Key steps to proper management of CMC with rare disease products

Meeting requirements for Chemistry, Manufacturing and Control (CMC) is complex for any drug developer, but is even more so in the rare disease space. These challenges are further compounded for non-EU sponsors seeking to initiate clinical trials in Europe where the differences in requirements and expectations from their home market are often not well-understood. Manoeuvring […]

Why an experienced SME regulatory consultant is key for non-EU companies

For any non-EU small and medium-sized pharmaceutical company, having a gateway to the European market is crucial. Those companies seeking to take advantage of the European Medicines Agency’s (EMA) SME status will need to work through an EU registered third party, ideally an experienced SME regulatory consultancy. The benefit of having a consultancy for the […]

What is SME Status and how does it benefit innovators?

The European Medicines Agency offers a significant and unique benefit to Small and Medium-Sized Enterprises developing medicines for human or veterinary use. The purpose of SME Status is to promote innovation and the development of new medicines. The SME Regulation, which was established in 2005, led to the creation of the SME office which “provides […]

Data transparency: Why it’s important to post trial results on EudraCT or CTIS

While there is much focus on transparency in the Clinical Trial Regulation, it’s important that sponsors understand their data transparency obligations for trials that were conducted in the EU and that will be wrapped up under the Clinical Trial Directive. Sponsors are expected to ensure results from those studies that do not need to transition […]

Allowing time for the legal rep evaluation in the move to CTR

The Clinical Trials Regulation (CTR) has added some complexities for non-EU sponsors, including with regard to the role of the legal representative. Sponsors who are not based in the EU must appoint a representative within the EU to act as a legal representative. While this has always been a requirement, under the CTR the legal […]

Understanding what’s required to work in CTIS

Before making a submission to the CTIS, sponsors transitioning clinical trials previously approved under the EU Clinical Trials Directive must take several preparatory steps. These can be time consuming and are not always well-understood so it’s important that you take time to prepare yourself before making the transition. To get started, you will need to […]

The multiple steps involved in the transition to CTR

There is a lot for sponsors to think about before they can even start transitioning their clinical trials to the Clinical Trial Regulation (CTR) and accessing the various portals. First, there are practicalities of how they will manage their ongoing studies to ensure they are suitable to transition to CTR. While this is something most […]

Liability or life-saving: The risks associated with IVDs in personalised medicine

Over the past 10 to 15 years, there has been a steady trend towards personalised medicine, particularly in the field of oncology. This has led to increased use of in vitro diagnostic products, or investigational IVDs, to determine the right medicine for the patient. It is worth noting that personalised medicine is not a new […]

It’s a new, more costly world for medical devices – preparation is key!

The medical devices and in vitro diagnostics world has changed significantly under much tighter regulations. One standout example is the Medical Devices Regulation (MDR), which was adopted by the European Parliament and the Council in 2017. The Regulation states that medical devices can be placed on the EU market under CE certifications issued until 26 […]

More work, fewer resources and broad uncertainty put pressure on notified bodies

When the Medical Devices Regulation (MDR) came into force, many notified bodies decided not to pursue accreditation. Others are still waiting to get approval. That has meant far fewer notified bodies to perform assessments and more experienced medical device reviewers retiring or moving to industry. The challenge for notified bodies is compounded by the fact […]

Why navigating the MDR is creating uncertainty for many smaller manufacturers

The Medical Devices Regulation has created some turmoil for manufacturers, particularly smaller companies, as they try to navigate a host of new requirements. Devices that previously would not have had to undergo conformity assessments with the notified bodies may now be classed differently and require assessment. That adds an extra layer of cost and complexity, […]

European Parliament’s approval of MDR delay brings relief but questions remain

The European Parliament’s approval of the proposal to delay the Medical Device Regulation transition period brings some reassurance to the industry. In February 2023, the Parliament voted by an overwhelming majority to adopt the European Commission’s proposal to delay the transition. The delay extends the deadline for higher risk class III and class IIb devices […]

The reasons, risks and benefits of changing your legal representative

Under both the Clinical Trials Directive and Clinical Trial Regulation, changing your legal representative is considered as serious as a change of sponsor, and involves a substantial amendment. The reason for this is that the liabilities of both the legal representative and the sponsor are tied to one another. For companies that have selected a […]

