Expedited Reviews: What you need to know to be Successful

A Case Study

By: Judith Plon, Principal Consultant, NDA Group

In the September issue of Pharmafocus, Frank Casty, MD, Senior Clinical Regulatory Advisor at NDA discussed “Expedited regulatory pathways and what you need to know to be successful.” This month I am following up with a case study of Bavencio (avelumab) injection, a Biological License Application (BLA) that was able to come to market quickly by utilising the benefit of multiple expedited review pathways.


Bavencio is a drug indicated to treat an aggressive neuroendocrine tumour of the skin for which there are no other treatments. With fewer than 20,000 cases per year in the US, the drug qualified for Orphan Status and, like other rare diseases, the primary review for the treatment effect was based on a small patient population, in this case less than 88 patients.1

As the sponsor had a well-planned regulatory strategy and utilised all the tools available to them during the drug development process, they were able to obtain both the Fast Track and Breakthrough Therapy Designation (BTD). Based on BTD, a preliminary advice meeting was held with the FDA to discuss an Accelerated Approval approach. The sponsor benefitted from several multi-disciplinary meetings with FDA, an important advantage of requesting and receiving this designation early in the programme.

The BLA also utilised a Rolling Review Process with the nonclinical sections of the application submitted ahead of the clinical data. Finally, the FDA granted the company a Priority Review status which means the application was reviewed in a six-month time frame. All of this was accomplished without the need for an FDA Advisory Committee Meeting!

This case study is an excellent example of how a well-planned and meticulously executed regulatory strategy can lead to a collaborative and rapid review, approval and, most importantly, availability of a new treatment for patients in an area with high unmet medical need.

At NDA, we support many companies in both the US and Europe seeking multiple expedited review procedures.

The full article was published in September 2020 in UK News – Pharmafocus.

Click to the full article
References: 

1. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761049Orig1s000TOC.cfm (Bavencio)

Remote audits – The new normal

By: Helen Kargaryani, Global Head of Quality at NDA Group.

In this whitepaper, Helen provides tips and good practices for remote audits as an alternative method to conducting traditional on-site audit.


The COVID19 pandemic is presenting unprecedented challenges to healthcare, the pharmaceutical industry’s supply chain and the ways in which companies operate “business as usual”. Quality and compliance activities have been particularly affected, areas that are crucial to ensure the long-term safety and efficacy of treatments.

Quality audits and inspections are essential aspects of the checks and balances in a pharmaceutical Quality Management System (QMS). With new guidance’s, constrained travel and limited access to buildings, suppliers, records and people, manufacturer, regulatory and quality personnel must now explore methods and techniques to evaluate quality and compliance in light of these restrictions. To meet these challenges, regulatory agencies and companies alike are adapting to the situation, relying heavily on remote mechanisms to continue delivering lifesaving medicines and products globally.

In this whitepaper, we will provide tips and good practices for remote audits as an alternative method to conducting traditional on-site audit. This paper will also cover circumstances in which remote audits may be necessary and preferable to an on-site audit. Furthermore, we will discuss potential challenges and benefits when auditing remotely and how to adapt your processes and systems to prepare your company for remote audits.

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Contact us to learn more about how our integrated team can provide advice and support into your drug development plan.

 

 

 

Integrated product development for ATMPs

By: Claes Buxfeldt and Dr Paula Salminkangas

In this article, published in the September issue of MedNous, Claes and Paula discuss why an integrated product development strategy for ATMPs is essential to meet both regulatory and HTA requirements.


Advanced Therapy Medicinal Products (ATMPs), which include cell and gene therapies and tissue engineered products, are a group of innovative products targeting diseases and conditions for which there are few, if any, effective treatments. The success of the first CD19 chimeric antigen receptor T cell (CAR T) products Kymriah and Yescarta against B-cell malignancies has raised the awareness of the high potential of ATMPs, but also shown the several challenges relating to their clinical use1. One of these challenges is the high prices asked by the manufacturers of these products, which are not always supported by national pricing and reimbursement bodies2.

In such cases, the discrepancy between a regulatory approval and a negative decision from a health technology assessment (HTA) body has raised concerns and questions from industry about how to ensure that an approved product also gets to the market and to patients. Many jurisdictions have created early access schemes and ways to communicate with regulatory and HTA bodies early on to ensure successful outcomes of both reviews3. However, the ATMP industry is facing challenges in both aspects.

