Join us on Thursday 3rd October 15:00 BST, 16:00 CEST, 10:00 EDT for this engaging webinar where Dr Paula Salmikangas, Director for Biopharmaceuticals and ATMPS, NDA Advisory Board and Claes Buxfeldt, HTA Director NDA Advisory Board will describe an integrated product development process for ATMPs, which will take key aspects of both reviews into account from the beginning, thus ensuring a positive path from clinical trials up to pricing and reimbursement.
Advanced Therapy Medicinal Products (ATMPs, or cell and gene therapy and tissue engineering products) is an increasing group of innovative products, often targeting diseases and conditions with high unmet medical need.
The success of first CD 19 CAR T products against B-cell malignancies has raised awareness of the high potential of these new products, but also shown the several challenges relating to their manufacturing and regulatory approval. The prices of the first approved ATMPs have been high and not always supported by the national pricing and reimbursement bodies.
In such cases, the discrepancy between regulatory approval and negative result of a health technology assessment (HTA) has raised concerns and questions within the industry, as to how to ensure an approved product also gets to the market and patients. Many jurisdictions have created early access schemes and ways to communicate with regulatory and HTA bodies early on to ensure successful outcomes of both reviews. However, the ATMP industry is facing challenges in both aspects.
Why you should attend:
See how the regulatory and HTA expectations differ
Learn how to build an Integrated Regulatory / HTA Product Development for ATMPs
Identify the need for interactions with regulatory and HTA authorities
Paula is a clinical biochemist by original training, with a Ph.D. in muscle cell biology. Her main research work career has been in cell and molecular biology of various inherited diseases. Since 2006, she has been an Adjunct Professor of Biochemistry for the University of Helsinki. Paula joined NDA in 2017 from her position as a Research Professor at the Finnish Medicines Agency (2003-2017). She has served as a member of the EMA Committee for Advanced Therapies (CAT) from 2009 to 2017 and as the Chair of the CAT 2014-2017. She has also been the Chair of EMA CPWP and a member of the BWP. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and CMC aspects of biopharmaceuticals.
Claes joined pharma-industry in 1992, have close to 20 years’ experience working in local and global market access and health economics positions, in a variety of disease areas. Prior to joining NDA he spent 10 years at AstraZeneca starting as a Value Demonstration Leader in Global Health Economics & Outcomes Research, and most recently as the Global Price & Reimbursement Director in Respiratory & Inflammation, in addition to CNS/Pain.
He has supported more than 30 molecules/brands in development in a global position. He has represented the payer voice in many development programs, including the development of payer evidence strategies, pricing and market access strategy, economic models, PROs, RWE and clinical program input. Claes has a MSc from University of Karlstad and more recently a postgraduate diploma in health economics from University of York.
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.
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:
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.
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.
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.
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.
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
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.
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.
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 firstname.lastname@example.org. Indicate if you would like to discuss them openly during the meeting; otherwise we can book separate meetings to discuss them after the seminar.
In this white paper NDA’s CEO, Johan Strömquist, discusses four ways that Health Technology Assessments will change under the new proposed European regulation.
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.
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.
Being able to demonstrate the expected value for health care systems and society for a new pharmaceutical is critical for market access including chances of reimbursement. Having a clear strategy for value demonstration as an integrated part of your drug development program can improve your chances of reimbursement leading to a faster market access. Knowing different payer’s expectations in terms of endpoints and supportive data as well as having an understanding of Health Technology Assessment (HTA) and how this is applied is critical to success.
Join us on Thursday 29th November, at 15:00 GMT, 16:00 CET, 10:00 EST for this engaging webinar where Claes Buxfeldt, HTA Director, will cover the following:
The needs/requirements of the HTA bodies and payers in all countries
How strategy could be implemented in TPP, value proposition and messages to secure value and reimbursement
What activities are needed to investigate and at what stage in development
design and improve a clinical development program to satisfy HTA bodies and deliver value for payers
design and improve programs to strengthen unmet need and the economic story
prepare and improve the ability to communicate the value story and improve payer negotiations skills
validate your strategy and plans using the NDA Advisory Board, both regulatory and HTA / payer
The webinar will be followed by a Q&A session for you to get direct feedback on key areas of uncertainty.
