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

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!

 

We are expanding our geographic reach by opening an office in San Fransisco, California, USA

NDA Group has helped companies get their products to the market for more than 20 years. Over the last few years we have established offices on the US east coast in Princeton and Boston. Today we announce a new presence in San Francisco on the US west coast, to support more frequent face to face interactions and strategic regulatory support on site. With this expansion we are announcing our first west coast based Principal Consultant, Alison McGregor.


Alison McGregor joins NDA Group from her position as Global Regulatory Affairs Leader at BioMarin Pharmaceuticals Inc. After completing a PhD in neuroscience from Cambridge University in the UK, she worked in academia at Carleton University in Ottawa, Canada and at the Karolinska Institute in Stockholm prior to moving into regulatory at the UK MHRA and then to AstraZeneca. Alison is an experienced global regulatory affairs professional with 20+ years in the pharmaceutical sector both with industry and regulatory agencies across all therapy areas and has extensive experience working with teams globally across sites and regions.

Alison will be based in San Francisco where she will be supporting clients with advice on their US and EU drug development programs in order to get their products into both markets faster.

Johan Strömquist, CEO of NDA Group commented:

“The US expansion is our main focus in 2018 and we are planning on expanding our support for FDA and EMA submissions, milestone meetings and de risking early stage data packages. Establishing ourselves on the US west coast is an important step to get closer to our clients in one of the most vibrant life science areas in the world. Bringing Alison on-board is particularly important as her experience represents the ideal we share across NDA – that of bringing diverse regulatory requirements together to create the best possible development programs as efficiently as is possible.”

Laurie Smaldone-Alsup, MD, COO / CSO of NDA Group commented:

“As our business and our client base in the US are growing at an incredible rate it is important for us and for our clients that we have people on the ground who are sensitive to their needs. Alison will provide our many clients on the west coast with direct access, not only to her own extensive experience of supporting development programs, but also to the aggregate expertise of NDA Group.”

NDA Group celebrates 20 years of championing treatments for rare diseases

Stockholm, 28 February 2018 – Leading drug development consultancy acknowledges the extraordinary contributions of patients and patient organisations around the world on global Rare Disease Day.

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

Over the years NDA has been involved in the development of more than 50 products for individuals with rare diseases and several of the key breakthroughs have been supported by the company.

Johan Strömquist, CEO of NDA Group:
“At NDA we take great pride in helping companies address large unmet medical need. The need is most significant when it comes to rare diseases.”

Strömquist continues:
“Through the experience and knowledge of our regulatory scientists and clinical experts from agencies and the industry we feel privileged to have made a significant difference for thousands of patients living with rare diseases previously considered impossible to treat.”

The expansion and increased involvement of patients in the drug development process is one of the key factors that have stimulated the development in the rare disease space over the last few years.

Dr. Markku Toivonen, MD, Scientific Director at NDA and former Chairman of EMA’s Scientific Advice Working party and former CHMP member comments:
“The development of patient advocacy and the involvement of patients in the development and regulatory process has truly been game changing. At many levels the experience of the patient brings perspectives that no amount of objective, scientific scrutiny can bring to the process. At NDA we take great pride in helping to empower patient advocates through training in regulatory science and medical research with Eurordis, the European Organisation for Rare Diseases. ”

At NDA, the process to support the development of novel treatments for rare disease continues. Dr. Laurie Smaldone Alsup, MD, CSO and COO of NDA commented:
“We are driven by our passion and commitment to bringing new treatments to people in need. The rare disease landscape is still a vast area of unmet medical need with few options for many serious and life threatening conditions. We continue to provide leadership in solutions for diseases without regulatory precedent staying at the forefront of novel product development by guiding industry through the regulatory hurdles. Importantly we help to find and listen to the patient voice that has been a crucial contributor to the understanding of these rare conditions.”

Smaldone Alsup continues:
“I believe we are still at the beginning of addressing rare diseases. With new scientific and technical advances every day we will be turning a corner where many more novel treatments will be discovered to address a range of difficult to treat disorders. We are poised and ready to support these innovators and help enable treatments to reach people in need around the world.”


