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/


 

Free Webinar – Brexit Impact: Are you ready?

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

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

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

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

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

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


Click here to book your place today!


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

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

 

 

Meet NDA at DIA Europe 2019

 

We will be attending in full force during this year’s DIA Europe 2019 – 5 – 7 February 2019| Austria Center Vienna

If you are planning to be there, we would really like to meet up with you!

You can find us throughout the event at booths: B71 & B72 where we have a range of experts covering the full regulatory affairs spectrum – from preclinical and clinical to pharmacovigilance and health technology assessment – all of whom are looking forward to meeting you:

  • Professor Beatriz Silva Lima, Non clinical Expert
  • Professor Steffen Thirstrup, Clinical development and regulatory strategies Expert
  • Lisa Peluso – Director, Coaching and Client Engagement
  • Shelley Gandhi, Ex MHRA regulator, Pharmacovigilance & Drug safety Expert
  • Dr Mira Pavlovic – HTA Expert
  • Brian Edwards – Principal Consultant, Pharmacovigilance & Safety

In addition, there will be other NDA’s expert consultants attending the event and happy to help.

We definitely recommend booking a meeting in advance to ensure availability, but do feel free to pop by booths B71 & B72.

If you would like to arrange a meeting, please contact my colleague Anna Perrin who will be happy to assist: Phone +44 (0) 1372 860 610 or Email anna.perrin@ndareg.com


Sessions

We will also have some of our team presenting on interesting industry topics during the event.

Please look out for them:

Prof. Steffen Thirstrup – Director and Medical Advisor, NDA Regulatory Advisory Board
Lisa Peluso – Director, Coaching and Client Engagement, PharmApprove, a member of the NDA Group
#SC02: Short Course 2 | Mon, 4th February – 14:00

European Regulatory Meetings – How to Best Prepare and Perform


 

Prof. Beatriz Silva Lima – Non Clinical Expert, NDA Regulatory Advisory Board member
#SP01: | Weds, 6th February – 15:15
Nanomedicines and Nanosimilars – Implications for Regulators, Payers and Prescribers Regulatory Considerations for the Approval of Nanomedicines and Nanosimilars

Hub 1 #CH104 | Thurs, 7th February – 09:15
Patient-reported, Patient-relevant, Patient-centred outcomes: definitions, roles and importance for health technology assessment

#S0206: | Thurs, 7th February – 10:45
Regulatory Innovation: Regulatory Science

 


Dr Mira Pavlovic – HTA Expert, NDA Regulatory Advisory Board member
Hub 1 #CH104 | Thurs, 7th February – 09:15
Patient-reported, Patient-relevant, Patient-centred outcomes: definitions, roles and importance for health technology assessment

 


Shelley Gandhi – Strategic Advisor, Pharmacovigilance & Drug Safety
#S0307 | Thurs, 7th February – 12:00
Capacity Building and Capability Building in Pharmacovigilance

 

 


Poster Presentation:

Brian Edwards – Principal Consultant, Pharmacovigilance & Drug Safety
Hub 2 | Tues, 5th February – 13:00
CAST analysis of UK pregnancies reported after isotretinoin administration

 


Click here to download the Preliminary Programme.


 

Meet us at DIA Europe 2019

To book a meeting contact Anna Perrin: anna.perrin@ndareg.com or visit us at booths B71 & 72.

We look forward to hearing back from you about a meeting and to seeing you at the event!

 


 

 

What Do Providers Need to Know About Biosimilars?

DIA Biosimilars Conference, held October 22 to 23 in London, United Kingdom

With a number of new biosimilars making their way to market and eventually to the clinic, it is crucial that healthcare providers become educated about and comfortable with biosimilar products. During a session at the fifth DIA Biosimilars Conference, held October 22 to 23 in London, United Kingdom, experts addressed a number of key areas for provider education.