Claes Buxfeldt, joined pharma-industry in 1992 and has close to 20 years’ experience working in local and global market access and health economics positions, in a variety of disease areas. Prior to joining NDA Claes spent 10 years at AstraZeneca starting as a Value Demonstration Leader in Global Health Economics & Outcomes Research, and most recently as the Global Price & Reimbursement Director in Respiratory & Inflammation, in addition to CNS/Pain. He has supported more than 30 molecules/brands in development in a global position. He has represented the payer voice in many development programs, including the development of payer evidence strategies, pricing and market access strategy, economic models, PROs, RWE and clinical program input. Claes have a MSc from University of Karlstad and more recently a postgraduate diploma in health economics from University of York.
On 24th October, Carole Longson, Dr Steffen Thirstrup & Claes Buxfeldt, NDA Advisory Services Ltd, will be holding a complimentary seminar on market access/Health Economics and Outcomes Research (HEOR), discussing the future of HTA.
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.
This seminar will discuss the past, present and future of HTA and market access.
Stockholm 2018-02-07 – NDA Group, the world’s leading drug development consultancy, signals their appreciation of the EU Commission’s proposal for a new HTA regulation in EU. The particular focus on Relative Effectiveness Assessment, the establishment of a new Coordination Group of clinical experts from the Member States and the continuation of the practices established by the voluntary European HTA network were all cited as reasons for a promising future for the regulation.
On Wednesday January 31st the EU Commission released its proposal for a new regulation on HTA for all Member States of the EU. The new regulation builds on the long-standing activities of the Member States HTA agencies, and particularly the voluntary network and joint action programs hosted by the EUnetHTA network.
Based on the EUnetHTA recommendation and the Impact assessment report (1) that provides an in-depth analysis of four policy options, the Commission has chosen to propose the option 4, advocating permanent cooperation on common tools, procedures, early dialogues and joint HT assessments. In the proposed regulation, the use of joint assessment of clinical aspects of technology evaluation becomes mandatory; all other aspects, such as cost-effectiveness and economic assessment, legal, ethical and societal aspects, are left for the Member States to continue to manage.
While specifying that regulatory and HTA process will remain well separated, the regulation focuses on three particular areas of interest to the drug developing industry:
Joint clinical assessments limited to innovative technologies,
Provision of scientific advice and guidance to developers, and
Identification of emerging health technologies.
The execution of these activities will primarily be carried out via a new Coordination Group of Member State experts, helped by specific sub-groups for each area of interest, and a new Commission Secretariat supporting the processes.
The regulation can be expected to be adopted in 2019, if successfully negotiated and approved by European Parliament and Council of Ministers, after which two subsequent three-year transition periods would kick in before it is fully adopted by the Member States.
The NDA Group has supported drug developing companies with joint regulatory and HTA assessment since 2011. Many of the experts employed by the company in this process have been involved in setting up the systems in place today, and the new regulation is no exception.
NDA’s Dr. Mira Pavlovic-Ganascia, who formerly coordinated the work on several EUnetHTA guidelines and work packages as well as the SEED (Shaping European Early Dialogues) sponsored by the Commission, commented:
“It is reassuring to see that the Commission has taken so much of the work of the [EUnetHTA] network on-board. I truly believe that a new Coordination Group will continue to profit from the actions achieved and structures already put in place, such as Early Dialogues Working Party and ED secretariat. As joint clinical assessments are the Commission’s key measure to promote market access for innovative technologies, Option 4 became the natural default position and with some minor modification this is what the Commission has chosen to go for.”
NDA’s Dr Thomas Lönngren, former Executive Director of the European Medicines Agency added:
“The work that was started over 20 years ago has now finally reached the legislative phase. This is a breakthrough, but the legislative process in Europe is difficult to navigate and it will take time before we eventually see this proposal adopted and implemented.”
When NDA established its Joint Advice service, it was to reduce the uncertainties inherent in drug development, particularly in gaining approval from regulators and efficiently ensuring that the product could be reimbursed by the national payers. One of the challenges has been the fragmentation and varying degrees of maturity across the Member States and an evolution towards the Commission’s proposal has been a favorite discussion topic in the consultancy over the last few years.
