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

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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.

European Regulatory Meetings – how best to prepare and perform

By: Steffen Thirstrup, Director, NDA Advisory Board 

In this article, NDA’s Steffen Thirstrup discusses three important factors to help you prepare for high stakes meetings with EU Regulators.


Steffen Thirstrup, Director, NDA Advisory Board

Having the possibility to engage face-to-face
with EU regulators is the ideal situation to help with authorisation of your product. However, most interactions in the EU are in writing and very few sponsors have the opportunity for a meeting with the EMA.

The most critical meetings are those where the scientific committees of the EMA would like to question the sponsor to fully understand the data and the sponsor’s position.

Such ‘high-stakes’ meetings like CHMP oral explanations are often the final chance for a pharmaceutical sponsor to convince the EU regulators that the benefit/risk balance of their product is positive.

We recommend that you apply the pertinent Benjamin Franklin quote,throughout your planning for a successful EU ‘high-stakes’ meeting.

“By failing to prepare, you are preparing to fail”

1.  Be prepared

Being well-prepared and performing professionally is the key to success. The sooner you can establish your team the better. Having your process and timelines mapped out will help prepare your team and maximise the chances of success. Plan for rehearsal sessions where you can work on the presentation, back-up slides and train comprehensively for questions and answers. If time allows, the team can be further coached by bringing in a ‘challenge panel’ of external experts who can act as a test-panel for your presentation and Q&A performance.

 

2. Know your data

EU regulators expect meetings like an oral explanation to be a scientific discussion based on data. To ensure this is the case, you should demonstrate intimate knowledge of your data to meet the regulators’ concerns as well as being able to present your position in a confident manner. To get to this stage takes meticulous preparation of key messages, slides and the script for the presentation and for potential questions.

One pitfall is to make use of already prepared slides as they will have been prepared with another aim in mind. Starting with a blank piece of paper, writing down the key messages that you believe the regulators need to hear gives you the best basis for creating slides that convey your key messages.

It is very important to make your slides as simple as possible, omitting any unnecessary information and this can be a challenge. Complicated slides distract the audience from your presentation; remember that the slides are there as an ‘aide memoire’ and not for the regulators to read verbatim as they will then not be listening to you!

In addition, keep graphics as simple as possible. A rule-of-thumb is a maximum of six points per slide with no more than six words in each.

 

3. Know your team

In many cases it makes sense to create sub-teams to address the individual topics, agree on key messages and create the slides. Group sessions can then be used to merge the slides into one presentation, optimise the script and ensure all information is in keeping with the key messages.

Additionally, there may be a need to coach key opinion leaders and/or patient representatives to speak or respond to questions on your behalf.

Finally, determining potential questions and being able to respond clearly and unequivocally to these are equally important to increase your chances of success. One way of doing this is to create a Q&A grid where all members of your team add likely questions, and questions they find difficult to answer. The team can prepare top-line responses, a more detailed explanation and any supporting slides for these potential questions and, of course, determine who is going to respond if the question is raised.

 

How we can help?

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

In Europe, we assist with Scientific Advice Group meetings, Oral Explanations, PRAC meetings and preparation for intense pricing negotiations. In the US, our Principals and Communications Strategists are particularly experienced at preparing for FDA Advisory Committee meetings, in addition to pre-IND,  end of phase 2, pre-NDA/BLA, PMA, and mid- and late-cycle review meetings. We offer our clients an unparalleled breadth of global experience and expertise to drive efficient product development and health authority interactions across the US and Europe.

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.

Article originally published in the Jan/Feb 2019 issue of Pharmafocus

Click to original article

Importance of Excellence in Scientific and regulatory Communication

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

In this white paper, PharmApprove’s Lisa Peluso, discusses how  excellent communications are  the backbone of streamlined, efficient programs achieving approval and market access.


Lisa Peluso, Director

The ever-changing complexities of the regulatory world demand excellence in communicating science, data, development program goals and intentions, as well as how foreseen risks are being managed.