The development of ATMPs has substantially increased with a focus on clinical trials in recent years. Several cell and gene therapy products have been authorised worldwide, most recently the CAR T product Tecartus from Kite Pharma Inc.4 in the US and Zolgensma for spinal muscular atrophy (SMA)5 in the EU.

Today there are more than 980 developers globally, the majority (78%) of whom are in North America and Europe.6 Over 1,000 clinical trials were underway worldwide at the end of 2019, two-thirds of which (64%) were in Phases 2 and 3. There has been a clear shift from early to late phase trials, as only four years earlier the majority of trials (> 90%) were in Phases 1 and 27. Since beginning of 2015, the overall number of ATMP clinical trials and ATMPs in Phase 3 has doubled, suggesting multiple new ATMPs will be approaching the marketing authorisation application stage in the next few years.

Today, the focus of ATMP development is heavily in gene therapy and genetically modified cells which constitute three-quarters of products in clinical trials. This is most probably due to the fast development of novel vectors and technologies, including genome editing. In addition, a lot of safety data has accumulated for certain gene therapy approaches (e.g. adeno associated virus vectors, AAV and lentivirus vectors, LVV), which reduces the regulatory burden before first clinical trials.

From an indication perspective, the majority of ATMP clinical trials (657/1066, 62%) 6 are in oncology, including leukaemia, lymphoma, and solid tumors, which may be explained by the great interest towards novel immunotherapies using genetically modified cells. In 2019, genome edited cells using the CRISPR/Cas9 approach proceeded to clinical trials both in the US and the EU8 and the first results from a trial studying an induced pluripotent stem (iPS) cell-derived product were reported in the EU 9.

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Contact us to learn more about how our integrated team can provide advice and support into your drug development plan.

 


References :

  1. Mohty M., Gautier J., Malard F., et al. CD19 chimeric antigen receptor-T cells in B-cell leukemia and lymphoma: current status and perspectives. Leukemia 2019, 33: 2767–2778
  2. Jönsson, B., Hampson, G., Michaels, J., Towse, A., Graf von der Schulenburg, JM. and Wong, O. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur. J. Health Econ. 2019, 20(3): 427–438
  3. Jörgensen, J. and Kefalas, P. Reimbursement of licensed cell and gene therapies across the major European healthcare markets. J Mark Access Health Policy 2015, 3: 10.3402/jmahp.v3.29321.
  4. FDA approved Cellular and Gene Therapy Products, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
  5. EU public assessment report for Zolgensma https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma
  6. Alliance for Regenerative Medicine: 2019 Regenerative Medicine Sector Report, available from https://alliancerm.org/sector-report/2019-annual-report
  7. 7.Alliance for Regenerative Medicine: Q1/2015 Regenerative Medicine Sector Report, available from https://alliancerm.org/wp-content/uploads/2018/04/ARM_Q12015_Data_Report_Web_Version.pdf
  8. CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001, available from https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-progress-clinical
  9.  Cynata Completes Clinical Study Report for Phase 1 Trial of CYP-001 in GvHD, available from https://www.globenewswire.com/news-release/2018/12/18/1668688/0/en/Cynata-Completes-Clinical-Study-Report-for-Phase-1-Trial-of-CYP-001-in-GvHD.html

 

 

Market access for immune-oncology products in the EU

By: Claes Buxfeldt, HTA Director at NDA Group

In this article, published in Volume 22 September 2020 of Pharmafocus, Claes Buxfeldt discusses key considerations in early development to succeed with market access for immune-oncology products in the EU.


Integrating market access considerations early into your development program saves money and the chances that your product will reach the market, or a premium at exit.

At NDA we work closely with development teams covering many disease areas. Reflective of the global pipeline, a large part of our time is spent helping clients with immune-oncology portfolios. Here we cover what activities should be considered in early development of new immune-oncology (IO) drugs to ultimately secure reimbursement or optimise the asset’s value. We also explore how IO treatments are different compared to other treatment options and important attributes to consider.

Background

Achieving regulatory approval by demonstrating your product’s appropriate benefit/risk profile is only one step to reach and treat patients. In many countries pricing and reimbursement bodies have additional requirements to be fulfilled. This includes demonstrating comparative effectiveness and value for money.

