Q&A - Launching ATMPs in Europe

[Music] hello I'm longy chain vice president and head of the celling therapy service line at Sora we're a team of dedicated experts who collaborate with biofarma companies on the complex Journey from research and development to commercialization of advanced therapeutic medicinal products or atmp our Focus lies in crafting and executing comprehensive strategies encompassing distribution Logistics patient support Market access and Regulatory Affairs throughout this crucial process Herbert I'll turn it over to you thank you L and I'm Herbert Alman I'm vice president of Market access and Healthcare Consulting in Europe I'm with this organization now a little bit more than one year and before that I was more than 15 years with Novartis also responsible as Market access hat for Europe in this context I had also the opportunity to participate in two pilot projects with the J joint action 3 procedure and also was the representative in the FP work group for HTA assessment um in Europe we have a team of around 300 people mainly in the countries which have direct interaction with our customers in the countries to to ensure that they bring Innovative medicines as fast as possible to [Music] patients yeah the general objective of the EU HD the EU HDA regulation which will be implemented uh very soon in January 2025 is uh uh threefold um first of all uh to reduce bureaucracy uh second to speed up the entire process and third uh to bring uh Innovative products in an equal manner to patients in Europe um of course uh Europe want to become an interesting place to invest of course and launch Innovative products for example compared to the US um and therefore this existing system that we have today so that a manufacturer after IMA approval has to go to um 27 different member states with the HDA assessment should be centralized in this process so to do such a submission jointly and conduct this assessment process the first out of three steps in this process in one uh um yeah in one jca joint clinical assessment procedure makes a lot of sense and helps uh the manufacturer to reduce bureaucracy [Music] atmps present a unique challenge for HTA processes due to their nature as products based on genes tissues or cells they are primarily utilized to treat rare diseases to date and essentially approved by the EMA now if you look at the HTA recommendations across different countries over the last few years on atmps there is a lack of consensus on the value of atmps compared to the standard of care this discrepancy is attributed to a couple of different things but mainly around a clinical trial design such as the size of the trial population diverse comparators uh and limited long-term clinical data at the time of submission resulting in incomplete evidence for evaluating their efficacy the other thing is the high cost associated with atmps that really complicates pricing and reimbursement decisions and raising a lot of concern around the affordability of healthc care system it is crucial to address these uncertainties surrounding atmps to ensure access to Innovative therapies that cater to high unmet medical needs um often serving as The Last Hope and resour for patients so to mitigate assessment variances the adoption of the new eua regulation could play a vital role in harmonizing evaluation practices and reducing disparity ities um that Herbert talked about earlier now by fostering better understanding and Alignment within HTA Frameworks we can really pave the way for more consistent evaluations of these Advanced [Music] therapies yeah very good question uh my first of all there is an EMA process which uh look looks into safety and efficacy of a product and principle and uh after this regulatory approval the HDA process makes an important uh next step to make a product uh um evaluated regarding pricing reimbursement of course and value versus other Pharmaceuticals and so far in Europe you have to go as a manufacturer to 27 different countries after EMA approval which is the centralized regulatory process to uh submit an HTA dossier which will be the basis for typical assessment here the HTA assessment is a next critical step for a manufacturer to get a product priced and reimbursed in the market and make it finally available for patients of course uh in the regulatory process you have an assessment of safety and efficacy and then the HDA bodies in the different member states have to assess how this product can fit into the local environment um from the affordability Point as Luni said before also pricing reimbursement what kind of patient population should be addressed uh and also criteria like which kind of Physicians can prescribe this product and uh of course this uh HDA assessments if you go to 27 different countries will have 27 different procedures and with the same evidence package to go to uh 27 member states of course results then in a a very different timeline and also um results from this uh assessment uh procedure so a launch sequence from Germany which is launching usually uh weeks after EMA approval until other countries which takes one and a half until 2 three years in some central eastern European countries um can spread can be a widespread in timing now what kind of criteria are important first of all um a country is evaluating the value of a product versus standard of care so there's a concrete comparator uh which is used to see what kind of value this additional value this product can have at the same time also safety criteria will be evaluated to make sure to that this produ product is meeting the standards and the different requirements and then this assessment report this HDA assessment report will be the basis for the following uh pricing and reimbursement discussions as said before so it's very important that the quality of the assessment is as high as possible so that an accurate positioning of the product in the market is possible [Music] after uh many years of um evaluating with the member states the European commission has decided in 2022 to implement this regulation and the start date for the first products which are oncology products and atmps is 12th of January 2025 so not far from here only 9 months from here and uh after that in 2028 orphan drugs will be the next class which will be assessed and uh included into this new process and then 202030 all products are supposed to be in the EU HDA regulation covered so there is quite some opportunity to learn now also how this system works and how the processes Works in with all the Healthcare System stakeholders manuf facturers but also the different countries and learn and improve the system as much as [Music] possible to ensure a smooth and effective jca process it is crucial to Kickstart the internal Market access planning at least 10 months prior to filing with the EMA this timeline provides ample opportunity for thorough preparation meeting jca dossier requirements and crafting a robust evidence package for submission critically before designing the phase three clinical trials it is essential to align the internal processes as well by establishing coherence within the organization early on companies can really