These complexities also mean that drug development companies must engage candidly with regulators and lead the scientific conversation with clarity and flexibility, yet be poised to negotiate their position.

While we work with global companies to strategize and execute successful engagements with regulators  at critical junctures such as Oral Explanations and FDA Advisory Committee meetings, excellent communications are  the backbone of streamlined, efficient programs achieving approval and market access.

A number of potential pitfalls await companies with even the most solid science and robust datasets

To avoid costly delays along the path to regulatory approval and commercialization, companies need to:

1.  Communicate internally

To ensure alignment of clinical goals and regulatory approvability with the reality of a product’s potential market value and accessibility the company needs efficient internal communication. The danger lies in a narrow focus on just regulatory considerations: A product may be effective but if there is no place in the market or doubtful economic defense of pricing, the company is wasting time and money. Development plans must be based on all considerations, requiring careful coordination and collaboration across all divisions.

 

2. Communicate with regulators

Unfortunately, too many companies engage with regulators much too late, or not at all, or lack the candor and skill needed to usher a product  smoothly  through the approval process. Excellent communication means asking the right questions, clarifying any questions from the regulators and responding in a collaborative and transparent manner. Remember that specific expertise in your company’s product is rare, hence it’s critical to clearly shape the story of not just the data but also the program, goals and risk management commitments.

 

3. Communicate globally

Just as internal divisions must be aligned, regional requirements vary in Europe and across the globe. Companies risk costly mistakes and a need to “redo” when they don’t share knowledge freely internally.

This is particularly important for US/EU programs. Learnings from multiple regulatory authorities or groups of payers must be transferred so there is no duplication of effort – or waste of time – on the other side  of the pond. Constructive interactions speed efficiency.

 

4. Engage physicians, patients and advocacy groups

Get the community involved early, including doctors and potential study sites as well as patients and advocacy groups. Communicating with these stakeholders also demands delivering the messages behind the data in a concise and compelling manner to a wide variety of physicians, KOLs and other influencers.

With such deep knowledge of one’s product, it’s a daunting challenge to distill the information, refine the message and keep it brief and clear for the audience.

 

5.  Negotiate with payers

Companies put enormous effort into developing slide  decks, internal FAQs and value proposition documents.  This plethora of planning and information is to no avail if the company does a poor job of interacting with payers. In addition to understanding the audience around the table and their goals, the company must respond deftly to questions, defend the data, and communicate persuasively. Every interaction is an opportunity and certainly, when it’s time to negotiate pricing, excellence in communications is critical.

 

How we can help?

NDA Group supports life science companies all over the world with the single 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 — a Type B meeting, a document submission, or a SAG hearing — our industry-leading professionals will help you strategize and execute successful engagements with any global authority. We’ll help you optimize every regulatory interaction and shape the dialogue about your product to create a more direct path to approval.

In the US, our Principals and Communications Strategists are particularly experienced at preparing for FDA Advisory  Committee meetings, in addition to pre-IND,  end of phase 2, pre-NDA/BLA, PMA, and mid- and late-cycle review meetings. In Europe, we assist with Scientific Advice Group meetings, Oral Explanations, PRAC meetings and preparation for intense pricing negotiations. We offer our clients an unparalleled breadth of global experience and expertise to drive efficient product development and health authority interactions across the US and Europe.

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Why should you engage early with regulators and HTA bodies?

By: Dr Mira Pavlovic-Ganascia & Claes Buxfeldt, NDA Group

In this white paper NDA’s Dr Mira Pavlovic-Ganascia and Claes Buxfeldt provide insight into the reasons to seek scientific advice and elaborate on what the expected outcomes of constructively engaging with regulators and HTA bodies could be.


Dr Mira Pavlovic-Ganascia
Claes Buxfeldt

In Europe there are many routes to gain scientific advice directly from official authorities. National regulatory advice is a routine practice of most regulatory agencies, as well as of the European Medicines Agency (EMA).