Immune-oncology (IO) therapies

Instead of targeting tumours directly, IO therapies engage the patient’s own immune system to stop them. This approach may offer a more effective treatment for some patients. Important characteristics of IO therapy include full tumour regression, often a more sustainable clinical outcome and improved health-related quality of life compared to standard chemotherapy. IO therapy often has a different side-effect profile and durability of response.

The development of targeted immune checkpoint inhibitors resulted in the first FDA approval in 2011 of Yervoy (ipilimumab – CTLA4 antibody) for melanoma. Additional studies of PD1/PDL1 antibodies have led to regulatory approval both as single agents and in combination with other agents. IO therapies are now approved by EMA and FDA in treating melanoma, lung, kidney, bladder, head and neck cancer.

Key distinctive features of IO therapies:
  • Immune-mediated mechanisms of action,
  • Significant and increased durability of response,
  • Unique kinetics enabling delayed response,
  • Potential for shorter treatment period,
  • Possibility of being “cured”,
  • Different, often more manageable, side-effect profiles,
  • Sometimes severe and systemic adverse effects,
  • Better health-related quality of life,
  • Flattening of the Kaplan Meier survival curve suggesting durable responses,
  • Administrated often in unique combinations of IO drugs.

Critical questions in developing IO therapies include targeting of those patients most likely to respond, combining IO therapies with other treatment options, mitigating related side-effects and reducing the resistance to therapies. How to practically use these therapies in an evolving health care environment and when to stop treatment are also important.

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Contact us to learn more about how our integrated team can provide advice and support into your drug development plan.

 

 

 

The challenges to overcome age-related disease

Through the lens of COVID-19

By: Tom Hughes, CEO Navitor Pharmaceuticals & Johan Strömquist, CEO NDA Group 

In this article, Tom Hughes and Johan Strömquist discuss the challenges facing longevity research and the adoption of health span as a viable target for medicines in the future.


Over the last few years the field of longevity and health span research has exploded and our understanding of the processes underpinning ageing and age-related disease has expanded greatly. In spite of this there are several challenges facing researchers, drug developers and society in general that must be overcome before we start seeing broader treatments that can fundamentally change the underlying pathology of ageing.

In the age of COVID-19 the need for progress has never been more urgent. The disparity in mortality rates we are seeing during the ongoing crisis is a stark reminder that age greatly impacts health. According to statistics shared by the CDC, age is a strong determinant of mortality in patients with COVID-19. There’s little doubt that people fall into a high-risk category for death from infection with this virus once they reach middle age.

A similarly notable finding was reported recently in The Journal of Infection that age-related comorbid conditions also represent a major risk factor for severe morbidity and death from COVID-19, including type 2 diabetes (OR 3.68), hypertension (OR 2.72), and underlying respiratory disease (OR 5.15). Whether directly associated with co-morbid conditions of aging or biological aging itself, it is clear that age and age-related diseases are in the spotlight as accelerants for serious complications of COVID-19.

The excess morbidity and mortality associated with aging, apart from COVID-19, comes with staggering implications for healthcare costs. This is driven in part from the fact that over 80% of people 65 years and older suffer from multiple comorbid conditions, and that over 70% of healthcare spending is allocated to the ~32 percent of people with more than one chronic condition.

To learn more about NDA’s  services and how we can help you, click here.


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About the Authors

Thomas E. Hughes, PhD, President and Chief Executive Officer,
Navitor Pharmaceuticals
Dr. Hughes currently serves as a member of the Board of Directors of miRagen Therapeutics, Inc., is an advisor to Atlas Venture, and is a member of several scientific and strategic advisory boards, including Broadview Ventures, HotSpot Therapeutics, and Nimbus Therapeutics. Dr. Hughes holds a Ph.D. in nutritional biochemistry from Tufts University, an M.S. in zoology from Virginia Polytechnic Institute & State University and a B.A. in biology from Franklin and Marshall College

Johan Strömquist, Chief Executive Officer,
NDA Group
As CEO of the NDA Group since 2013, Johan has worked to expand the NDA’s capabilities across the continents working with a team of exceptional skill and expertise. Johan has a background as serial entrepreneur in the IT and technology industry, serving the life science industry for over 20 years. His main focus remains on developing NDA’s unique ability to provide world leading advice and a client experience second to none.