Efficient, effective, independent: Getting the most out of your legal rep

For any study conducted in the EU by a non-EU sponsor, there is a requirement to have a legal representative. While you can choose to appoint a different legal representative for each study, there are efficiencies of scale to having the same representative across them all. However, if you select to use a legal representative […]

IDEA Origins and the Legal Representative

Why IDEA set out to provide sponsors with a trusted, independent EU legal representative While the Clinical Trials Regulation has clarified the role of the legal representative, IDEA Regulatory’s founder Dr Francisco Harrison recognised early on that it was more complicated and involved than many sponsors and CROs interpreted it to be.  Having supported clients […]

Start with the end in mind: Planning your clinical trial strategy early on

It has become more important than ever to start thinking and talking about your drug development plan as early as possible. From more complicated protocols and adaptive design to electronic data capture and electronic patient reported outcomes – new technologies are creating opportunities but also adding to the complexity of a clinical trial design. That […]

Keeping your EU submissions moving with our Regulatory-on-Call service

Despite having talented teams, small companies are forced to work with limited resources and access to specific expertise. They often lack the resources and experience in navigating specific regulations, the nuances of working in different countries and have limited time to spend on regulatory intelligence. Working with a regulatory expert who is familiar with your […]

Balancing risk and reward: The cost of providing legal representative support

When regulations change there are always a few aspects where the careful reader finds themselves asking “did they really mean that?”. In that vein there is a somewhat ominous clause in the new Clinical Trial Regulation, at least from the point of view of the legal representative. Article 74 of the regulation states: “Such legal […]

Speaking up: How a conversation with the regulators can set the stage for success

There is very often a them-and-us mentality when it comes to how biotech companies think about the regulators. New companies, in particular, tend to view what is said by the regulators as prescriptive and are afraid to speak to them or get the scientific advice that is available. However, if they did take time to […]

Navigating a complex regulatory landscape for IVDR

The In Vitro Device Regulation (IVDR) comes into force on 25 May 2022, which means companies must start complying with the regulations. While we’re seeing many companies have aspects in place, there are many issues that they may not have considered. For example, if you’re using a biomarker assay in your clinical study, has it […]

Report study results or face a big stick: the pros of regulatory penalties

Reporting your clinical trial results is both a legal requirement and a moral imperative. Whether your product failed early on, failed in later-stage trials, or was successful, the data from those trials could be transformational. Until recently, though, companies faced few consequences if they didn’t report their results. In April 2021, the FDA threatened to […]

The Importance of QMS and Vendor Oversight for Non-EU Sponsors Under The CTR

Good clinical practice is well-entrenched in global biopharmaceutical companies, and almost every sponsor company adheres to the Declaration of Helsinki with regards to following ethical principles in their medical research. For US companies, the standards established by the Food and Drug Administration (FDA) are very much in line with what is required in the European […]

Independent Oversight: Why It’s a Risk to Have Your CRO as Your Legal Representative

In theory, there is no reason and nothing in the Clinical Trial Regulation (CTR) that prevents a contract research organisation (CRO) being named as the EU Legal Representative by a Sponsor. However, it does beg the question as to whether there would be a conflict of interest, since what it means is the sponsor is […]

Brexit Poses Challenges for Medical Devices Companies as UK Goes Its Own Way

While it has been nearly two years since the UK withdrew from the European Union, much still remains uncertain about the direction that the Medicines and Healthcare product Regulatory Agency (MHRA) will take with its requirements. Will change happen, and if so when? It’s a bit of a baptism by fire with the regulators only […]

Asking the Right Questions: Staying Ahead of the CTR and MDR with the EU Legal Representative

The EU legal representative plays a unique role in the life sciences industry and requires a distinctive skill set – with a background in regulatory affairs and quality assurance, but from inside the good clinical practice (GCP) sphere. Now, with the added complexity of the Clinical Trials Regulation and the Medical Devices Regulation, the role […]

The QA to the Sponsor’s QA: Why the Legal Representative Should be Separate from the CRO

The role of the legal representative for clinical studies – both in pharmaceuticals and now in medical devices under the EU Medical Devices Regulation (MDR) – might be described as quality assurance of the sponsor’s QA role. In future, under the new EU Clinical Trials Regulation (CTR) and MDR, the legal representative will be responsible […]

