A New Oncology Era

Dr Terese Johansson, Regulatory Consultant, NDA Group has written a new article on tissue-agnostic drug development: A New Oncology Era 

Read about how recent developments in tumourigenesis genomics have paved the way for a molecular marker defining a disease that spans multiple histology-based tumours in a tissue-agnostic manner, in an article written by Terese Johansson, published in European Biopharmaceutical Review October 2018 pages 32-37.

To learn more please click on the brochure below and flick to pages 32-37.

Healthcare White Paper published

On Thursday 4th October, at the prestigious Royal Society of Medicine in London, the Chartered Institute of Ergonomics & Human Factors (CIEHF), launched their much-awaited White Paper setting out CIEHF’s vision for the integration of Human Factors in Health & Social Care.

The launch event was a great opportunity for individuals working in Health and Social Care, both clinical and non-clinical, to understand how human factors expertise can help and benefit patients, staff and their organisations.

Dr Brian Edwards, Principal Consultant, Pharmacovigilance & Drug Safety, NDA Group gave a talk, outlining the multi-faceted work of CIEHF’s unique Pharmaceutical Sector Group and its growing influence in a number of important areas such as manufacturing, technology and device design.

Click here to find out more

The White Paper is now available as a digital download. Get your copy here.





Dr Brian Edwards, Principal Consultant, Pharmacovigilance & Drug Safety, NDA Group

How to commercialise ATMPs in the EU

NDA’s Paula Salmikangas, Director for Biopharmaceuticals and ATMPS, NDA Advisory Board and Steffen Thirstrup, Director NDA Advisory Board, have co-authored the article ‘How to commercialise ATMPs in the EU’.

Cell and gene therapy medicinal products, together with tissue engineering products (so-called advanced therapy medicinal products, ATMPs) are under active research globally. In the EU the legal and regulatory framework
has been in place for a decade.

However, the speed of scientific progress is challenging the available guidance and existing rulesets for ATMPs. This discrepancy has been noted by the European Commission (EC) and the European Medicines Agency (EMA), which, together, released an action plan for ATMPs in December 2017.

This article addresses recent findings from the EMA PRIME scheme and provides information about procedural updates and evolving guidance in the ATMP area.

The article is also featured in Regulatory Rapporteur – Vol 15, No 7/8, July/August 2018. Regulatory Rapporteur is TOPRAs respected, peer-reviewed journal and it is published 11 times per year and is free to TOPRA members.





Regulatory authorities expand their contribution to successful drug development

In recent years, the support from regulatory authorities available to pharmaceutical companies has increased significantly, with a particular focus on SMEs.

In the latest SwedenBio’s fact sheet (in Swedish), Eva Lilienberg, Service Area Lead Global Development NDA Group, details the levels of support pharmaceutical companies can expect to receive from the authorities and how to best prepare for any agency interactions.





Regulatory strategies for value maximisation of early clinical stage assets

Prepare for partnering – Optimizing value in early development

There are many important reasons why a company would choose not to develop and commercialise a product on their own. The cost of development increases exponentially, making the final stages impossible to finance for many companies. Building a commercial organisation covering vast territories can be an equally daunting task.

Companies we work with have a clear definition of what it is they want to do and often their strategy is limited to discovering and developing proofs of concepts of products in a certain region; the rest is simply not part of their focus. Whatever the reason, the goal for many companies we work with is often to develop the product to a stage where partnering or out-licensing becomes feasible.

This white paper written by Niamh Kinsella, Biologics Expert, Paul Chamberlain, Biopharmaceuticals Expert, Josi Holz, Clinical Strategy Expert and Eva Lilienberg, Service area lead Global Development, focus on how you can strengthen your partnering negotiation position, reduce the time to the clinic and to help you determine value-adding regulatory milestones by applying integrated regulatory strategies, ultimately optimising the value of your asset at the point of exit.






Niamh Kinsella
Paul Chamberlain
Josefin-Beate Holz
Eva Lilienberg








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

(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

(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



Assessing the consequences of the EMA’s relocation

The EMA is relocating to Amsterdam – but what are the consequences to the Agency’s priorities and to the industry?

