TOPRA 2025

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The Organisation for Professionals in Regulatory Affairs

Our vision To be the leading global organisation for all professionals in healthcare regulatory affairs Our mission To represent and promote the profession of healthcare regulatory affairs globally. To do this we provide exceptional support, world-class education and definitive accreditation for all regulatory professionals, and celebrate excellence in regulatory practice.

Belong to a vibrant global regulatory community

Engage share and learn from your peers

Develop your professional competence

Advance your career

Refresh your knowledge on the latest regulatory developments

Benefit from membership privileges

Contribute to the development and recognition of the profession globally

Access authoritative information and high-quality learning opportunities


TOPRA 2025 – Aspire | Adapt | Achieve

Let us go forward together

TOPRA 2025 embodies the Board’s strategic plan; demonstrating our commitment to being as relevant, vibrant and strong, if not more so, when we come to celebrate our 50th anniversary in 2028. Fostering a healthy appetite for change – evolution rather than revolution – gives impetus to building on our highly regarded heritage of support for skills development and driving excellence internationally; adapting in response to a complex, rapidly evolving environment; and continuing to be a fit-for-purpose membership body, focused on enabling and promoting the healthcare regulatory profession of which we are all rightly proud. Setting standards and providing learning experiences for professional development is TOPRA’s raison d’être . We provide neutral settings for regulators and regulated alike from all sectors (human and veterinary medicines, devices and diagnostics, biologicals and advanced therapies) to come together, share expertise, and develop mutual understanding. Growing a dynamic and diverse community of practice, with ‘must have’ signifiers of professional standing (such as apprenticeships, MSc, RSci, CSci, MTOPRA, FTOPRA), ensures a sustainable pipeline of regulatory professionals and potential future leaders.

Professionalism is the collective achievement of all that stems fromour members’ daily undertakings – applying regulatory science, delivering for stakeholders and, ultimately, enabling patients to have treatments they need

Professionalism doesn’t just originate from our seminars, summits or symposia. It is the collective achievement of all that stems from our members’ daily undertakings around the world: applying regulatory science, delivering for customers and, ultimately, enabling patients to have treatments they need. All who share an interest and similar passion for our core values are encouraged to join the TOPRA community: extending out into a vast and multi-faceted stakeholder network, building and sharing collective regulatory knowledge for common good. Steve Hayes | TOPRA President 2020

Our Community


Professional Excellence


Nurturing Innovation and Science


Engagement and Connectivity


Global Reach and Value


Sustainability and Resilience



TOPRA 2025 – Aspire | Adapt | Achieve

Our ambition

• Be the recognised hub for regulatory expertise, bringing together theory and practice in

regulatory innovation, collating and providing evidence-based advice, guidance and support to regulators and regulated alike • Enhance the value and impacts of regulatory

H e a l t h c a r e p r a c t i t i o n e r s

R e g u l a t o r s

Medical devices & IVDs


professionals, promoting high quality education and professionalism

G o v e r n m e n t

S u p r a n a t i o n a l )

• Raise awareness of issues affecting regulatory affairs through thought

( i n c l u d i n g

P a t i e n t s

P r o f e s s i o n a l E x c e l l e n c e S u s t a i n a b i l i t y

Pharmaceuticals (Rx & OTC)

G l o b a l R e a c h & V a l u e

E n g a g e m e n t &

leadership, stimulating debate and promoting dialogue on the regulatory environment • Extend our international community, expanding the profile of our membership and driving

C o n n e c t i v i t y


R e i m b u r s e m e n t )

p r o f e s s i o n a l

& R e s i l i e n c e

( H T A , P r i c i n g &

O t h e r

P a y e r s

b o d i e s

& S c i e n c e N u r t u r i n g I n n o v a t i o n


best practice globally by promoting shared

Biologicals & ATMPs

professional standards, supporting new agencies, helping to develop new markets, and growing a dynamic and diverse professional community

P h i l a n t h r o p i c o r g a n i s a t i o n s

A c a d e m i a

• Increase awareness and value of TOPRA as the global forum-of-choice for professionals in healthcare regulatory affairs.