streamline their approach to jca and optimize the outcomes of their clinical trials prioritizing the development of cross functional cap capacity and capabilities is also important building a team that encompasses diverse expertise and skills when not only enhance collaboration but also bolster the overall preparedness for successful Market access strategies so really by adhering to these key principles of initiating early Market access planning aligning internal processes proactively and prioritizing cross functional capacity building companies can set themselves up for more seamless an efficient jca process while maximizing their chances of success in gaining Market access um Herbert anything to add no I think you're absolutely right L that uh an early and a cross fun an early start and the cross functional interaction is super important for uh being successful so um first of all it's also the JSC The Joint scientific consultation which manufacturers can use nowadays or in the future to get advice from EMA and HDA bodies at the same time then as a next step in the preparation you have also the opportunity to do Pico simulation so to find the right uh patient indication comparators and outcome parameters which are all assessed then from the HDA bodies from the different countries um and in this collaboration it's also important that the country organizations or country HDA bodies are consulted to make sure that they learn about atmps and the new products because very often especially in smaller countries it's not so easy for the authorities to get familiar with this new technology and uh therefore for the markets uh as well as for the HDA bodies it's super important to start early engage early and do it as a cross functional team [Music] atmp developers have a great opportunity to leverage insights from Pilot assessments such as joint action three um to tailor their approach strategically uh to enhance their evidence generation and to navigate potential challenges effectively by doing so they can significantly increase their chances of successful uh regulator approval but also Market access now there's a couple of things first um you want to analyze the outcomes of the pilot assessments uh developers can gain a deeper understanding of the specific requirements and expectations of regulatory bodies and HTA uh bodies concerning atmps this knowledge is crucial for aligning development plans with the standards set by these authorities moreover these assessments enable developers to proactively identify by potential challenges or gaps in their evidence package early on by addressing these issues during the development phase companies can then strengthen their overall strategy and ensure a more robust submission for approval furthermore leveraging insights from Pilot assessments helps in optimizing evidence generation strategies to meet regulatory standards effectively developers can learn best practices for collaboration with Partners regulators and HTA bodies based on the experiences shared during these assessments and really kind of enhance that cooperation and streamlining the processes the last part is atmp developers can refine their Market access strategies by learning from the successes um adapting proven methods based on pilot assessment allows companies to tailor their strategies for better outcomes in these reviews and Market access decisions so to just sum it up by incorporating these key learnings into their development plans a atmp developers can position themselves for success in navigating the complex landscape of regulatory approvals and Market access for these Innovative therapies you're absolutely right L um I was able to participate in two of the pilot projects when the unetta Consortium 21 was offering this opportunity to get familiar with the new process and there were a lot of internal discussions how to prepare for these products and for these assessments uh but on the other side there was also a lot of dialogue between the different uh um countries as assessing our products and therefore I can only agree that an early uh engagement makes a lot of sense to uh identify the gaps in the evidence package as they are perceived from The Authority point of view [Music] and in this context The Joint scientific consultation offering that we have now available in the new EU HDA regulation plays an important role the manufacturer has the opportunity to engage with the EMA the regulatory body and at the same time with several HTA bodies and for the first time you get a Consolidated feedback from Regulatory and HDA bodies about the request what kind of evidence is needed to get a product uh um assessed and Regulatory approved and then of course afterwards qualifying for pricing and reimbursement process in the country at that stage usually the manufactured have not decided what kind of phase two phase three clinical trial design is in place and how to structure it fully but has a clear expectation how this can look like and therefore it's good to resonate with the HDA bodies also at that stage already to make sure that the evidence package is not only ready for regulatory approval with Emma which historically was the focus but also meets the requirements of an HDA body in Europe [Music] many atmp developers particularly emerging companies often face challenges due to the lack of wellestablished local affiliates by actively engaging with national HTA bodies these developers can effectively navigate the intricate landscape of local reimbursement and access processes ultimately enhancing their Market penetration strategies that's right Luni and there is a big difference between smaller companies and the bigger farmer companies the bigger farmer companies have a well structured organization and they can learn from the different products that they are launching over time while the biotech companies or Smiths the smaller companies which are engaging with one or two compounds over a period of 3 five years don't have the opportunity to learn National HDA bodies offering valuable insights into specific Regional requirements preferences and decision- making criteria that significantly influence Market access strategies for atmps in individual countries understanding these nuances is essential to tailor evidence generation approaches and value propositions to align with each Market's unique needs collaborating with national HDA bodies enables developers to customize their strategies based on local insights thereby increasing the likelihood of successful HTA evalu ations and favorable reimbursement decisions in diverse markets this targeted approach enhances the relevance and the impact of their submissions by familiarizing themselves with the expectations and procedures of national HTA bodies early on atmp developers can proactively anticipate and address challenges Paving this way for smoother Market access Pathways for their Innovative therapies this proactive stance minimizes obstacles during the evaluation process another important aspect is that engagement with national HDA body serves as a platform for fostering relationships and dialogue between developers and key stakeholders across different