Why “Ensuring” the Sponsor’s Compliance is Not the Right Role for CROs

The Medical Devices Regulation will bring massive and rapid change to the industry. Not only must companies adjust to new clinical investigation requirements, but they will suddenly find themselves in need of a number of new experts. Medical device companies wanting to run clinical investigations of their products in Europe will need a qualified person […]

This Changes Everything: MDR and the Role of the EU Legal Representative

The medical devices industry has changed dramatically in recent years. Historically, medical devices had more in common with product manufacturing industries such as aviation, which meant testing and standards were very engineering focussed: Is the product built robustly? Are the materials adequate for their intended purpose? Will the product and materials last? While those standards […]

Getting Ready for the Clinical Trial Regulation

Blog series: Maintaining Regulatory Momentum in the Covid-19 era: It’s been a difficult year for many biopharma companies and perhaps even more so for clinical research organisations, with Covid-19 forcing the postponement or even cancellation of many if not most clinical trials.  It is however important to keep a finger on the pulse of the […]

Medical Devices Back in the Spotlight

Blog series: Maintaining Regulatory Momentum in the Covid-19 era: Preparation for the new regulations  The global disruption caused by the pandemic meant regulatory plans for 2020 needed to be delayed to give the life sciences industry and authorities breathing room. However, it is important to keep up to date with imminent regulatory changes that will […]

Data protection to take centre stage in 2021

Blog series: Maintaining Regulatory Momentum in the Covid-19 era: In early December, the EMA announced that it had been hit by a cyber-attack and that documents related to the Pfizer-BioNTech Covid-19 vaccines had been accessed. The agency issued a brief announcement after the attack to say a full investigation had been launched but provided no […]

Orphan Unknowns: What the MHRA Guidance Will Mean After Brexit

Blog series: Maintaining Regulatory Momentum in the Covid-19 era: Developers of orphan drug products face some important changes with the approval process in the UK after Brexit. To help companies prepare, the MHRA published new guidance: “How the MHRA will manage orphan medicinal products from 1 January 2021 in Great Britain (GB)”. While most of […]

Why Transparency in reporting negative results is key for public trust

A recent perspective piece published in the New England Journal of Medicine highlights an issue that I have long felt reflects poorly on the industry, and that is public mistrust in ’Big Pharma’. This issue has come to the fore with the race to bring a Covid-19 vaccine to market. While the authors talk about […]

Navigating Clinical Trials: The Complex Roles of the Data Protection Officer and Legal Representative

Tamsyn Frost (IDEA Regulatory) and Xavier Gobert (MyData-Trust) New laws and regulations present challenges to all businesses, but the way they impact clinical trials is especially complex. When the GDPR was introduced in 2018, it was designed with broad data protection in mind, but these requirements don’t apply neatly to clinical trials. The GDPR states […]

Be Systems and Process Aware: Records, e-Signatures and Validation

Over the past couple of blogs – Be Prepared and Understand the eCTD challenge – I have talked about the challenges pharmaceutical companies with limited marketed product experience face, particularly when seeking to bring products to the larger markets. An important consideration is to understand the expectations and preference of the authorities. Regulators want to […]

Understand the eCTD challenge: The what and when of dossier submissions and changes

As I discussed in my last blog, solid document management is paramount for successful regulatory submissions in the larger markets. In the majority of major markets submissions must be done electronically through the electronic common technical document (eCTD). In principle, the eCTD is a simple structure to allow the dossier of all those documents created […]

Be Prepared: Document management for the uninitiated – Part 1

The push to conduct clinical trials and bring products to market in the EU often catches companies with limited marketed product experience off-guard. As they progress through the clinical development phases, they develop a large library of completed and approved documents which comprise their dossier. There is an assumption that this emerging monument to effort […]

Start with the end in mind: how to build a solid Target Product Profile

Start with the end in mind. That is one of the most important considerations for companies seeking to conduct clinical trials in the global market. A common mistake made by smaller biotech companies — and even older companies that have not done a lot of R&D in recent years — is to think only of […]

Just how patient-centric is R&D? The desperate need for accessible rare disease data

There is much talk today about patient-centricity in the clinical trials, from discussions about how to ease the burden for patients, to how to empower and engage patients. But to my mind a central aspect of patient centricity that is poorly addressed is how patients and advocacy groups get better access to medical research, or […]

Why the Hiatus to Clinical Trials Amid Coronavirus is a Chance to Improve Processes

It’s hard to consider a bright side to the current coronavirus pandemic the world finds itself in, but the delays and temporary suspension of many clinical trials do present an opportunity to step back and address gaps in many trial processes. The question, then, is how can you use this time efficiently and effectively to […]

Quis custodiet ipsos custodes: Who will hold the sponsors to account?