Paul Chamberlain, immunogenicity specialist at NDA Advisory Services, spoke about one of the biggest concerns for prescribers who are wary of biosimilars: immunogenicity

Here’s a summary:

Immunogenicity.
According to Paul, it is critical to clarify the definition of immunogenicity, which is an undesirable host immune response to administration of a therapeutic agent. The innate immune response, the adaptive immune response, and immune tolerance are the key drivers of immunogenicity, and product factors (such as glycans, process-related impurities, or process changes) and patient factors (such as comorbidities or concomitant therapy) can impact the balance of those 3 factors.

While it is not feasible to predict how each factor might interact with others to affect immunogenicity, he explained, individual factors can certainly be controlled within acceptable limits. Furthermore, regulators look at both the individual and population level for immune response, and they also require a well-defined risk management plan for a product.

Thus, said Paul, immunogenicity is “the wrong elephant in the room” for prescribers, because physicians typically conflate the incidence of antidrug antibodies (ADAs) with immunogenicity. “Please do not just use ADA rate … as a term which is equivalent to immunogenicity. It absolutely isn’t.”

To illustrate his point, Paul gave the example of CT-P10; in looking at ADA titters between the biosimilar rituximab and its reference, regulators can see that while ADA rates for the 2 products may not appear comparable, the ADA titters overlap very closely. However, he said, few physicians see these reassuring data.

For a more complete picture please see Pauls slide presentation.

To read more about what the experts had to share please click here.

 

 

 

Free Webinar – How to demonstrate value in drug development programs to improve chances of reimbursement

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
  • How to:
    • 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.

Click here to book your place today.

 


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.


 

 

Meet NDA at DIA European Forum for Qualified Person for Pharmacovigilance (QPPV)

Helen Powell, & Brian Edwards, Principal Consultants at NDA Group have been invited to chair two Sessions at DIA European Forum for Qualified Person for Pharmacovigilance (QPPV), London, 10-11 October 2018

“Are You Compliant Enough? – Audits, Inspections And QMS”
Helen Powell, Principal Consultant, NDA Group has been invited to chair session 7

 

 

“Am I Impacted By Brexit?”
Brian Edwards, Principal Consultant, Pharmacovigilance and Drug Safety, NDA Group, will chair session 8

 

 

Overview
This is still the only forum designed for QPPVs by QPPVs. This Forum continues to identify key trends requiring QPPV awareness, input and oversight. This year’s objectives, will build on past successes which have been shaped by valuable feedback provided by the participants of the past eleven meetings, plus many years of QPPV and Regulator interaction at this Forum.

Objectives
• Hear the latest updates and hot topics relating to the role of the QPPV
• Explore long term PV visions, future directions of the ‘PV world’, and potential impact on the role of QPPV
• Network with colleagues and meet regulators
• Learn from and share experience and ideas with like-minded QPPVs in a neutral environment
• Take away practical hints and tips
• Better understand regulatory and inspectorate expectations of the QPPV
• Identify the expanded expectations of the role in the context of the continually evolving regulatory framework
• Examine current areas of real challenge

To find out more about the event click here.

Europe vs USA: new drug product approvals in 2017

By Terese Johansson PhD, Consultant, NDA Group

It’s been an exciting year for new drug approvals! Many of the new drugs approved during the year address significant and meaningful needs or give additional therapeutic choices for patients and physicians. In the US we have seen a ground breaking approval in oncology that changes the way we look at and relate to indications; in addition the first digital pill has seen the light of day.

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.

 

More approvals and more novel drugs

Last year there were a total of 103 new drug approvals granted in US and EU together that meet our selection criteria (i). Of these new products, 15 were approved only in the EU, 52 only in the US, and 36 were granted approval in both regions. It’s a large improvement compared to last year’s figures that showed 19 only in EU, 19 only in US and 36 in both regions, with a total 74 new approvals. In addition, 56 of the new approvals in 2017 were classified as novel drugs (ii). Our data show that the trend to apply for approval in the US prior to registration in the EU is, as usual, still a regular practice.