Scientific advice in the HTA arena is more recent. In this context, national HTA advice is provided against the backdrop of country or region-specific policies and legal requirements. Early dialogue involving multiple HTA bodies is also rather recent, provisioned in the context of the European Network of Health Technology Assessment (EUnetHTA) Joint Action 2 as well as specific actions financed by the European Commission (SEED)(ref) and involving both EMA and EUnetHTA. Indeed, in recent years, EMA and EUnetHTA have led several combined regulatory-HTA pilots to get experience both on the process and the content of such an exercise (1,2,3).

Since the creation of our NDA Joint Advice service offering in 2011, the NDA Advisory Board has also conducted numerous such projects for clients, focusing on ensuring a thorough understanding of the varying regulatory and HTA requirements and bridging the relative efficacy – relative effectiveness gap.

The means to gain scientific advice are many and diverse, but so are the reasons companies strive to get it.

Why seek scientific advice?

The combined scientific advice/early dialogue processes allow a company to engage relatively early in discussions with regulators and HTA bodies. The label on the tin indicates that the main interest of such an engagement would be to gain scientific input into the development program of a product to be able to steer it away from regulator, HTA and payer pitfalls and towards rapid patient access.

The scientific input usually covers the proposed study design(s), comparators, endpoints, target study population and inclusion criteria’s, study length and other key aspects important both for regulatory and HTA bodies. More in general, this early discussion will allow a company to check if the evidence to be generated for EMA is relevant for HTA submissions.

Asking for a combined regulatory and HTA advice is even more important in cases where treatment guidelines are weak or inexistent and/or there is no relevant HTA decision available in the field of interest.

Reality, however, is even more diverse than this and the reasons and rationales for companies to go for scientific advice therefore vary greatly.

1. Stake holder engagement

Engaging early with regulators and HTA bodies can be a crucial motivation for engaging in the scientific advice process, as it provides a unique opportunity to introduce these two key stake holders to the science and circumstances behind the company’s product. This can benefit the process in two ways:

  1. In any future engagements the regulators and HTA bodies will know the product/technology and targeted patient population better. This will potentially enhance future interactions and remove communication hurdles.
  2. By engaging with the right individuals in the right agencies, interest in the product can be sparked which can lead to constructive and positive input along the product’s development path. Building rapport with assessors are again an important vehicle to friction less communication.

 

2. Demonstrating progress

Engaging with external experts in a structured manner is also an excellent way to demonstrate to boards and stake holders that the product is progressing through the development process. Constructive feedback from regulators and HTA bodies can help steer the product development but can also be a value driver for the company.

If this is a main driver for expert engagement or seeking scientific advice, other reasons should also be carefully considered to optimize the value of the interaction. Seeking endorsement for the sake of it is rarely the optimal use of the time spent by external experts or regulatory agencies and HTA bodies.

 

3. Compliance

Many multi-product pipeline companies have highly controlled processes for how to progress products through the development process. This may well include the consideration and execution of a formal scientific advice procedure.

As this reason is not a value driver in itself, the reasons that the company put this requirement in place in the first instance should be carefully considered when running through the motions. Teams following a check list risk missing the underlying strategic reasons for why the process is necessary and may therefore not consider alternatives or options that might fit better or add more value.

 

4. Internal alignment

Although not its primary purpose, internal alignment is an incredibly valuable potential outcome of any scientific advice/early dialogue process. Teams working towards a clear goal along a clear timeline tend to glue together and more easily visualize the ultimate outcome. At NDA Group we’ve seen many occasions where the formal process has created strong composite teams. These teams are primed to progress the product through development with a determination and shared purpose that would not have been possible without the structure and clear goal that the scientific advice process offers.

Achieving internal alignment should therefore always be considered a potential beneficial outcome and should be planned for accordingly.

 

Read the full white paper

Don’t miss the opportunity to hear to Dr Mira Pavlovic-Ganascia and Claes Buxfeldt speak about HTA and Europe at our complimentary seminar:  Optimizing Value – Regulatory and Market Access Considerations in Stockholm on the 21st May.