TOPRA 2025 – Aspire | Adapt | Achieve

Our Community

Healthcare regulatory affairs is a multi-faceted community connecting at all levels: locally, nationally and across the world. TOPRA engages across a broad membership and connects with numerous stakeholder networks involved with medicines, medical device and diagnostics regulation, providing a unique perspective and offering a neutral forum for stakeholder discussions. Our members are the core to TOPRA’s community. Extending beyond our membership is a broader community of individuals from agencies and industry and other stakeholders who engage with TOPRA – as speakers, authors, advisers, supporters and patients. Their collective knowledge, expertise and experience is invaluable to our members. Conversely, our members’ knowledge, expertise and experience is beneficial to our stakeholders in the endeavour to support healthcare providers and practitioners deliver better, safer, more innovative treatments to patients. At a global level, the increasing convergence of regulatory affairs offers opportunities for regulatory professionals to get involved in international partnerships, both governmental and non-governmental, contributing to the widespread improvement of public health and helping build regulatory capacity in developing countries. Role of regulatory affairs Regulatory affairs professionals are at the forefront of protecting the public, helping to ensure that any medicine, medical device or in vitro diagnostic brought to the clinic is safe and effective. Regulatory affairs covers both regulators and regulated – whichever perspective they have, the priority is ensuring innovative treatments go through the regulatory process in a timely way, establishing benefit–risk profiles, meeting unmet patient needs, eg, for rare conditions, and access. Regulatory professionals contribute to all stages of the process – from initial clinical trials, to large-scale global clinical development, registration and approval to post- marketing surveillance of drug products and devices.

At the heart of healthcare Regulatory affairs delivering safe and effective healthcare systems


TOPRA 2025 – Aspire | Adapt | Achieve

Our wider stakeholder community Development and deployment of safe and effective treatments and innovative therapies for patients involves extensive collaboration across a broad range of disciplines. As regulatory affairs professionals are drawn from a wide variety of backgrounds – eg, biologists, chemists, pharmacists, physicians, veterinarians, engineers (and many others) – TOPRA members reflect this diversity. Individually, and as a whole, the profession embraces a widespread network of stakeholders around the globe; engaging with healthcare practitioners, professional bodies, academic researchers, patient groups, and charities. At governmental level (both national and international), this extends beyond primary interactions with regulators to include payers, eg, health technology assessment (HTA) and reimbursement. All have common cause in advancing the science of medicines, medical device and diagnostics regulation.

At the forefront of regulatory professionalism In the late 1970s as regulatory affairs began its journey as a separate function in the pharmaceutical arena, people involved in delivering the registration of medicines started to come together to share experiences and develop regulatory training that addressed their specialist needs. In the UK, BIRA (British Institute of Regulatory Affairs), the precursor to TOPRA, was born. In the following decade a companion organisation ESRA (the European Society of Regulatory Affairs) was set up. Ultimately both amalgamated to form TOPRA in 2004 bringing together a significant legacy of delivering symposia, introductory, postgraduate and continuing regulatory education courses focused on European regulatory affairs. The foundation of The Organisation for Professionals in Regulatory Affairs, which incorporated a global remit, was a cornerstone of recognising the professionalisation of its membership. All members demonstrate a commitment •8


TOPRA 2025 – Aspire | Adapt | Achieve

profession as a whole and for the wider benefit of society and public health. TOPRA prides itself on providing an ideal forum in which neutral discussions about regulatory issues can take place, eg, on potential impacts and consequences of proposed regulations and guidelines. These discussions can take place on a small scale within Roundtables, or at a higher level in Summits, the Annual Symposia or single issue- focused Horizon conferences. TOPRA does not give an opinion on consultations, but as a professional association we provide a means for individual regulatory professionals to share their observations and get their voices heard, especially if they are not able to comment on public consultations via their employers or trade associations. This neutrality and regulatory expertise enables TOPRA to work closely with allied professional groups, governmental and regulatory authorities at national and international level.