In the US expedited drug development and nonstandard review approval pathways are the new normal. In 2017 special approval and designation procedures like Fast Track, Breakthrough (BTD), Accelerated Approval and Priority Review was used for 37 of the new approvals, in many cases more than one of these approval pathway designations was granted per product. FDA has a higher rate of granting special approval status compared to EMA, 37 vs 10. One can only conclude that the policy groundwork laid by FDA in the past years to speed up drug approvals with the introduction of shorter nonstandard approval pathways has a clear overall effect on shortening the mean approval timelines.

Ground Breaking Oncology approval and the rise of CAR-T therapies

It’s been an exciting year for oncology with a total of 27 new approvals, so far 12 of these are only approved in the US and one of them were rejected by the EMA in 2008 (Mylotarg, gemtuzumab ozogamicin), however EMA now granted approval during 2018. A ground breaking approval was granted in the US where FDA (CDER) approved Keytruda (pembrolizumab) by Merck & Co Inc as the first drug ever where a biomarker (PD-1 (programmed death receptor-1) defines the indication (iii). The scientific rationale underpinning the Keytruda approval has effectively created a single therapeutic approach for patients with different tumour types, allowing extrapolation of the observed treatment effect to diverse tumours. The approval is likely to have implications for how the drug development process is pursued in the future, in oncology, but most likely also for other therapeutic areas as science progress.

Furthermore, also in oncology, the two first chimeric antigen receptor T-cell (CAR-T) therapies have been approved by FDAs CBER unit, its Novartis Kymriah (tisagenlecleucel, for the treatment of B-cell acute lymphoblastic leukemia) and Gilead’s Yescarta (axicabtagene ciloleucel, for the treatment of relapsed or refractory large B-cell lymphoma). Both drugs are currently under assessment in EU with Kymriah being granted an accelerated assessment by CHMP.

The Approval of a Pill with a Digital Sensor

Other noteworthy approvals from the US includes Abilify MyCite, the first pill with a sensor that digitally tracks if patients have ingested their medication (aripiprazole, for the treatment of schizophrenia, acute treatment of manic and mixed episodes associated with bipolar disorder and as an add-on treatment for depression) (iv).

Noteworthy Orphan Approvals

Several important ultra-orphan medicines have been approved in the US and EU, Mepsevii (vestronidase alfa-vjbk, for the treatment of the inherited metabolic condition mucopolysaccharidosis type VII, also known as Sly syndrome, approved in the US only) and Brineuria (cerliponase alfa, an enzyme replacement therapy for the treatment of Batten’s Disease, approved in both regions). With more treatments for orphan diseases hitting the market the debate on the pricing of these drugs intensifies: Drug developers are increasingly meeting the treatment demand from the patients and physicians but are the payers willing to pay the price? Drug developers will benefit from being prepared early on to develop strategies to ensure patient access to and affordability of their orphan agents.

The Birth of EU Public Hearings

2017 is also the birth year of public hearings at EMA. The EU Pharmacovigilance legislation enabled the Pharmacovigilance Risk Assessment Committee (PRAC) to hold public hearings during certain safety reviews of medicines allowing the committee and EMA to engage with citizens in the EU. Unlike the US, where public hearings for new drugs approvals has been going on for years, the system in EU focuses on post approval hearings in the context of urgent safety procedures / referrals and public hearings for new drug approvals is out of scope for now.

In the US public hearings of new drug approvals and how to deal with them can be an important part of drug developers planning for success. Drug developers aiming for US approval should consider building awareness of public hearings into their planning and ongoing relations with medical societies, patient organisations, physicians and other healthcare professionals. The perspectives from these groups can provide an important context for the safety and efficacy data submitted by drug developers and have additions to the severity and impact of a condition and the limitations of current standards of care. The Sarepta Exondys 51 approval in 2016 (for the treatment of Duchenne muscular dystrophy, only approved in the US, a review decision from EMA is expected during 2018) is an excellent example of a public hearing playing an important role in the approval process of a new drug. The approval showed the US Advisory Committees receptiveness to public perspectives as they evaluate the benefit-risk of new drug under review.