NDA media contact: anna.perrin@ndareg.com

About NDA
NDA is an independent consulting group with offices in Stockholm, London, Zürich, Munich, Princeton, Boston and San Francisco. Its mission is to ensure that good medicines reach patients without unnecessary delay. It accomplishes this by providing the pharmaceutical industry with a comprehensive range of professional drug development services focusing on regulatory affairs, health technology assessment, pharmacovigilance and quality assurance. NDA’s team of more than 150 full time professionals is comprised of more than 25% ex-regulators from major EU Agencies, and a specialist Advisory Board consisting of Europe’s leading regulatory and HTA experts. The NDA Advisory Board provides scientific advice to pharmaceutical companies supporting them in gaining fast and constructive assessment of applications from regulatory agencies and HTA bodies. www.ndareg.com

Strategic alliance to support efficient, ethical practices in biopharma industry

Clinton Allen CEO Ethiprax LLC
Fabiana Lacerca-Allen President Ethiprax LLC
Johan Strömquist CEO NDA Group

 

New Collaboration offers broad range of Compliance-and Risk Management Strategies in Europe and US to Life Science Industry


Hayward, CA and Stockholm, Sweden (07-12-2017) – Two leading drug development and regulatory consultancies announced today that they have formed a new strategic collaboration to help life science companies navigate an increasingly complex global environment. Ethiprax Associates LLC – a leading Compliance and Ethics solutions consultancy based in the US – and NDA Group – a world leading integrated regulatory/drug development consultancy are now collaborating to provide the life science industry with truly integrated, trans-Atlantic Compliance-and Risk Management strategies.

Fabiana Lacerca-Allen, President of Ethiprax LLC commented:
“As we are intimately familiar with compliance requirement both in the US and Europe, we are able to help clients develop and maintain effective cultures of compliance. Too often companies hire one firm for US and another for EU and fail to implement a compliance culture across the entire organization. This collaboration offers a single partner who can provide global solutions.”

Johan Strömquist, CEO of NDA Group commented:
“We recognize that life science companies operate in different regulatory environments but fundamentally they all need to implement global best practices to maximize their opportunities for success. This is where we can add tremendous value. Doing the right things right doesn’t just feel good – it is the optimal way to create value.”

Strömquist continues:
“Together with Ethiprax we extend the reach of our services and our geographic foot print on the US west coast – much in line with our growth strategy. This continues to bring the experience biotechs and pharmaceutical companies need closer to where they are.”

The NDA Group and Ethiprax collaboration will focus primarily on the following:

  • Assess regulatory compliance readiness in early stage programs.
  • Provide strategic assessments of emerging compliance needs, review existing programs and provide advice regarding best practices to optimize critical business processes.
  • Align diverse teams to support manufacturing, clinical and product safety reporting requirements to connect the dots in compliance program development and implementation.
  • Offer compliance assurance program design, policies, procedures and program execution.

 


About NDA Group
NDA is an independent consulting group with offices in Stockholm, London, Zürich, Munich, Princeton, Boston and San Francisco. Its mission is to ensure that good medicines reach patients without unnecessary delay. It accomplishes this by providing the pharmaceutical industry with a comprehensive range of professional drug development services focusing on regulatory affairs, health technology assessment, pharmacovigilance and quality assurance. NDA’s team of more than 150 full time professionals is comprised of more than 25% ex-regulators from major EU Agencies, and a specialist Advisory Board consisting of Europe’s leading regulatory and HTA experts. The NDA Advisory Board provides scientific advice to pharmaceutical companies supporting them in gaining fast and constructive assessment of applications from regulatory agencies and HTA bodies.

NDA supported over 40% of the new medicinal products that were approved in the EU over the past four years. http://www.ndareg.com/

About Ethiprax
Ethiprax is a specialized consultancy operating out of Hayward CA in the USA. Ethiprax was formed to help companies manage compliance and ethics risk. Drawing upon extensive experience related to healthcare-related organizations, Ethiprax helps clients develop and maintain effective cultures of compliance. Ethiprax brings industry best practices to the life sciences industry, ensuring that compliance requirements are addressed. http://ethiprax.com/

NDA Media Contact
Anna Perrin anna.perrin@ndareg.com

Ethiprax Media Contact
Allen Clinton clinton.allen@ethiprax.com

Meet us at the DIA and BIO International conventions

DIABIO1

 

 

 

This June, NDA will be attending the DIA USA and BIO convention with a strong line-up of world-leading speakers and experts.

The 53rd DIA Annual Meeting 2017 is being held on June 18-22, at McCormick Place, Chicago. Whilst, the BIO International Convention is on June 19-22, at San Diego Convention Center, San Diego.

If you are planning to attend this year’s DIA or BIO, we would really like to meet up with you. If you’d like to arrange a meeting with us, please email anna.perrin@ndareg.com.

Hear the experts

NDA’s experts will present on a number of critical drug development related topics at both the DIA and the BIO meeting.