TOPRA members’ knowledge, expertise and experience is beneficial to our stakeholders in the endeavour to support healthcare providers and practitioners provide better, safer and more innovative treatments to patients. to professionalism by signing up to a Statement of Values, and undertaking continuing professional development. Members are also eligible to apply for professional registration as Chartered Scientists or Registered Scientists as TOPRA is an awarding body for the Science Council. A neutral forum TOPRA membership is individual and personal. Our members come from all backgrounds, specialties and types of organisation. They come together not only to develop themselves but to contribute to the development of the

CASE STUDY Embracing the patient perspective

Among the many examples of regulators and drug developers engaging with patients in order to develop better medicines is an initiative by Swissmedic (Swiss Agency for Therapeutic Products). Cordula Landgraf is the agency’s former Head of Networking and has led the project through the pilot stages to the establishment of the Patient and Consumer Group as a permanent standing group in February 2019. She described the group’s development at a TOPRA Summit. Swissmedic increased patient involvement in its processes in a step-by-step approach. Transparency and training are key to enabling patients and consumers to become more involved in decision-making processes. The initiative started in 2016 with training of a heterogenous group of patients in order to help everyone get to a similar level of understanding. The first input was into patient information leaflets (PILs) assessments with the group feeding back to companies applying for marketing authorisations before submission to Swissmedic. Plans for the standing Patient and Consumer Group include helping to review the SwissPAR lay summary and safety communications.

Cordula Landgraf


TOPRA 2025 – Aspire | Adapt | Achieve

Professional Excellence

Our commitment • Listen to the regulatory and stakeholder community and explore the changing needs of regulatory training • Ensure our education remains relevant and adapts to the needs of regulatory professionals • Advocate and develop professional registration through access to Chartered and Registered Scientist status • Offer accreditation to academic institutions with regulatory affairs related undergraduate and postgraduate modules and degrees • Align our membership categories with standards in the TOPRA Competency Framework, reaffirming MTOPRA as a valued badge of professional expertise and a tangible commitment to continuing professional development • Recognise and share excellent regulatory practices within the healthcare regulatory profession and beyond On the right track TOPRA offers training and professional development activities across the entirety of a regulatory professional’s career. TOPRA has evolved a pathway of educational support – both face-to-face and online – for regulatory professionals, whether exploring options or a seasoned influencer, whatever their background, specialty or organisation.

Raising the bar An ethos of continuous improvement

TOPRA 2025 – Aspire | Adapt | Achieve 10



For regulatory leaders and opinion-formers engaging in discussion, debate and networking opportunities across the regulatory profession and at a global level INFLUENCE

For experienced professionals committed to developing their regulatory knowlege, expertise and management competencies CONSOLIDATE


For those wanting to take their first step into regulatory affairs OR

For those who have recently joined the regulatory profession OR For those whose regulatory experience is outside the EU

For senior-level regulatory professionals taking on additional/ strategic responsibilities OR

For those in related disciplines who are

considering a change of career direction or who need an overview

for those looking to diversify their skills

Mapping competencies The TOPRA Competency Framework enables regulatory professionals to gauge the level at which they are working, and identify areas where they may require further support to take on more responsibility or improve their performance. It incorporates input from a wide range of regulatory professionals and educationalists and is subject to regular review to accommodate regulatory and scientific advances that require new skillsets and knowledge. Reflection and recording Continuing professional development (CPD) is not only about taking a course or a webinar. Work shadowing, reading and sharing experiences with other professionals informally are also opportunities to learn and reflect. Key to keeping up to date, developing regulatory competencies and acquiring new knowledge is recording and evaluating those activities. TOPRA’s online recording tool supports members track their CPD – and counts towards RegisteredMember status, ie, the MTOPRA designation. Setting standards for excellence in regulatory affairs and providing the necessary support for development of effective, qualified and successful regulatory professionals is TOPRA’s raison d’être.

Professional registration and parity TOPRA is an awarding body for the Science Council offering Chartered Scientist and Registered Scientist for regulatory professionals. Presence on the Science Council’s internationally recognised registers as a CSci or RSci denotes parity with other scientific disciplines. Every year, more andmore TOPRA members undertake the competency-based registration process to achieve this recognition for their level of professional practice that also provides a personal sense of satisfaction.