The Era of New Designation Pathways Continues

It’s also been an exciting year for drug developers in advanced medicines (e.g. ATMP in EU and Regenerative Medicines in US). FDA has during 2017 launched its new designation pathway Regenerative Medicine Advanced Therapy (RMAT) to further enable the development of these drugs. RMAT may be considered as analogous to BTD for regenerative medicines, with some additional advantages in comparison: it does not require evidence to indicate that the drug may offer a substantial improvement over available therapies. The RMAT designation gives drug developers access to increased meeting opportunities in a manner comparable to BTD therapies.

In EU, special designation pathways and approval procedures is not as common as in the US. In March 2016, EMA launched PRIME (PRIority MEdicines), the EU counterpart to FDAs BTD. Last year the NDA Group published data showing that most products approved for PRIME was from companies based in US and most of the companies also already had BTD (v).

It is too early to tell if RMAT and PRIME will add to the strategies used by some companies that see cumulative advantages and/or benefits from obtaining multiple designation pathways, a phenomenon used mainly in the US and known as ‘layering,’ or ‘stacking’ of special designations with the intent to increase overall product value.

Continued Negative Trend of First-in-class Medicine Approvals

The approvals for targeted novel, first-in-class mechanism of action drugs continue to decrease, a trend that has been going on for years. Is it a sign of drug developer’s increasingly competitive nature around targets that “work” in specific diseases? If this is the case, drug developers can expect the competition on pricing amongst innovator products to increase and become the norm from the beginning. Historically, price competition has been occurring later on during the life cycle. In an environment where it becomes increasingly important to distinguish oneself, drug developers should focus on creative clinical strategies for differentiation. This could include co-development of biomarkers and tests to target sub-populations, companion diagnostics and innovative designs for dosing and patient follow-up.

Small and Medium Sized Pharma Dominate

For the first time since we started mapping the new drug approvals small and medium sized pharma have surpassed big pharma (vi) – quite an achievement! In total small and medium sized pharma contributed with 51% of the new drug approvals, to be compared to 49% from big pharma.

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 2017.
To read the statistics of new drug product approvals from last year click here.


Data collection and disclaimer

(i) The data was gathered from the EMA and FDA official websites, as reported on the FDA and the EMA official websites on January 2018, The data collected contains drug approvals for new active substances (chemical, biological, biotechnology or radiopharmaceutical substance), new molecular entity, new biological entity, new drug combination, biosimilars, new active ingredient and vaccines, excluding only generic and duplicate applications from the data. As it is challenging to pull together data from two regions with different classification and reporting styles some general inclusion and exclusion criteria to create consistent indicators of the yearly trends in the EU vs the US has been applied. The above article provides an overview of the key findings and an analysis of what the data means for the industry. The data is also visually represented in an infographic. As experience tells us, the final number of approvals reported normally fluctuates for some time after the end of the year, as the Agencies go through their house keeping processes. There could therefore be some slight changes to the findings outlined in this report before the data is completely finalised. The PRIME analysis was based on publically available data from the EMA website and by mapping publically disclosed BTD.

(ii) FDA Novel Drug Approvals for 2017
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
https://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/ucm547553.htm

(iii) First FDA Approval Agnostic of Cancer Site – When a Biomarker Defines the Indication. Lemery S, Keegan P, Pazdur R. N Engl J Med. 2017 Oct 12; 377(15):1409-1412

(iv) FDA approves pill with sensor that digitally tracks if patients have ingested their medication
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm584933.htm

(v) PRIME time for early designation pathways in Europe, T Johansson, Pharmafocus June edition 2017

(vi) The list of the top 50 pharma companies in 2017 was obtained from: EvaluatePharma 2017 Evaluate Ltd www.evaluate.com


 

 

Join us on a 3 day Course, learn about the Development of Biological Drugs

On the 28th – 30th May, NDA will contribute to a 3 day Course in the Development of Biological Drugs in Stockholm and/or distance, in conjunction with Sweden Bio and Swedish Academy of Pharmaceutical Sciences.