NDA at DIA USA

Neelu AgarwalPete_TaftNeelu Agrawal, Principal, PharmApprove

Pete Taft, Founder and Chief Executive Officer, Taft and Partners/PharmApprove

 

Tutorial:  Preparing for a US FDA Advisory Committee Meeting
Track: 5 Regulatory Affairs #30
Day: Sunday June 18
Time: 13:00 – 16:30

Shelley_GandhiShelley Gandhi, Pharmacovigilance and Drug safety Expert expert, NDA Group
Title: The Good Pharmacovigilance Practices in the EU: Global Applications
Track: 6 Safety & PV #43
Day: Sunday June 18
Time: 09:00 – 17:00

Title:  Future Regulatory Goals of the Periodic Benefit-Risk Evaluation Reports (PBRER ): Are They Being Achieved?
Track: 6 Safety & PV #280 Developing Pharmacovigilance Policy based on Impact
Day: Tuesday June 20
Time: 16:00 – 17:15

Lesley_WiseLesley Wise, Principal Consultant, Pharmacoepidemiology, NDA Group
Title: Incorporating Patient Preferences in Benefit-Risk Assessments: Interpreting ICH M4E (R2)
Track: 4 Patient Engagement, Forum #251 Defining the Science of Patient Input to Enhance Drug Development & Approval: Regulatory
Day: Tuesday June 20
Time: 14:00 – 15:15

 


NDA at BIO USA

Lisa-PelusoMarkku ToivonenLisa Peluso, Director Coaching and Client Engagement, PharmApprove

Markku Toivonen, Scientific Director, NDA Group

 

Title: Bridging the Atlantic: How to Involve Patients in Orphan Drug Development and Approval Processes Given Differences between FDA and EMA
Day: Wednesday June 21
Time: 15:00 – 16:00 Room 3

 


To find out more or schedule an appointment with members of the NDA team, please email anna.perrin@ndareg.com

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

 

About NDA
NDA Group is a leading global drug development consultancy providing small as well as large, multi-national pharmaceutical companies with strategic advice and operational support to get good medicines to market and keep them there. Based in Boston, London, Munich, New Jersey, Stockholm and Zurich, NDA offers a range of professional drug development consulting services that spans from early development phase to lifecycle management of a medicinal product. These services span Development Strategy, Translational Science, Procedure & Submission Management, High-Stakes Meetings and Process Design & Optimisation. Clients are supported by a team of over 150 regulatory affairs, health technology assessment, pharmacovigilance, quality assurance and strategic communications professionals. Backing all major NDA projects is the unique NDA Advisory Board comprising industry experts, many of whom are ex- European Agency and FDA staff. Learn more at www.ndareg.com and follow them at www.linkedin.com/company/nda-group-ab.

About PharmApprove
The NDA PharmApprove team comprises leading strategic, regulatory, and scientific communications professionals. PharmApprove focuses on strategic and tactical support to companies facing high-profile, high-stakes events and engagements anywhere along the road to approval and commercialization – including FDA Advisory Committee meetings. PharmApprove helps clients win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs. Learn more at https://www.pharmapprove.com/ and follow them socially at twitter.com/pharmapprove.

NDA Group Releases Data Comparing FDA and EMA Ahead of Annual DIA EuroMeeting in Glasgow

Drop in US drug approvals but no similar trend is seen in EU

GLASGOW, Scotland, March 28, 2017 /PRNewswire/ — NDA Group announced findings from their fourth annual comparison of drug approvals in Europe and the United States, ahead of this year’s DIA EuroMeeting in Glasgow. This year’s Status of New Drug Approvals report emphasises the need for a streamlined global development and commercialisation process across the world’s two biggest markets.

The report – based on preliminary research figures from the EMA and FDA websites in January 2017 – found that there has been a drop in US approvals but not in EU. For 2016 there were 74 new drug approvals granted in the US and EU. Of these new products, 19 were approved only in the EU, 19 only in the US, and 36 were granted in both regions. However 17 drugs that were approved in the EU in 2016 had received prior approval in 2015 or earlier in the US, while only six products registered in the US in 2016 were previously approved in the EU.

Johan Strömquist, CEO, NDA Group “Understanding the evolving regulatory landscape and requirements is a key concern for the drug developing industry, as is it for us at NDA. It is intriguing to see how expedited pathways shape this year’s statistics just like it did last year, but with a very different outcome. It’s also interesting to see the continued rise of smaller companies in the percentage of products taken to market.”