Through the years I have thought creatively and generated new ideas in the workplace, refined various processes to simplify the way we work, mentored colleagues and built my own capability both within and outside the profession. Applying for CSci made me scrutinise my work further and consider whether I really have what it takes to gain chartership. I have diversified my regulatory experience by being granted professional registration. Professional registration for me is a journey, a constant reminder of enhancing my experiences to maintain professional development throughout the year. Eesha Vyas, CSci Global Regulatory Submission Manager, GSK

TOPRA 2025 – Aspire | Adapt | Achieve 11

Winners of the 10th Awards for Regulatory Excellence

Celebrating excellence Since 2010, the outstanding contributions of regulatory professionals in sectors from pharmaceuticals and veterinary medicines to medical devices and diagnostics have been celebrated through the TOPRA Awards for Regulatory Excellence, which is the premier awards programme for the worldwide regulatory community. Each year nominees from industry, agencies, consultancies, academia, SMEs andmany other settings with a regulatory focus are put forward. They come from all over the world – from as far afield as Australia to Cuba. Entries are evaluated by an independent judging panel of high-profile regulatory experts and stakeholders from diverse backgrounds and experience. The Awards are not only for TOPRA members, they help set a benchmark for aspiration among the entire regulatory community. It’s a great accolade to be a finalist or winner.

We were incredibly proud to win the TOPRA regulatory excellence Award for Innovation for the Rx to OTC switch of Viagra Connect in the UK. For myself and my regulatory team to receive this recognition from our peers and professional body was such an honour. The Awards ceremony was a very special evening, and it was wonderful to hear of all the great work and advances being delivered by regulatory colleagues across our industry. Kate Stockman Pfizer, UK

TOPRA 2025 – Aspire | Adapt | Achieve 12

Nurturing Innovation and Science

Our commitment • Establish an Innovation Advisory Group to actively track scientific developments and disruptive technologies with potential regulatory impact • Ensure education and training programmes reflect scientific and technological advances, new R&Dmodels and digital health • Develop bespoke high-level conferences focused on innovation and impact More than ever, the regulatory profession needs to be alert to the nature and speed of technological, political and social change, and capable of adapting to new circumstances. The COVID-19 pandemic more than anything demonstrated this need and the speed at which the regulatory community responded and delivered support to a wide range public health initiatives was exemplary – from vaccine trials to increasing ventilator supplies. A brave newworld The pace of technological advancement is exponential. As individuals, social media has extended our networks from local to global; the internet and digitalisation has made immediate access to the most up-to- date information routine; advances in healthcare technologies andmedical research have saved lives andmet formerly unmet health needs. Our expectations have also been raised – the pressure is on to deliver innovative medicines and treatments safely and effectively as soon as possible.

At the cutting edge of healthcare Embracing disruptive technologies, medical discoveries and real-time data

TOPRA 2025 – Aspire | Adapt | Achieve 14

Regulatory professionals are striving to meet these challenges and adapt against a backdrop of constant change. TOPRA acts as a conduit to help regulatory scientists to explore, consider, discuss and share potential solutions and best practices – always with the benefit of the patient in mind. Innovation and impact summits

Each year leading regulatory professionals and interested stakeholders gather together at the TOPRA Summit to focus on issues affecting regulatory science at a top level from a multidisciplinary perspective. Discussions are wide-ranging and the ideas are distilled into reflection papers that are then sharedmore widely. Topics debated include: challenges to the regulatory paradigm and increasing the sustainability of medicines development; the importance of regulatory convergence in improving access to healthcare; the impact of disruptive technologies, using digital technologies to validate and gather health data; and the collection and analysis of digital data in real time to support better regulatory decision-making. The establishment of an Innovation Advisory Group – tasked with horizon scanning for technological and procedural advances as potential short, medium and long-term disruptors of established regulatory practice – will enable us to remain abreast of