 

Enhancing Pharmacovigilance

To give oncology products the best possible opportunity

By: Dr Brian Edwards, Principal Consultant, NDA Group 

In this article, Dr Brian Edwards follows on from his very successful webinar, Opportunities to enhance Pharmacovigilance in Oncology, to argue that to maximise the benefits of innovation in our products we need innovative pharmacovigilance.


We are witnessing a tremendous expansion in oncology products with great opportunities that will benefit patients. Regulatory agencies have responded with expedited review pathways and schemes to enable early access. Data sources such as patient reported outcomes and real-world evidence are being adopted, especially once the product is marketed. The products may often be dependent on biomarker kits which have arisen from the exciting progress in genomic and stratified medicine.

Throughout healthcare and the life sciences, technologies such as machine learning or artificial intelligence are being introduced with great expectation about increased cost efficiency and productivity. With this greater importance of pharmacovigilance (PV) within a changing environment, we need to adapt to recognise limitations of certain data so that the quality that can be reasonably expected will differ if we are to strengthen current standards of PV.

However, we put all this progress at risk if we do not respond effectively to warning signs about medical quality, timely follow up and completeness of adverse drug reaction (ADR) case reports that could undermine confidence in novel oncology products by impairing the ability to make informed decisions.

For that reason, in their 2015 Guidance, the FDA urges sponsors not to report all serious adverse events, including those where there is little reason to consider them suspected adverse reactions.* Study investigators agree. There seems to be misinterpretation of what should be sent to sites resulting in examples where all reported adverse events are sent to every site that conducts a trial that uses that agent, regardless of relevance.

The full article was published in Volume 69 Spring 2020 of Pharmafile – Therapeutic areas in focus. 

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To learn more about NDA’s Pharmacovigilance services and how we can help you, click here.

*Safety assessment for IND safety reporting guidance for industry, Food and Drug Administration, 2015, https://www.fda.gov/files/drugs/published/Safety-Assessmentfor-IND-Safety-Reporting-Guidance-for-Industry.pdf

Potential Consequences of SARS-CoV-2 to ongoing clinical programs

The FDA Perspective

By: Laurie Smaldone, CMO/CSO, NDA Group 

In this white paper, NDA’s Laurie Smaldone discusses the potential impact of COVID-19 on on-going clinical trials.


Laurie Smaldone
CMO/CSO

The virus SARS-CoV-2 and the resulting COVID-19 disease have created devastating impacts around the globe on lives and livelihood, including major disruption of ongoing research and development activities for innovative therapies. From many perspectives the spread of SARS-CoV-2 has impacted and will continue to impact ongoing global registrational programs for a variety of disorders especially those not directly addressing the treatment or prevention of the SARS-CoV-2.

This disruption impacts all facets of the drug development process, from the Sponsor company, to the supply chain of investigational treatments to the productivity of study sites. The FDA has issued guidance to address the challenges that may arise from a range of disruptions that could impact the validity and integrity of clinical programs.

The FDA has noted that the spread of the virus may lead to GCP violations and protocol deviations, including impact on trial endpoints and safety collection.   FDA’s Center for Drug Evaluation and Research Director Janet Woodcock stated that “trials may be able to shift to tele-outcome assessments”, but others “may be damaged and may have to halt and not start up again until we can interact more freely”.

FDA’s recent guidance, “Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic”  addresses the potential impacts to ongoing trials.  The guidance provides stakeholders in the drug development and approval process with critical considerations for ongoing trials.

Potential COVID-19 impacts

Trials may be impacted in a variety of ways. Some sites have had to close down or have witnessed significant recruitment delays due to the inability of patient travel, illness, or redeployment of study personnel to address COVID-19 patients or trials.  These limitations can greatly impact study continuity and procedures to maintain study integrity.  In making decisions on trial continuity, Sponsors will need to do a robust   assessment of  trial conduct that may impact patient safety, GCP, drug storage and administration, and protocol procedures .

 

Click to read the full white paper

What did we learn from the 2009 pandemic?

By: Thomas Lönngren Strategic Advisor, NDA Group

In this commentary, NDA’s Thomas Lönngren discusses the regulatory learnings from the 2009 pandemic and how these are applicable to the current pandemic.


In April 2009 we were in the middle of an ongoing influenza pandemic and as the Executive Director of the European Medicine Agency I was ultimately responsible for the Agency’s response.