Olga Björklund, Senior Consultant, NDA Group is on the programme committee and has been invited to give the opening presentation, Paul Chamberlain, Immunogenicity specialist, NDA Advisory Board will be presenting on Immunogenicity, Ira Palminger Hallen, Senior Consultant will cover Biosimilars – preclinical and clinical aspects, and Terese Johansson Regulatory Affairs Consultant, will give an overview on Advance Therapy Medicinal Products (ATMPs).

The three-day course will provide insights into all components that should be addressed to effectively develop new biologics, from discovery to product launch.

Who should attend?

The course is aimed for those involved in drug development of biologics (eg in small and medium-sized companies), and need a comprehensive picture of the complex development chain.

Distance course

The course will not only be offered as a traditional course but also as a distance course. Those who choose to attend online will have access to all streamed lectures for 1 month.

Click here to find out more about the event and to register.

 

 

Meet NDA at DIA Europe 2018

 

We will be attending in full force during this year’s DIA Europe Meeting 17- 19 April, in Basel, Switzerland.

If you are planning to be there, we would really like to meet up with you!

You can find us throughout the event at booths: 72 & 73 where we have a range of experts covering the full regulatory affairs spectrum – from preclinical and clinical to pharmacovigilance and health technology assessment – all of whom are looking forward to meeting you:

  • Professor Beatriz Silva Lima, Non clinical Expert,
  • Dr Thomas Lönngren, Former head of the EMA and strategic advisor to NDA Group
  • Professor Steffen Thirstrup, Clinical development and regulatory strategies Expert
  • Dr Markku Toivonen, Clinical development and clinical strategies Expert
  • Shelley Gandhi, Ex MHRA regulator, Pharmacovigilance & Drug safety Expert

In addition, there will be many of NDA’s expert consultants attending the event and happy to help.

We definitely recommend booking a meeting in advance to ensure availability, but do feel free to pop by booths 72 & 73.

If you would like to arrange a meeting, please contact my colleague Anna Perrin who will be happy to assist: Phone +44 (0) 1372 860 610 or Email anna.perrin@ndareg.com


Sessions

We will also have some of our team presenting on interesting industry topics during the event. Please look out for them:

 

Shelley Gandhi – Strategic Advisor, Pharmacovigilance & Drug Safety
Dr Bill Richardson – Medical Assessor, Pharmacovigilance & Risk Management Expert
(Pre-Conference Short Course)
Short Course 3 | Mon, 16th April – 14:00-17:30
Moving from Risk Management to Benefit-risk Management-Embedding Pharmacovigilance Principles into the product life cycle


Prof. Beatriz Silva Lima – Non clinical Expert
DIAlogue 2 – Session 1100 | Tues, 17th April – 14:00 -15:30
The New EMA first-in-human (FIH) guideline Part1: Non – Clinical aspects

 

 


Dr Brian Edwards – Principal Consultant, Pharmacovigilance & Drug Safety
Session 0502 | Wed, 18th April – 14:00-15:15
Innovative approaches to safety information
A proposal for a new systems-based approach to medication errors

 


Shelley Gandhi – Strategic Advisor, Pharmacovigilance & Drug Safety
Session 0504 | Thurs, 19th April – 08:30-10:00
Five years on – pharmacovigilance legislation Delivers on long-promised elements

 


Click here to download the Preliminary Programme.


 

Meet us at DIA Europe 2018

 

To book a meeting contact Anna Perrin: anna.perrin@ndareg.com or visit us at booths 72 & 73.
We look forward to hearing back from you about a meeting and to seeing you at the event!