Forsome time now,bigpharma hasbeen

adeviceasa consistentlyperforming system, manyAI systemswithalgorithms thatevolve and sharpenon theirownarealready in development. The FDAhasclearedmedicaldevices that relyonAIalgorithms for themarket,but so far, theagencyhaspassedonlydeviceswith “lockedalgorithms”— those that remain the sameuntil theyareactivelyupdatedby the manufacturer. Withmany futureAIapplications, the

time togetprepared.As innovationprogresses, sowill thedemandson regulatoryaffairs,and particularly initially thosewithin themedical technology sector. AIwithinmedtech lifesciences AIhit theheadlines in theUK in summer2019 when itwasannounced that£250mwillbe spentonboosting the roleofAI in thenational health service (NHS). 2 UKHealthSecretaryMatt Hancockstated thatAIhadenormouspower to improve care, save livesandensuredoctors hadmore time to spendwithpatients.Emerging technologiesareplayingakey roleacross the value chain, fromdrugdiscovery through to patientcare.

preparing for the AI revolutionand manyareeither in collaborationwith, orhaveacquisition


Potential impactofAIon regulatory affairsmedical technology roles

of,emerging technologies

accuracyofoutput informationwillcontinually improveasusage increases.Thiswillproducea perpetuallydynamic systembydesign.Without anyprior technicalknowledge, itwillbehard fora regulator toassess the safetyandefficacy ofaproduct.Notonlywill regulatoryaffairs leadersneed tounderstand the concept fully, theywillneed tobeable toexplain itaccurately to the regulatorybodies.As such,an increasing levelof technological literacywillundoubtedly becomea core competency for regulatory affairsprofessionals in the future. However thatdoesnotmean regulatoryprofessionalswillhave

intervention.AI isanemerging technology that isbeingexplored inevery facetof pharma, fromdrugdevelopment todiagnosis

A rtificial intelligence (AI) is rapidly emergingwithin thepharmaceutical industryand is likely to transform themedicinal technology environment in the imminent future. Although suchadvancesarewidelywelcomed – since theywillultimately improvepatient outcomes– it isnotyetunderstoodhow they willaffect thevarious teamsacross thedrug pipeline, including regulatoryaffairs. It isclear thatadvances inAIwillbringnew challenges

andevenpatientcare.Theglobalmarket forhealthcareAI solutions isexpected to increase froma reportedUS$1bn in2017 to more than$34bnby2025. 1 Over thepast 12 months, thepacehasgatheredconsiderably, withvarious “fast-tracked” FDAapprovalsof AI-based technologies, further cross-industry collaborations,and raised interest from investors.New technologies,newpartnerships andnew ideasarebeing reported in the mediadailyand,although thesearewelcome developmentswith clearbenefits,what is lessunderstood ishowmachine learningand automationwillaffect traditional roleswithin pharmaand life sciences, including regulatory affairs. AI is thereforegeneratingbothexcitement andadegreeofuncertaintyacross the life sciences industry.Despitebeing yearsaway frommaking itsgreatest impacts,now is the

For some timenow,bigpharmahasbeen preparing for theAI revolutionandmany areeither in collaborationwith,orhave

Advances in artificial intelligencewillbringnew challengesanddemand new skillsets. In this article, insightsare shared from companiesand candidates in regulatory affairs,particularlywithin themedical technology sector

acquisitionof,emerging technologies.There areanumberofways inwhichAI isbeing–or willbe–usedand the scaleandbreadthof potentialapplications isastounding.There is scopeateach stageof thedrug lifecycle, from discovery through to researchandanalytics, and,most interestinglyofall, improved outcomes forpatients. Forexample,BioMind’s artificial intelligencemedical systemwas able tooutsmarthumanexperts inpredicting theexpansionofbrainhaematomas,making correctdiagnoses in87%of225 cases in

tobecomeexperts insoftware developmentandcybersecurity themselves.Thekey isa multidisciplinaryapproach thatbuilds relationshipswith peoplewhoareexpertsbut whomaynotbebestsuited to navigatingcomplex regulatory challenges,andpossessing sufficientknowledge to beable toask the right questionsandknowwhere

anddemandanew setof skills,butwhat is lessobvious isexactlyhow specific roleswill beaffected. In thisarticle, insightsare shared frombothhiring companiesandcandidates in regulatoryaffairs,particularlywithin themedical technology sector. The riseofAI Artificial intelligence (AI)within life sciences refers to the systemof interconnectedand automated technologieswhich can function autonomously,with littleornohuman