EMA’s responsibility was to ensure that we could get a vaccine approved as soon as possible and evaluate potential antivirals for the prevention and treatment of the infection. We also set up a robust surveillance system monitoring the safety of vaccines and antivirals when they were put on the market. All of this had to be done while ensuring that staff and experts working at the agency could continue to operate and deliver advice to developers of vaccines and antivirals while maintaining their own well-being.

One of these proposals was the idea to create what became known as the Mock-up vaccines. Specific guidance was developed by the Agency for an assessment procedure for pandemic influenza vaccines

 

It was an intense period with many meetings, ensuring the operation of the agency as well as daily teleconferences with European Commission, WHO, other regulators such as the FDA, and the industry. Luckily there were preparations in place.

When the pandemic broke out in April 2009 we were not taken by surprise. Warning signals came from WHO as early as the beginning of 2000. The SARS outbreak in 2002 had also served as a wakeup call. The outbreak of bird flu caused by H5N1 virus was an additional indication that a pandemic was imminent.

Click here to read the full commentary
 

To learn more about our how our advisors can support your drug development program click here

 

Related article: What do we need to know before the next influenza pandemic: by Vaccines Expert Peiter Neels

Medical Devices and their growing regulatory challenges

By: Tina Amini, Division Director Medical Devices, NDA Group 

In this article, NDA’s Tina Amini explains what companies need to look out for in the growing area of device regulation.


Tina Amini

Recent scientific advances and improvements in enabling technologies have opened new avenues for convergence among medicines, diagnostics, and devices. The medical technology industry continues to be one of the most diverse and innovative sectors.

Major shifts in the health care environment including regulatory requirements make it increasingly difficult for medical technology companies to sustain traditional growth and profitability.

Why can we say that the regulatory challenges in the medical device field are growing?

With the introduction of the new Medical Device Regulation (MDR), and the pending In Vitro Diagnostics Regulation (IVDR), the regulatory landscape in the EU has undergone tremendous change. The new situation gives rise to uncertainties and unknowns and it will take time before it settles and becomes predictable again.

 

Why was the new regulation introduced?

“The new regulation aims to boost patient safety and effectiveness of all the medical devices that are commercialised but also to increase transparency to make the process clearer to everyone involved.”

“Shortcomings in the Directive in divergent interpretations also resulted in different output from the Notified Bodies. The new regulation attempts to address this as well.”

 

How does the new regulation change the European device market?

“The situation is currently uncertain. The delay in the database EUDAMED, an insufficient number of designated Notified Bodies, and lack of sufficient Guidance documents are all causing challenges. There is a lot of guess work awaiting official decisions and guidance. It is however certain that the new regulations will bring substantial change to how medical devices are brought to and maintained in the market.”

“The regulation also impacts the Notified Bodies as they have to be re-designated under new regulations. The time and cost associated with this has actually resulted in some Notified Bodies not applying for designation under the new regulations.”

“Up until now only eleven Notified Bodies have been designated under MDR, and three under IVDR and not all with full scope, compared to about 80 Notified Bodies that were operating under the MDD in the early 2010s. Today there are not enough Notified Bodies to pick up all the work. For companies this means longer timelines as you queue to have your product reviewed. We’re already seeing the manufacturers struggling to find a Notified Body to take them on.”

 

Article originally published in the April 2020 issue of Pharmafocus

Click to read the full article

How we can help?

NDA’s Medical Device Division has extensive experience with both medicines and medical devices. We can support you with your interactions with Notified Body  and assess your product from a scientific, technical, and regulatory perspective to be adequately prepared to meet the requirements. We also have the expertise to help manufacturers to implement the requirement of the MDR and IVDR regulations.

To learn more about how we can support your company, click here to see our full range of services.

NDA Medical Device Division launched

We are proud to announce the creation of a new division within NDA, focusing on advice and support to companies facing increasing regulatory challenges in the device space.

The new medical device division is led by Dr. Tina Amini, formerly Head of Notified Body and Certification Authority at LRQA, and aims to support drug development and medical device companies under pressure from increased regulatory requirements.

Recent scientific advances and improvements in enabling technologies have opened new avenues for convergence among medicines, diagnostics, and devices. The medical technology industry continues to be one of the most diverse and innovative sectors.