Read more and register
 

The state of the Swedish orphan pipeline

In a short piece on the rare and orphan disease pipeline in Sweden, Business Sweden, together with SwedenBIO, have outlined the current state of Swedish orphan development. The report describes a vibrant drug development landscape that is reflective of the global move towards orphan development and the strong emphasis on oncology.

Over the last 19 years Swedish companies have been granted 60 orphan designations in the EU and 47 in the US. Unsurprisingly, oncology and neurology are the leading therapeutic areas representing 38% and 11% of the Swedish orphan pipeline respectively.

In total, Swedish companies are developing orphan drugs in 16 different therapeutic areas and it is interesting to note that transplantation comes in as the third largest area of interest in terms of number of compounds.

If the number of products in the pipeline and the activity of the life science networks and communities are any measures of future success – the Swedes and Danes are in for a very exciting ride!

In addition to the findings outlined in the report, we have looked at the geographic origin of the companies contributing to this space and two areas clearly dominate this development; Stockholm/Uppsala and Medicon Valley in the Malmö/Lund/Copenhagen region.

If we were to include Danish companies in the Medicon Valley area, we are sure the picture would change, but the two regions are undeniable hot-spots when it comes to pursuing orphan targets in the Nordics.

The Swedish biotech stage is thriving and expanding – at last count the industry organisation, SwedenBIO, had 265 member companies and it keeps growing. Medicon Valley Alliance is a network of life science organisations, from academia and local health care bodies, to life science companies and service providers, that span the Copenhagen/Malmö/Lund region. They have also grown at a steady pace and comprises over 250 organisations today.

If the number of products in the pipeline and the activity of the life science networks and communities are any measures of future success – the Swedes and Danes are in for a very exciting ride!

You can read the original report at over at Business Sweden’s website.

4 ways that HTA will change under the new European regulation

By: Johan Strömquist, CEO, NDA Group

In this white paper NDA’s CEO, Johan Strömquist, discusses four ways that Health Technology Assessments will change under the new proposed European regulation.


Johan Strömquist, CEO

Over many years the European Network for Health Technology Assessment (EUnetHTA) has been working to HTA bodies into the same room to harmonise and develop thinking around the assessment of medicines from a societal point of view. The approaches to HTA assessment across Europe vary significantly across member states causing confusion, challenges and increased costs for drug development so the work of this voluntary network has been greatly appreciated by industry, payers and politicians alike.

However, the network’s voluntary nature and a lack of a formalized framework for the continuation of the activities have caused issues to progress and harmonization throughout its existence. To stabilise the situation and institutionalise the continued efforts, the European Commission (EC) has therefore presented a new HTA regulation back in January 2018 that will be up for decision in the European Parliament in spring 2019.

The regulation does not put any restrictions on how health economic decisions should made or how pricing of products should be carried out

Should this new regulation be endorsed as it is it will have important impact on drug developing companies and the way that drugs will and should be developed if drug developers want to optimise their path to market.

The regulation addresses four things that every biotech CEO and industry leader should be aware of.

1.   Joint Clinical Assessments

Years of experience and significant respect for the subsidiarity principle enshrined in the Maastricht Treaty has enabled a pragmatic approach in the draft regulation. The regulation does not put any restrictions on how health economic decisions should made or how pricing of products should be carried out. Instead it focuses on the area of commonality that has been agreed and pushed with EUnetHTA (with varying support from different member states). This area is clinical assessment or the assessment of relative effectiveness of the product.

Should the regulation pass as it is this means that all novel products (defined as any medicinal product passing through the centralised procedure or any medical device or IVD that receives an opinion under the new Medical Device Regulation) will be assessed under a new centralised clinical assessment scheme. This would in theory replace national clinical assessments and could straighten a product’s road to market.

Once a product has been assessed for its clinical benefits, national authorities would still have to assess its value in the local market but one crucial step in this process would have been eliminated and the outcome would be harmonised across the EU.