“Our analysis for 2016 shows that NDA maintains an exceptional position in supporting new drug product approvals in Europe. During the last four years NDA supported over 40% of the new drugs approved with a broad range of services. I’m also excited to see the increase of products going through the FDA that received NDA’s support. NDA supported over 20% of new drugs that achieved approval by the FDA during 2016.”

Terese Johansson, PhD, NDA’s consultant behind the research commented: “The findings show that FDA has had a significant drop in drug approvals but continues to grant more expedited and nonstandard review approval status than the EMA. The drop is not as pronounced in the EU but is likely to be more prominent in 2017. Expedited drug development and nonstandard review approval pathways are the new norm in the US, but in the EU special approval procedures are not as common. The US situation could be explained by the increased use of the shorter nonstandard approval pathways since there has also been a significant increase in complete response letter (CRL). During 2016 FDA issued 14 CRLs, compared to just two in 2015.”

“The report also highlights the continued trend that many companies first seek approval in the US. Both the EU and US show increases in drug approvals from small and medium sized pharma but big pharma still dominates the drug approval statistics. The busiest therapeutic area was oncology.”

NDA-Infographic-2016

The pooled statistics showed that of the new drug approvals, 35 were classified as novel drugs (e.g. NAS, NME or BLA), nine were approved only in the EU, nine only in the US and 17 in both regions. For the EMA, the number represents the fewest NASs approved since 2011, while the FDA has not approved this few NMEs/BLAs since 2010. Big pharma represented 53% of the new drug approvals in 2016 vs 47% from small and medium sized pharma. For big pharma this is a decrease compared to previous years. Of all new products that received marketing approval in 2016, 30 products underwent special approval procedures like Conditional, Fast Track, Breakthrough, Accelerated Approval and Priority Review, 18 in the US, seven in EU and five in. In many cases more than one of these pathways was granted per product.

NDA Strategic Advisor and former Chief Exec of the EMA, Dr. Thomas Lönngren, as well as the company’s Scientific Director, Dr. Markku Toivonen and the Director of NDA’s Regulatory Advisory Board, Prof. Steffen Thirstrup will be present at DIA EuroMeeting together with a line-up of experts, and available to discuss these findings.

NDA staff can be found at booth C 10, 11 of the Congress Center and in the following presentations:

  • Shelley Gandhi & Bill Richardson – Ex MHRA Regulators
    (Pre-Conference Short Course) Short Course 4 | Wed, 29th March – 09:00-12:30
    Moving from Risk Management to Benefit / Risk Management –  Embedding Pharmacovigilance Principles into the product life cycle
  • Dr Mira Pavlovic –  HTA Expert, Session Chair
    Session 0101 | Wed, 29th March – 16:30-18:00
    Global dossier for clinical development
  • Prof. Steffen Thirstrup – Director NDA Regulatory Advisory Board – Session Chair
    Session 0302 | Thurs, 30th March – 11:00-12:30
    EU clinical trial regulation and its implications
  • Prof. Beatriz Silva Lima – Non clinical Expert
    Session 1002 | Thurs, 30th March – 11:00-12:30
    Optimising the development of paediatric medicines
  • Dr Markku Toivonen – Scientific Director & Medical Advisor – Session Chair
    Paul Chamberlain – Biopharmaceutical Development & Immunology Specialist – Session Speaker
    Session 0303 | Thurs, 30th March – 14:00-15:30
    Immunogenicity assessment-risked-based approaches

For more about NDA’s involvement with the 2017 DIA EuroMeeting, visit https://www.ndareg.com/meet-nda-at-the-dia-euromeeting-2017/.

To explore the full Status of New Drug Approvals for 2016 report, visit https://www.ndareg.com/wp-content/uploads/2017/03/NDA-Infographic-2016.pdf.

About NDA
NDA Group is a leading global drug development consultancy providing small as well as large, multi-national pharmaceutical companies with strategic advice and operational support to get good medicines to market and keep them there. Based in Boston, London, Munich, New Jersey, Stockholm and Zurich, NDA offers a range of professional drug development consulting services that spans from early development phase to lifecycle management of a medicinal product. These services span Development Strategy, Translational Science, Procedure & Submission Management, High-Stakes Meetings and Process Design & Optimisation. Clients are supported by a team of over 150 regulatory affairs, health technology assessment, pharmacovigilance, quality assurance and strategic communications professionals. Backing all major NDA projects is the unique NDA Advisory Board comprising industry experts, many of whom are ex- European Agency and FDA staff. Learn more at www.ndareg.com and follow them at www.linkedin.com/company/nda-group-ab.