In this commentary NDA’s CEO Johan Strömquist and Strategic Advisor Thomas Lönngren, formerly Executive Director at the EMA, assess the impact on the Agency in light of its business continuity plans.




Thomas Lönngren
Johan  Strömquist



When the pressure is high Prepare to Win

FDAimageKey numbers drop, but Advisory Committees hold steady in complex year for drug approvals.

Earlier this year NDA released its 2016 annual report on drug approvals in both the EU and the US. One striking finding: US approvals (NMEs) plummeted from 45 (2015) to 19 — a drop of almost 60 percent.

That dramatic number wasn’t the only thing that caught our attention though. When we drilled down into all the applications, we spotted some important trends that you can read about in this article written by Christine Chirdo – Service Area Lead: High Stakes Meetings.

You will also learn how we can support you with key activities to make your high-stakes meetings a success. Prepare to Win.











Christine Chirdo – Service Area Lead: High Stakes Meetings, NDA Group/PharmApprove



Have you ever wondered why compliance with regulations is so difficult?

What do we mean when we say ‘safety is everybody’s responsibility’? What is meant by risk-based compliance? What is the basis for an effective safety system?

The reality is that humans will make mistakes so that ultimately safety and quality is all about human performance.

To find out more read the full article that has been published in Quasar written by NDAs Dr. Brian Edwards.





Dr Brian Edwards





Dr Brian Edwards Principal Consultant, Pharmacovigilance & Drug Safety, NDA Group



Communicating with Payers

checkPartnership with Payers: Communicating and Negotiating with Impact.

Your product is barreling toward approval and you are on the cusp of bringing it to market and to the patients that need it most. Your company has put enormous effort into developing your value proposition documents, slide decks, internal FAQs and objection handler booklets.

This plethora of planning and information is disseminated to your Market Access and Medical Affairs teams across the globe.

Then what?

Read the full article written by Lisa Peluso, Director Coaching and Client Engagement, to learn more about why excellence in communications is critical when it’s time to defend the value and negotiate pricing. Prepare to win.











Lisa Peluso – Director Coaching and Client Engagement, NDA Group/PharmApprove



Novel Drug Movers and Shakers 2013-2016

Small and Medium Sized Companies in North America Lead Novel Drug Innovation

Small and medium sized companies and academia are important drivers of innovation in drug development. To investigate the profile of the organisations originating and developing novel drugs approved in the EU and US during 2013-2016 we have compiled all medicinal products containing novel substances (e.g. NAS, NME and Biologics) from the EMA and FDA websites.

For each novel drug we mapped the approval pathway, rare disease (orphan) status and post-approval licensee. The innovator(s and the development organisation(s) were mapped in accordance with the ADIS insights database. Each originator and developer was categorised as a large pharmaceutical company, a small or medium sized pharmaceutical company (SME) or an academic and/or public body.

We also mapped and analysed the geographical origin (e.g. Europe, North America or Rest of the World) of the SME or the academic and/or public body. Altogether, 178 novel drugs received approval from the EMA and/or the FDA during 2013-2016.

To read the White Paper written by Dr Terese Johansson, Regulatory Affairs Consultant NDA Group click here.








Dr Terese Johansson, Regulatory Affairs Consultant NDA Group


Maximising value in early development

Capture1Selecting product candidates with the highest probability of success and applying rigorous risk-based management in development maximises the value of biopharmaceutical products and increases speed to the clinic and registration.

Due to the high costs for product development, few small and medium sized pharmaceutical companies will take their product to the market on their own. Therefore, the goal is often to develop the product to a stage where partnering becomes feasible. However, the aim should be to add as much value as possible to the product during early development, as this will maximize the likelihood of a successful deal with a corporate partner and provide alternative exit opportunities for the company.

Read the full article written by Niamh Kinsella, Principal Consultant, VP Early Stage Development, Paul Chamberlain Biopharmaceutical and Immunogenicity Expert and Josefin-Beate Holz, Clinical Strategy Expert, to learn how you can maximise the value of your product by having an early development strategy.






Niamh Kinsella
Josefin-Beate Holz
Josefin-Beate Holz
Paul Chamberlain