At NDA we will be ready to support clients managing this process when / if the regulation kicks in. Through our extensive experience in the market access area and by support of collaboration partners   we are ideally placed to work with companies pulling their HTA dossier together.

 

2. Joint Scientific Consultations

The practice of scientific consultations has become increasingly well-established yet is a relatively under-utilised mechanism for companies to improve their understanding of the different stake holder requirements. One reason why this is the case is because the process is time consuming. Another is because the procedure is under resourced and to a great extent based on voluntary contributions from the member agencies.

Under the new regulation all scientific consultations would be managed centrally, including the parallel consultations with the EMA.

This is most likely going to have relatively little impact on the use and performance of consultations to begin with, but by establishing a long-term platform for these activities there will be greater room to expand resources, improve the process and harmonise the contributions from the member states.

NDA’s Joint Advice services addresses several of the weaknesses of parallel consultations. By relying on the experts that built the European regulatory systems we deliver high quality, high speed advice. This is used by many companies as a proxy for formal consultation when time is tight, or as a way to prepare for the formal process to maximise the value that can be attained from engaging early with HTA bodies.

 

3. Identification of emerging health technology

Horizon scanning as it’s popularly called is a resource intensive activity when it is performed at the member state level. Consolidation of this to a central European function will allow more efficient use of resources, but it will also result in an overall higher quality of the output and material that will be used for training and intelligence at the member state level.

In the long run biotechs and pharma companies can expect their HTA counterparts to be more up to speed with emerging health technologies and that the playing field will become gradually more level across countries thanks to this.

 

4. Voluntary Cooperation

A clause that is easy to dismiss in the new regulation is where it speaks of voluntary cooperation. However, this means that the current cooperation, which has been moving slowly but has left important marks in the way assessments are carried out, has a formal home. This is important as it gives legitimacy and encourages the continuation of EUnetHTA’s activities in a new and more official format.

Expect this to lead to increased cooperation between HTA bodies in Europe and an increased exchange of scientific and methodological ideas across the member states.

NDA continually monitors the regulatory development to cover any relevant regulatory changes. As many changes are not publicised through regulation, but are a matter of practice in the agencies, our exposure to on-going procedures and 25% of our staff having experience working at a regulatory body are essential in staying on top of the change. Over the last five years NDA has been involved in more than 40% of the new medicinal products approved in the EU.

 

Will it lead to harmonisation?

There are still big differences in the legal traditions, pricing and reimbursement systems and the socio-economic circumstances across the member states of the EU. We will not see these things change overnight, hence it will take substantial time for these collaborative efforts to bear fruits across the range of the spectrum. Since health care systems and financing is a country matter, appraisals of value and decisions if a new medicine should be granted access is still a country matter. This includes all decisions around pricing as well.

We have however already seen how collaboration, exchange of ideas and development of joint therapeutic area specific methodologies are spreading. This does lead to harmonisation in a few crucial areas around clinical assessment and this is not just progress, but also important for drug developers across the world, as it has the potential to make HTA more transparent and therefore predictable.

The new HTA regulation builds on these important steps to increase the transparency and predictability even further. Despite the challenges that remain, this is progress.

As a partner NDA is ideally placed to support drug development companies navigate through the changes that this new regulation entails. By tracking and reflecting the current thinking and practices of the agencies and by providing tailor made, actionable advice and the help to operationalise this we are looking forward to seeing how this new regulation can help increase predictability and improve the speed with which important therapies reach patients in need, all across the EU.

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Observations of a market access expert

An insightful interview with NDA’s Claes Buxfeldt, HTA Director, entitled “Observations of a market access expert” featured in Aprils edition of Pharmafocus.

Reimbursement is the fourth hurdle of pharmaceutical development. Payers and Health Technology Assessment (HTA) bodies demand compelling evidence of how new interventions improve the standard of care and public health.

In the article Claes reflects on his experiences, challenges & opportunities and highlights the key trends and drivers for changes that will affect the Life Science industry in the HTA and market access space.