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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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!

 

 

 

Europe vs USA: new drug product approvals in 2018

By Anna Leitgeb, Consultant, NDA Group

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

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

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

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

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

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

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

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

Successful Exploration of Novel Drugs

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

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

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

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

A Nobel Prize and its result in a Novel drug

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

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

SMEs and Approved Orphan Drug Designations

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

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

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

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

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

Improvements within Precision Medicine

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

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

Expedited approval of novel drugs

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

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

NDA supported over 40% of the approvals in the EU

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

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


References

i. The data was gathered from the EMA and FDA official websites, as reported on the FDA and the EMA official websites on January 2019.
ii. Fire A., et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391:806-811.
iii. Mullard A. FDA approves landmark RNAi drug, Nature Reviews Drug Discovery 2018;17:613
iv. https://www.ndareg.com/europe-vs-usa-new-drug-product-approvals-in-2017/


 

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!

 

 

 

Design and optimisation of a quality target product profile for ATMPs


The quality target product profile (QTPP) is an inherent part of product development and provides an overview of all the elements that have an impact on the quality, safety and efficacy of the product in a given clinical indication. The concept is defined in the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use guideline Q8 and may be more familiar for those developing conventional pharmaceuticals.

However, it also provides an excellent tool for advanced therapy medicinal product developers and should be used to consider all elements that have an impact on the ultimate quality of the product and, consequently, the safety and efficacy in clinical and commercial use. Building up the QTPP should start at the research phase and continue up to the marketing authorisation application (MAA) phase; if it is put together properly and regularly updated, it provides the skeleton for the entire chemistry, manufacturing and control module of the MAA.

To find out more read the full article written by NDAs Director of Biopharmaceuticals and ATMPs Paula Salmikangas. The article is also featured in Regulatory Rapporteur – Vol 16, No 2, February 2019.

 

 


By Paula Salmikangas – Director of Biopharmaceuticals and ATMPs, NDA Advisory Board

 

 

 


 

 

Rare Diseases and FDA Advisory Committees: Be the Experts in the Room


checkWhen it comes to advisory committees that are convened for rare diseases, everyone must become an ‘educator’. This includes the applicant, external experts and open public hearing participants.

By definition, a disease is considered rare if it affects fewer than 200,000 people in the United States. However, an estimated one in ten Americans has a rare disease and about one third of all new drugs approved by FDA are now for rare diseases. In fact, in 2017 the FDA approved a record 80 new treatments for are diseases.

Whenever an FDA advisory committee is convened as part of the approval process, the stakes are high and there can be communication challenges. However, for applicants preparing for an advisory committee that is for a rare disease, the challenges are unique.

Read the full article written by Neelu Agrawal, expert in high stake meeting preparations, including FDA Advisory Committees, Oral Explanations, and Scientific Advisory Groups, to learn about some of those challenges, along with key factors for success.

 

 

 

By Neelu Agrawal – Principal, NDA Group/PharmApprove