 


 

Advanced Workshop in QPPV Toolbox – Your Key to Success

Shelley Gandhi to conduct an Advanced Workshop in QPPV Toolbox – Your Key to Success – in conjunction with DIA Learning

Shelley Gandhi, Strategic Advisor Pharmacovigilance & Drug Safety NDA Group, has been invited to lead the Advanced Workshop in QPPV Toolbox on the 12-13 March 2018 at Adina Apartment Hotel Berlin Checkpoint Charlie, Berlin, Germany.

 


The workshop is designed to include small group interaction and discussions and is based on suggestions from the QPPVs themselves. The workshop will allow you to be more efficient in solving the problems in your daily business, learn the right thinking processes to land at good results and hear from solutions from other in similar situations.

LEARNING OBJECTIVES
At the conclusion of this course, participants will be able to:

• Master the obligations of marketing authorization holder and QPPV – your
responsibilities
• Prepare and go through the audits and inspections without major issues
• Navigate the changes in the QPPV role in a global commercial environment
• Achieve oversight of the PV system
• Set up a complete system: a QPPV Backup and delegating PV activities

KEY TOPICS
• PSMF oversight
• Quality management
• Vendor management
• Delivering a successful inspection
• QPPV in the global environment – European and international considerations

WHO WILL ATTEND
This workshop is intended for QPPVs who are already established in their role and would like to
improve their daily practice.

Click here to find out more

Common failures in drug development

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.

 

 

Dr. Lönngren presents on hot topics at ARCS Executive Round Table

Dr. Thomas Lönngren, Strategic Advisor to NDA Group and former Executive Director of the European Medicines Agency (EMA), has been invited to present at the ARCS Executive Round Table on January 31st at Atlantis Dining, North Ryde NSW, Australia.

Dr. Lönngren will address the following hot topics:

  • Evidence for regulatory approval and Market access in EU and US – Expedited pathways
  • Use of RWE in decision making
  • From cost effectiveness to budget impact and affordability
  • Brexit and its effect on the pharmaceutical sector in EU (and worldwide)
  • How will this affect Pharmaceutical/ Biotech companies in Australia

This roundtable is for senior managers (10+ years’ experience) who are keen to engage with their peers, exchange views and discuss current challenges that the sector is facing in an open and constructive dialogue. The meeting will be of interest to both regulatory affairs and reimbursement professionals.

To read more about the event click here.

 

 

Meet us at the Phacilitate Cell & Gene Therapy World 2018

Dr Paula Salmikangas, NDA Director for Biopharmaceuticals & ATMPs, will be moderating a 1-hour ‘Working Lunch’ Panel Discussion on “Safety aspects of genetically modified cells” at 1.00-2.00pm on Tuesday, January 23rd 2018 at the Phacilitate Cell & Gene Therapy World conference in Miami.

Paula was previously Chair of the Committee for Advanced Therapies at EMA and has a longstanding experience on ATMPs as a CAT Rapporteur and CMC expert.

The conference brings together industry leaders from the pharma and biotech communities to deliver the ultimate in strategic knowledge exchange and networking.
Reflecting the growing enthusiasm around cell and gene therapy by small and large companies, investors and regulators.

To read more about the event click here.

 


Free Webinar – Development of Cell based Cancer Immunotherapy products

Optimising the quality of cell-based immunotherapy products is critical to maximising safety and efficacy of novel cancer treatments. Employing the right manufacturing approach is necessary to ensure your product’s long term success.

Join us on 8th February to hear NDAs Dr Paula Salmikangas, Director of Biopharmaceuticals and ATMPs, discuss:

  • How the CMC aspects impact safety and efficacy of cell-based cancer immunotherapy products
  • The importance of early product characterization
  • Cellular markers and correlation with responders
  • Critical quality aspects to ensure safety

She will also provide an overview of recent updates in the EU regulatory system for ATMPs.

Dr Salmikangas will be available to answer questions at the end of the session.

 

Click here to book your place today.

 


Dr. Salmikangas 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. Dr. Salmikangas 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.