Johan Strömquist, CEO of NDA Group, commented:
“It’s quite incredible to see what we’ve expected, for such a long time, presented as a clear legislative proposal. Our experts predicted this development several years ago and the new regulation will certainly make the lives of hundreds of developers easier and more predictable.”
“As the leading experts in the field we welcome the Commission’s proposal. We stand at our clients’ disposal to support in the creation of the necessary submissions and preparation for engaging with the new Coordination Group to attain the best possible advice for their product.”
Dr. Thomas Lönngren, Strategic Advisor NDA Group and former head of the EMA, has been invited by BioMelbourne Network to present at the CEO lunch forum entitled “A CEOs nightmare – Regulatory and Market access failure, How to get a good sleep?”
This exclusive forum is being held on 2nd February at Norton Rose Fulbright, Melbourne.
Dr. Lönngren will present and discuss the common failures in drug development from a regulatory and market access perspective and how to avoid these failures; best practices for regulatory and market access strategies and discuss with the audience the latest pharma sector hot topics including the Brexit.
Early integrated regulatory and market access strategies are critical to success. By planning early and well, your team can play chess, not tennis, with decision-makers.
Join us on 30th November to hear NDAs Laurie Smaldone Alsup, MD, COO/CSO, discuss:
Insight into the HOW to avoid market delays that impact patients and ultimately reimbursement
The importance of early planning, pressure testing and preparation for the make or break end game
How to convince payers that your product brings important value to patients and to the system.
In addition, she will share proven methods to critique and provide strategy through expert panels that pressure test the pivotal trials designs and value documentation. These methods can help align companies internally and efficiently focus investments. Cross functional teams that can benefit from these strategies include regulatory, clinical, outcomes research, marketing, market access, commercial and medical affairs operations.
Ultimately at the end of product development the key product messages must be communicated through the pharmacoeconomic value dossier in seeking reimbursement. We will focus on the importance of internal communication across development and commercial teams, and on how to prepare for payer negotiation training and affiliate alignment.
Dr. Smaldone Alsup will be available to answer questions at the end of the session.
Dr. Smaldone Alsup has over 25 years of global leadership experience in the pharmaceutical and biotechnology industries in drug development, regulatory strategy and regulatory approvals across all major therapeutic areas. She has led the development and commercialization of drugs for oncology, HIV/AIDS, cardiovascular, metabolics, immunology, infectious diseases, neuroscience, dermatology, inflammatory and immune-based diseases. She has led numerous multidisciplinary teams in over 50 US FDA and European EMA proceedings, including preparations for numerous key FDA meetings, Advisory Committee hearings and EU Scientific Advice and Oral Arguments.
Dr. Thomas Lönngren, Strategic Advisor NDA Group and former head of the EMA, has been invited to speak at the The World’s First and most Prestigious Early and Managed Access Programmes, EMAP, event on the 24th – 26th October in London.
The Pharmaceutical Industry continues to turn to Early and Managed Access Programmes as the front line in fighting rare diseases and epidemic outbreaks. In many cases, critical patient or environmental conditions mean that any delay in access can be fatal.
Dr. Lönngren will present in the session ‘Regulatory development and alignment between the USA and Europe’ where he will discuss:
Differences of market access in EU and US
How to create a collaborative environment between the EMA and FDA – where to
Analyse what Brexit means for the UK, EU and for medical technology
and pharmaceutical companies
Professor Ken Paterson, HTA expert at NDA Group, has been invited to present at the EACPT Focus meeting on September 8th in Opatija, Croatia. The title of the meeting is “How to Assess Medicines from Research to Clinical Practice? Efficacy, Effectiveness, and Economic – 3E Assessment” and Professor Paterson session is titled “Rapid Health Technology Assessment of New Medicines – Lessons from 15 Years Experience in Scotland.”
The European Association for Clinical Pharmacology and Therapeutics (EACPT) is a learned society in the field of clinical pharmacology. It is the leading society in Europe serving the European and global clinical pharmacology and therapeutics community. The EACPT includes all national organisations for clinical pharmacology in Europe and provides educational and scientific support for more than 4000 individual professionals interested in clinical pharmacology and therapeutics throughout the European region, with its congresses attended by a global audience.
To read more about EACPT click here and to read more about the Focus meeting click here.