnson & Johnson.