markets these interactions facilitate constructive discussions on evidence requirements Health economics and patient access issues promoting Mutual understanding and collaboration toward achieving shared goals I think you're absolutely right L also because we have quite some experience with early scientific advice in Germany that always offers an opportunity for any kind of manufacturer to assess if the clinical trial design the comparator selection or the PO patient population are relevant also for the HDA system but it has to be shown if this also works in this new environment because there are many smaller countries which don't have this kind of experience yet so interesting situ ation to see how this develops you're exactly right through active engagement with national HTA bodies atmp developers can gain a Competitive Edge by tailoring their strategies to meet diverse Market needs effectively while building meaningful Partnerships within the healthcare ecosystem you're right Lungi but let me add that it's also important to add the patient perspective because it's not only important to evaluate if the new drug or the new technology has the right comparator right clinical trial design but it has to also take into consideration the unmet medical need of patients and that this new technology will reach patients as fast as possible in this collaborative approach between HTA body and manufacturers yeah I canot agree more Herbert because for atmps so many of them are targeting rare or ultra rare conditions where there were no treatments before so patient really is at the center of what these developers are trying to help so that patient perspective cannot be forgotten during the whole process absolutely and uh especially as smaller countries may have only a few patients not even a patient within a year but only two or three within five years so they had to they have to get more familiar with the technology in an early phase to also prepare their environment to imp to prepare the healthc care system and the budget uh the M that's also [Music] important this is a question that's really top of mind for a lot of developers um the atmp sector is on the cusp of this transformative ERA with new treatment paradigms entering the market and a wave of therapies targeting broader patient populations just on the horizon if you look at the projections as many as six atmps could secure regulatory approval in Europe in 2024 signaling a significant expansion in Innovative therapies now what new regulations hold promise for enhancing patient access to Cutting Edge treatments they also present notable challenges that demand our attention there are concerns regarding the applicability of the current methodologies to address atmps effectively of particular concern is this requirement for randomized control trials which may not always align with the unique characteristics of atmps this insistence could potentially result in inconclusive jca reports for numerous atmps highlighting the need for tailored evaluation framework to accommodate these Advanced therapies more effectively you're absolutely right L but let me add that and remind ourself that this is only the first step of the the HTA assessment in the jca there's next step is that the jca report is delivered to all the 27 different countries and then an additional assessment is happening there so we have to make sure that there are not too much additional requirements because that's an additional concern uh then for the manufacturer and it will delay this new Innovations to come to patients as fast as possible that's a really great point because the jca doesn't replace the local level decisions and review right so I think when you think about the resources and just the processes on both ends yeah the I would only hope that over time the there are not many additional requirements especially for atmps and rare conditions because already in the jca process every member state has an opportunity to contribute so in this process additional requirements should become very less let's hope so we need to acknowledge both the opportunities and challenges within this Dynamic sector and work towards fostering an environment that supports Innovation while ensuring robust evaluation processes that align with the distinct attributes of atmps [Music] yeah I think it's uh very important that uh manufacturers are proactive planning and doing a lot of collaboration with uh the HDA bodies on a local or U on a European level um there is still a lot of uncertainty the first uh draft of the implementing act has just been published and uh it created uh some concerns with manufacturer how to best interact in this new healthcare system environment so far it's not obvious that manufacturers can participate in giving input to the entire um process uh during the evaluation of their product it's also not very clear how patient organizations and K can participate and um launch their opinion about a certain technology which is super important because in these atmp and ology conditions it's important to have their uh very good input from the beginning therefore I think it's important to partner to leverage the different learnings from the industry associations which are available for example fpia and yop but also look into other Partnerships to learn from these examples which went already through the process yeah so I think her that's really well said and I would be remissed not to mention mention the eua center of excellence we just established as Sor um as an industry leader we really want to see new medicin being accessible to more patients in Europe um I think we have a really good track record of navigating companies throughout this very complex process uh from a national level and now with experience that we have with JC uh and very soon jca uh we are very proud of what we can do for patient uh with our expertise with our experience I think to your point this is new for a lot of people we're learning as everybody else is learning as well but I think the focus the dedication and the attention that we'll provide to our uh clients current and perspective is going to be so critical to uh bringing more successes uh to uh the you EU Community uh when it comes to having these uh Innovative medicines you're right ly and I think in addition we can even leverage all the experience that we have already how to do dosses in Germany with the amnok procedure for example and Germany is one of the leading countries in shaping this EU HDA process in the future we see this also with the first implementing act that a lot of requirements how to do the dossier are very similar to the amnok process and we have done so many of these dosses but we have the same capabilities in Spain or in Italy or in other countries in Europe so um hopefully we can learn fast and therefore help our partners to bring their Innovative medicines as fast as possible to patients I agree 100% I think we have a really really strong Foundation based on what we've done and we can Le it better from here [Music]

Channel: AmerisourceBergen

Views: 1,836

Keywords:

Id: OKdUmbwKghY

Length: 27min 30sec (1650 seconds)

Published: Wed May 08 2024