The long-awaited new Clinical Trials Regulation looks set to be introduced in 2020. Initially adopted in 2014 and due to go into force soon afterwards, it was held up by technical difficulties with the database, which is necessary to underpin the new environment. The latest testing suggests these problems have been resolved. The new regulation […]

Silver Linings: How Regulatory Learnings from the Covid-19 Pandemic Could Bring Future Improvements to Research

I’m a huge believer that even in the very worst of circumstances, there is always a silver lining (or two) to be found. We  have already seen some of these playing out during this pandemic in the many ways people are coming together to spread hope and goodwill between and across communities. One positive that […]

Two in one go: Don’t shy away from joint consultation with EMA and HTAs

It’s understandable that companies tend to be focused first on getting their products approved. But that’s just part of the battle. The other part is getting your products paid for. In July 2017, EMA began offering consultations in parallel with the European Network for Health Technology Assessment (EUnetHTA). The objective is to ensure that you […]

Can you Meet GMP Requirements? The Importance of Preparing Manufacturing Data

In drug development, it’s often the area you least expect that causes the most problems. In my experience, one of the biggest hold-ups to completing regulatory submission dossiers relates to manufacturing, not the safety and efficacy data. The information needed for the Investigational Medicinal Product Dossier (IMPD) or CTD Module 3 (M3) — stability testing, […]

Lost in translation: Manoeuvring through the complex world of regulatory language

The regulatory process is complex and can be very difficult to navigate. It’s not just about gathering data and meeting milestones, but also about understanding the regulatory language. Often,  young biopharma companies come to me after completing a scientific advice procedure with the regulators and are under the impression that the agency in question have […]

Keeping everyone in the loop: why all stakeholders need to hear the patient voice

In a previous blog post (Linked here), I raised the issue of patient-centricity and my concern that it is being used more as a marketing tool than a way to build truly patient-centric products. To shift that thinking, there needs to be broad commitment to bringing patients into the fold at every stage of the […]

Dream big but remain focused: Why lofty goals can derail the development process

Start-ups and emerging biotech companies often start out with lofty goals about what their products can do. That’s very noble but it’s also an easy way to run out of money halfway through the development process. While it’s good to have long-term goals, success depends on developing a well-defined target product profile (TPP). Start with […]

Less talk more action: Building real patient-centricity from trial design

Patient-engagement or patient-centricity is now widely spoken about, but it’s rarely brought into the process of product development early on. As a regulatory consultant, my role is to fulfil the objectives of our clients as I help them navigate the clinical trial approvals process and I strongly believe that this would be a good point […]

IDEA Regulatory Ensures Continuity for Clients Amid Coronavirus

The global coronavirus pandemic, or COVID-19, has brought about both a health crisis as well as personal and business uncertainty. Yet bringing new life saving and life changing products to market remains a pressing priority. Patients are waiting for cures and their needs cannot be postponed. As a leader in providing regulatory affairs expertise and […]

Is Brexit putting your clinical trial at risk?

FAO: ALL UK-BASED SPONSORS OF EU CLINICAL TRIALS If you are a UK-based sponsor with a clinical trial in the rest of the EU, Brexit could have a profound impact on the legal status of your study. Clinical trials in the EU are governed by the Clinical Trials Directive 2001/20/EC. According to Article 19 of […]

Medical Device Regulation: Leveraging expertise from Pharma

When the Medical Device Regulation (MDR) and In-Vitro Diagnostic Device Regulation (IVDR) transition was launched earlier this year, medical device manufacturers were faced with a steep mountain of regulatory, legal, process and resource challenges that would need to be climbed in order to introduce new and continue supplying existing devices for use in healthcare. In […]

Applying for Orphan Drug Designation: Should the USA always be your first regulatory target?

In this article we examine 5 common myths surrounding FDA vs. EMA applications for Orphan Drug Designation applications. Download PDF: Microsoft Word – ODD – Should the USA always be first regulatory target