Many innovative pharmaceutical and biotech companies find that their medicinal products rely on appropriate diagnostics or delivery systems operating under a different regulatory framework. Major shifts in regulatory requirements have put increased pressure on traditional medical technology companies to sustain growth and profitability.

The increased scrutiny by regulators on both sides of the Atlantic, most recently embodied in the new European Medical Device Regulation (MDR), has created challenges for these companies to achieve marketing approvals and certifications, as well as to remain competitive.

The increased dependence on sophisticated diagnostics for patient stratification, along with the convergence of the fields through advanced combination products creates new scientific and regulatory challenges – how do you assess this product? Is it a device or a medicine?

“It is clear that the impact of different types of medical technologies, including software, on drug development and other crucial medical interventions is enormous”, comments Johan Strömquist, CEO

NDA. “With the promise of big data and AI, as well as stratification of patients down to the genome level, this is a trend that will only continue to expand.”

He continues:

“We see that even the regulators are struggling to understand the convergence of these new technologies and it is in such areas where clear advice and guidance is most valuable to innovative companies. With the creation of the NDA Medical Device Division, we are taking a crucial step to formally move into this area to partner with our clients on the medical device aspects of their development programs as well.”

NDA Group’s ambition to leverage synergies between drugs and devices has provided the company with a special focus for the new division. The challenges associated with borderline classifications, drug / device combinations and in-vitro diagnostics are core points for the new division.

Dr. Tina Amini, Division Director, Medical Devices, comments:

“The clear focus for us is to support companies with the medical device aspects of their development program to enable access to the market for the medicines that these products enable. NDA Group’s excellence in the field of medicines is now complemented by an impressive understanding of the regulations surrounding medical devices on both sides of the Atlantic.”

She continues:

“Being able to work with one partner to address the regulatory development challenges for both the drug aspects and the device aspects of their product is going to be a game changer for many companies. This will expedite faster access to market and will create great efficiencies compared to trying to manage and coordinate multiple vendors. When you pair that up with the unprecedented experience and expertise of the NDA team you’ve got an unmatched resource that will be able to make a huge difference in getting the right products to market as quickly as possible.”

Learn more about our new Medical Device Services here 

or

Contact us now to talk about how we can support your team.

Hopes and challenges in the future of the immuno-oncology field

There is no doubt that the area of oncology development has exploded in the last few years. The global oncology pipeline at the end of 2019 had almost doubled since 2014, reaching an all-time high with 374 drugs entering Phase 1 and the approval of several new drugs with unique modes of action. But all is not rose-tinted news, in this article published in the February addition of Pharmafocus, Dr Laurie Smaldone Alsup, Chief Medical and Chief Scientific Officer at NDA predicts some challenges in the field of oncology.

“Historically, clinical trials are designed so that the patient is relatively unencumbered by other diseases, to make it easier to assess whether the drug works or not. But in reality, people may be elderly, have organ failure and/or have other disorders that require chronic medications for other conditions. Understanding how these new therapies behave in the real world is critical”

 

Click here to read to Article

The future of NDA in the Nordics

 

NDA has a new general manager


In August this year Robert Kronqvist joined the NDA team as General Manager for the Nordics. With his background from large pharma, small biotechs and research institutions he is bringing a plethora of experiences with him to the NDA team in the Nordics.

Through this short interview we tried to figure out what makes him tick.

 


 

You have worked for the best part of your career in life science. Tell us a little bit about what motivates you.

“Thank you! It’s great to have the opportunity.

To begin with I’ve always found life science and pharmaceutical development fascinating. Working in as diverse contexts as large pharma, where I’ve spent a significant part of my career at AstraZeneca, and then seeing things from both a small biotech and research institute perspective, I feel I’ve got a great breadth of experiences.

What ties them all together though is the wish to make a difference, and in drug development you really can make a difference – both every day at work, but of course at the end of the day for the patients we serve.”

So, what is the biggest difference between working at a large company and then taking over as CEO in a small biotech?

“Well, one of the most obvious differences is of course the access to resources. In a large pharma you have resources and capabilities that have been built up over many years relatively easily accessible, whereas in a small company you have to find partners and different ways to collaborate to get the different aspects of drug development covered.”

