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. 

Click to the full article
 

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.

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
 

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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Webinar – Is an Early Development Strategy Really Needed?

Due to the high costs for product development, it is difficult for small and medium-sized pharmaceutical companies to take their product to the market on their own. By integrating project-specific development strategies and deliverables in the overall product development objectives can help develop a strategic roadmap to add value during product development which in turn can optimise exit strategies.

Join us on 16th May at 15:00 BST, 16:00 CEST, 10:00 EDT for this engaging webinar where Dr Niamh Kinsella, Biologics Expert, VP, Early Stage Development, NDA Group will cover the following strategies:

  • See how to implement a stage-gate approach for the early development of your product
  • See how a global regulatory strategy can be implemented
  • Determine your strategy for agency interactions.

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

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


Click here to book your place today!


Dr Niamh Kinsella, Biologics Expert, VP, Early Stage Development, NDA Group.
Niamh has worked in regulatory affairs for over 15 years and has 5 years’ experience in biopharma industry prior to entering regulatory affairs. Niamh joined NDA in January 2010 and specialises in CMC / quality aspects of biological products, including recombinant proteins, monoclonal antibodies, vaccines and advanced therapy medicinal products. Niamh has strategic experience in CMC/quality and regulatory development throughout the product lifecycle and also has experience in regulatory operations in the preparation of documentation for regulatory submissions (Agency meetings, IMDS/INDs and MAAs/BLAs).

 

 

Effective presentation of immunogenicity related data in regulatory dossiers

NDAs Paul Chamberlain, Biopharmaceuticals & Immunogenicity Expert and NDA Advisory Board member, provides practical advice about how to present immunogenicity-related information in regulatory dossiers, with a particular focus on a model for an Integrated Summary of Immunogenicity to be submitted in the marketing authorization application for novel biopharmaceutical products in ICH regions (EU, USA and Japan) in the latest publication of Bioanalysis.

A format that links the analysis of potential risk factors to a justification of the methodology applied for risk evaluation and conclusions for riskmitigation is presented as a model that can be adapted according to the weight of evidence to be submitted in support of the assessment of impact on overall clinical benefit versus risk for the particular situation.

 

 

 

If you are interested in hearing more from Paul he will be presenting at the following events in March:

DDF Summit on; Biosimilars – Regulatory Primer for Formulation Scientists 12th March in Berlin. To learn more click here.

NDAs breakfast seminar; Immunogenicity for investigational biopharmaceutical products – Start with the goal in mind, 14th March in Munich. To learn more and to sign up for the event click here.

 

 

Free Webinar – Brexit Impact: Are you ready?

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

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

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

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

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

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


Click here to book your place today!


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

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

 

 

NDA Group expands presence in Denmark

In time for the European BIO meeting in Copenhagen, global development consultancy, NDA Group, is expanding the physical presence in Denmark. With the appointment of Katja Gustafsson as Senior Consultant the company will be working closely with clients in the region. The news follows several recent announcements of the company’s expansions in Europe and the US.


Katja is joining NDA’s Professor Steffen Thirstrup, Director of NDA’s regulatory Advisory Board in Copenhagen.

Johan Strömquist, CEO of NDA Group commented: “We’re delighted to be joined by Katja in Copenhagen, further reinforcing our commitment to Nordic life science and the incredible development that we see in the Medicon Valley region. Even though the Nordics is the cradle of NDA and we’ve had a strong presence in the market through our Stockholm office, there is nothing like having feet on the ground when it comes to serving our clients.”

Consultant and client interface

In her role as Senior Consultant, Katja will work closely with companies in the Copenhagen / Malmö / Lund region and act as client liaison, in addition to serving the need for professional regulatory development advice. Together with Prof. Thirstrup, Katja will ensure that the region’s clients gain access to the wealth of intelligence, advice and support represented by the capabilities of the NDA Group. Both Katja and Prof. Thirstrup will be available to meet with companies at the BIO Europe meeting in Copenhagen 5th– 7th November.

Prof. Thirstrup commented: “Copenhagen is my home and I’m delighted to see the vibrant activity in the region. With the expansion of NDA’s capabilities in Denmark we will be able to support the region even better in its growth and innovation. This can only be good news for patients.”
Katja joins NDA from Ferring Pharmaceuticals, where she has spent the last 18 years in different roles in Global Regulatory Affairs. Her previous role was as Associate Director in Life Cycle Management and Urology. Katja has been regulatory lead and team member for several development projects and driving global submissions and approvals for various pharmaceutical products. She has a MSc (Pharm) from the Royal Danish School of Pharmacy.

Margareta Busk, General Manager, NDA Nordics commented: ”Katja’s energy, professionalism and long international experience will be a great asset for the NDA team as well as for our clients. We are very much looking forward to continuing our expansion in the Medicon Valley with her support.”


NDA media contact: anna.perrin@ndareg.com

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

 

 

Recommendations from PRAC – Insights and implementation

Welcome to NDA’s free breakfast seminar Wednesday the 21st of November 08:30 – 09:30

All marketing authorization holders (MAH) are responsible for monitoring and implementing PRAC’s recommendations for signal detection, PSUSA procedures, and referrals, to name a few.

Join us to hear Shelley Gandhi, NDA’s Strategic Advisor of Pharmacovigilance & Patient Safety, share her experience and provide her insights into how PRAC works, how recommendations are decided and how these recommendations are expected to be implemented.


About the speaker

With 19 years experience at the MHRA, and her work with the European pharmacovigilance legislation, has provided Shelley Gandhi with an enormous breadth of experience and knowledge within this area. During her career she has focused on the processes that ensure the safety of medicines and related devices.


When: Wednesday 21st November 08:30 – 09:30. Breakfast from 08:00

Where: NDA Regulatory Service AB, Johanneslundsvägen 2, 2tr, 194 61 Upplands Väsby

The breakfast seminar will be an open and interactive workshop with the opportunity to ask questions to Shelley. Specific questions can also be sent in advance to frukostseminarium@ndareg.com

Please advise if you would like to discuss them openly during the meeting; otherwise you can book meetings to discuss them separately after the seminar.


Registration: RSVP by Wednesday 14th November to frukostseminarium@ndareg.com

Contact: Denise Strömquist, Client Relations, Nordics, +46 (0)8 590 778 00 or email frukostseminarium@ndareg.com


Welcome!