About PharmApprove
The NDA PharmApprove team comprises leading strategic, regulatory, and scientific communications professionals. PharmApprove focuses on strategic and tactical support to companies facing high-profile, high-stakes events and engagements anywhere along the road to approval and commercialization – including FDA Advisory Committee meetings. PharmApprove helps clients win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs. Learn more at https://www.pharmapprove.com/ and follow them socially at twitter.com/pharmapprove.

Sarepta Receives Historic, Accelerated Approval from FDA

PharmApprove congratulates Sarepta Therapeutics on receiving accelerated approval for the first drug to treat Duchenne Muscular Dystrophy (DMD) in the US.  EXONDYS 51 has been approved for patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

This is an historic moment for Duchenne boys, their families and the larger rare disease community.

PharmApprove Hosts Tutorial at 2016 DIA Annual Meeting

How to Prepare for an FDA Advisory Committee Meeting?
PharmApprove Hosts Tutorial at 2016 DIA Annual Meeting

The leading consultancy will share best practices and “must avoids” in advisory committee preparation

Lawrenceville, NJ (June 21, 2016) – PharmApprove – a member of the NDA Group and the leading strategic, regulatory, and scientific communications consultancy in the U.S. – will lead a tutorial on FDA advisory committee preparation at the 2016 DIA Annual Meeting. The session will take place on Sunday, June 26th at 1:00 p.m. at the Philadelphia Convention Center, during DIA’s 52nd Annual Meeting. PharmApprove has more than 15 years of experience preparing companies for advisory committee meetings, and boasts an over 85 percent success rate.

“We ensure that our clients are 100 percent prepared for all scenarios when they appear before the FDA,” explained Lisa Peluso, director of coaching and client engagement for PharmApprove. “We understand what it takes to make a winning case before an advisory committee, and how to avoid potential pitfalls. Our expert support and coaching allows drug and device developers to clearly articulate the case for approval, and then focus on what’s really important – bringing new cures and treatments to patients.”

Peluso will lead the tutorial, which was developed with support from PharmApprove Founder and Executive Communications Strategist, Pete Taft. The tutorial helps corporate executives and other professionals across all R&D disciplines to effectively prepare for successful advisory committee meetings. PharmApprove has led thousands of training workshops and coaching sessions for regulatory, Medical Science Liaison, business development and payer negotiation teams at pharmaceutical companies around the world.

“When PharmApprove merged with NDA Group a few months ago, it heralded a truly exciting time for the company, with expanded global reach, expertise and talent. But that’s only one part of the equation,” said Laurie Smaldone MD, COO/CSO of NDA Group. “Our experienced coaching team and unsurpassed advisory committee preparation practices remain our strongest assets, allowing us to continue offering the best FDA approval consulting in the business.”

Visit https://dia201652ndannualmeeting.sched.org/event/61Nm for registration and scheduling information about the upcoming tutorial. Follow the social conversation online with the hashtag #DIA2016.

 

About PharmApprove

PharmApprove is the leading strategic, regulatory, and scientific communications consultancy to the pharmaceutical and biotech industries. The firm offers both strategic and tactical support to companies facing high-profile, high stakes events and engagements anywhere along the road to approval and commercialization – including FDA Advisory Committee meetings. PharmApprove helps clients win health authority approvals, deliver compelling regulatory communications, and make persuasive pharmacoeconomic arguments to payers and HTAs. PharmApprove merged with NDA in March of 2016. Learn more at pharmapprove.com or ndareg.com/pharmapprove and follow them socially at twitter.com/pharmapprove and facebook.com/pharmapprove.

About NDA

NDA Group is a leading global drug development consultancy providing small as well as large, multi-national pharmaceutical companies with strategic advice and operational support to get good medicines to market and keep them there. Based in Boston, London, Munich, New Jersey, Stockholm and Zurich, NDA offers a range of professional drug development consulting services that spans from early development phase to lifecycle management of a medicinal product. These services incorporate regulatory affairs, health technology assessment, pharmacovigilance and quality assurance. Clients are supported by a team of over 100 consultants and a unique Advisory Board comprising industry experts, many of whom are ex- European Agency and FDA staff. For more information, visit ndareg.com.

Tutorial at DIA Annual Meeting

We’re at DIA2016!  PharmApprove, as the leader in FDA Advisory Committee Preparation, will once again be teaching an informative, engaging and interactive class on “Preparing for a FDA Advisory Committee Meeting.” Can you identify the critical factors in preparing for a successful advisory committee meeting?  We’ll help you recognize and evaluate those factors that are most applicable to your team, and successfully design the most effective preparation for your team. Join us on Sunday, 26 June at 1:00.  Register here:   Click here