“It’s not uncommon to bring in different people to satisfy specific and very discreet needs in a small company, just because you have that need right there and then. There is always a risk that continuity will suffer from that, or that there are challenges with knowledge management across collaborators. In that respect it’s great that we have such an incredible breadth and depth of expertise here at NDA, since this gives so much expertise in one provider.”

How would you describe the Nordic Life Science scene right now?

“Nordic Life Science is quite remarkable, even though it certainly has its challenges. We have a very strong reputation of high-quality science and innovation in this corner of the world. We are also pretty good at taking science from academia and spinning out companies to take this forward, but we struggle more when it comes to financing of and doing good drug development when we reach those stages.”

“I think the situation for the industry has changed quite a bit in the last ten years. Both because we’ve got a good tradition and track record in spinning out innovation, but also because of large pharma’s increased willingness to rely on external innovation and in-licensing to fuel their pipelines.”

It seems like there are a few important hubs across the Nordics. Is this a trend you think will continue?

“Yes, I think so. Part of the explanation to the origin of these hubs comes from large pharma who have left or even continue to contribute to expansion of infrastructure in very specific locations that are attractive to smaller biotechs. But regardless of whether there is infrastructure in place or not we need proximity, partnerships and networks to build a creative environment for science and innovation to flourish.”

 

How do you think the Nordic Life Science scene will develop over the next couple of years?

“In two years’ time I believe we will see a number of the biotech companies with promising treatments will have achieved important scientific milestones as well as increased financing. With the current influx of biotech clients, I also believe that NDA as a consultancy company will have been able to have an even greater impact on the development of good medicines in the region.”

Speaking of which, what do you think that NDA will be able to contribute to the small biotechs?

“I think it’s a matter of providing coaching and guidance. We have so much experience from helping companies from all over the world optimize their development programs and it offers us a fantastic opportunity to bring this experience to the Nordic market.

This can provide such great benefits to companies, both in terms of plotting their route to market to enable good execution of their development activities, and, if they are contemplating earlier stage exits, to make sure that they get maximum value out of any out-licensing deal that they might be considering.”

Based on your experiences, what do you think that you can bring to small biotechs as a part of NDA?

“I think it’s really two things. Firstly, I have a lot of experience that is highly relevant to the small biotech company’s situation. As a CEO of one of the companies out of Karolinska Development’s portfolio I’ve been there and seen what it is like.

Secondly, I’ve got a pretty good network by now. This will be beneficial both because I know many companies out there that will benefit from the support that NDA can offer, but also because science and innovation really is a team sport and identify the right players for your team requires a large network. This is something that I can help with.

These two things coupled with a great commitment and enthusiasm for drug development is really what I think I can contribute with.”

What do you see as NDA’s greatest challenge in the Nordic region?

“I believe strongly that we have some fantastic opportunities engaging with small biotech companies in the Nordic region, supporting their development and augmenting their capabilities. That said, we have for the past ten years been an incredibly international company with our client facing people spending almost more time in the United States than here in our home markets.

I think that this has created the perception that we are not interested in supporting our local biotech scene, something that couldn’t be further from the truth. The fact that we have been very international and learned a lot from companies from all around the world is of course fantastic, but the perception that we are not available for our colleagues here in the Nordics is a challenge. I will work a lot on ensuring that we are present – that we are visible here at home – and that we are available, flexible and responsive. I think all those things will be necessary for us to be allowed to share our experiences in the Nordics.

I am really looking forward to the opportunity to meet with and get to know more of our colleagues in the Nordic Life Science community to learn more about their thoughts and needs so we can find ways to support in an optimal way.”


 

What do we need to know before the next influenza pandemic?

Dr Pieter Neels

NDAs Advisory Board member and vaccines expert Pieter Neels together with a group of scientific and public health experts and key stakeholders convened for the 2nd International Alliance of Biological Science (IABS) to review the status of the current knowledge regarding the relationship between narcolepsy and the administration of the adjuvanted pandemic influenza vaccines, with the goal of being prepared for the next influenza pandemic.

The result of the discussions have been documented in the report: Meeting report narcolepsy and pandemic influenza vaccination: What we know and what we need to know before the next pandemic?

The highlights from the report include:

  • The association between the reported influenza vaccine and narcolepsy has been consistent in the countries in which it has been studied.
  • There are no clear associations observed between development of narcolepsy and the other pandemic adjuvanted vaccines.
  • The public health response during a pandemic is critical.
  • International collaboration and the capacity for data sharing should exist before the next pandemic.
  • Research on narcolepsy therapy should be supported and clinicians skilled in management should be available.

To read the report in full click here.

 

Global capabilities for local growth

In this commentary, NDA’s Johan Strömquist and Thomas Lönngren provide their reflections on the unique opportunities the Nordics present for the Life Science Sector.


Nordic life science has been on a roller-coaster for the last twenty years. It was at the epi-centre of the creation of some of the world’s most successful pharmaceutical companies, such as AstraZeneca, Pharmacia, Novo Nordisk, Leo and Lundbeck. The downsizing and movements of the Swedish giants left a vacuum in the region that was hard to fill.

It has taken until now for the Life Science community to stage its comeback to the world stage. This time however, the success does not belong to one or two big companies, but to hundreds of small, agile and innovative biotechs. The problem these smaller companies have faced has been funding to allow them to properly develop and commercialise their innovations. But even in this arena, we see that great strides are being taken to address the challenges.

We are quickly approaching the four-year anniversary of the announcement that Janssen licensed one of Alligator Bioscience’s early stage immune-oncology agents for a sizeable amount. Last year Wilson Therapeutics was acquired for a whooping SEK 6.6 billion and AstraZeneca invested heavily in SOBI. This year we’ve seen significant scientific and/or financial successes from smaller companies such as Cantargia, Xbrane, Immunicum, Cereno Scientific and Xintela and more biotechs than ever have found their way to the stock market via Stockholm Nasdaq.

However, using the capital that this success unlocks comes with its own challenges. How should a small biotech prioritise and use the capital in an optimal way to ensure success and that scientific advances are within parameters that are both approvable and reimbursable?

For over twenty years NDA has worked to support companies overcome these hurdles and make sure that precious resources are used to optimise the outcomes.

Says Johan Strömquist, CEO NDA Group:

Johan Strömquist CEO

“The Nordic life science community is remarkable – it is resilient, innovative and able to create a lot from very little. What I see right now is incredibly exciting and encouraging – significant strides in cross-company and cross-border collaboration.”

He continues:

“Through organisations like Sweden BIO, Medicon Valley Alliance and, more recently, the LSX Nordic Congress in collaboration with Stockholm Nasdaq, I see companies getting together to discuss common concerns. As a trusted international advisor, we have a unique opportunity to bring learnings from all over the world into the Nordic life science scene for the benefit of everyone.”

 

NDA’s Strategic Advisor, Thomas Lönngren, formerly Chief Executive of the European Medicines Agency, agrees:

Dr. Thomas Lönngren

“The US remains the most important development region for new medicines in the world. How do they do it? By bringing everything together – capital, universities, hospitals, entrepreneurs and large companies. Just look at Boston; you have everything within walking distance!”

He continues:

“Though the goal may not be to emulate the American success story 100% the movements we see toward more collaboration and cross-fertilisation is very positive. We have strong academia and are very good at spinning out companies in the Nordics. Now we just need to improve the way we develop new medicines so they can reach the market and the patients that need them consistently to make the Nordics an important hub of drug innovation.”

To learn more about our services and how we can help click here

Prime – The European approach to expedited pathways

By: Steffen Thirstrup, Director, NDA Advisory Board & Eva Lilienberg, Principal Consultant 


Steffen Thirstrup, Director, NDA Advisory Board

A timely market introduction is a critical component of any drug development strategy – not only from a commercial stand point, where an early introduction can mean beating competition to market or simply significantly increased revenue, but also to patients awaiting better or alternative treatment options.

The debate around how regulators can facilitate this process on both sides of the Atlantic has resulted in new pathways for new medicines of major public interest. The FDA have the Breakthrough/RMAT pathways, and in the EU, EMA has put the PRIME process in place.

In this white paper, Steffen and Eva discuss the different tools available to European regulators and the experiences so far with the PRIME pathway.

To read the full article download the PDF

Download PDF

How we can help?

NDA Group supports life science companies all over the world with the aim to streamline the global development and commercialization process in order to accelerate patient  access to important medical therapies.

Whatever regulatory hurdle you’re facing, we can help you optimize every regulatory interaction and shape the dialogue about your product to create a more direct path to approval.

To learn more about our services and how we can help click here

Or contact us at meet-us@ndareg.com to talk to one of our experts.