AKEELHUSSAIN , RegulatoryAffairsRecruiter, Talentmark,UK

about 15minutes,versus66%accuracy in30 minutesbya teamof 15doctors. 3 Currently,a wearabledevice thatusesmachine learning to remotely trackandanalysemultiplevital signs hasbeen clearedby theUSFDA,expanding the scopeofhomemonitoring systemswhich willhelp tokeepmore chronically illpatients outof thehospital.MayoClinicphysicians andengineersaredevelopingAI foravariety ofclinicaluses, suchas screeningpatients forearly signsofheartdiseaseandwarning peoplewithepilepsyaboutoncoming seizures. ElonMusk’sNeuralink 4 isdevelopingabrain implantwhich theyhopewill read thebrainsof paralysedpatients.Theoutcomesof theseand similardevelopmentscouldbe far-reachingand evenhelp todiagnosepatientswith “hidden” symptoms. Impacton regulatoryaffairs

theyneedspecialistassistance. Regulatoryprofessionalswill need toexpand theirnetworks to include technicalexperts inawide rangeoffields. Increasingly,muchof this expertisewillbe foundvia

Transparency iskey ina regulatoryenvironment duringassessment, yetmostAIapplicationsare a “blackbox”, lacking clarityandencompassing agamutofcomplexities.AIapplicationscan producevalidconclusions thatare counter- intuitive to thosewhichhuman teamsmay derive.Comparedwitha standardmedical device,whereby regulators reviewandclear




FALKEHMANN , Chairof theEuropeanMedicinesAgency’s InnovationTaskForce (ITF) ,describeshis role, theachievementsandchallengesof the task force,andprovides tipsandadvice toorganisations I t is easy for us to focus on the immediate history of innovation in medicine. Indeed,wewouldguess that, ifasked todescribe thefirst truly novelapproach topatientcare,anyoneworking in regulatoryaffairs today would generally only think back around 30–40 years. However, innovation is a progressive path, and one which builds up as new technologies develop.Would James Blundell, who performed the first recorded human blood transfusion back in 1818, ever have imaginedwe would see a time ensure thatwekeeppacewith currentdevelopment,and ensure consistentcontactwith stakeholders throughmeetings, workshops,andcommentonupcomingguidance.Wehave to listen, takenoteof innovationandassess the impactof the challenges theymaypresent fordevelopersand theexisting regulatory system. It is the stakeholders (academia, researchand developmentcentres,andcompanies) thatbring innovation to theagency,andwe currentlydealwitharound80 requestsper year.The ITFhas toassesswhat’s importantand this involves both internalandexternaldialogue throughout itsnetwork, so weworkwith regulatoryagenciesacrossEuropeandwith the24 national innovationoffices to createa collaborativeenvironment thatenablesus to improve thedetection rate for innovation.At theendofNovember2019, theEMAheldaworkshopon the regulatory science strategy to2025 (seep15)andakeymessage thatemerged from thatmeetingwas thatweneed to facilitate innovation in theEUproactively, so thiswillbeakey focusover thenextfive years. Q: Howcan industrycontribute toknowledgesharingwith regard to innovation? A: Wemustnot forget that innovation isnot justan industry activity, theEMAalsohasastakeholderdivisionand liaiseswithpatientgroups,academicgroups,healthcare professionals, individualsandotherparties. I think thereare two recommendationsonknowledgesharing:firstly,weneed toencouragedevelopers tobringnoveldevelopments toEMA ora local innovationofficeassoonaspossibleand regardless ofdevelopmentstage.Secondly,weneed toencouragevarious stakeholders tocommentonEMAdocumentssuchasdraft guidanceandattendworkshops.TheEMAalso tries tocollect informationviabusinesspartnermeetings,andundertakeshorizon scanning to lookatwhatmightbe landingonourdoorstep in the nextfive to tenyears.We likedevelopers tobeproactive. In thepast two years, theEMAhasestablisheda research anddevelopment stakeholderplatformwhere itcollaborateson different topics, suchas theEUMedicalDeviceRegulation.This providesagoodopportunity toengagebut,again,weencourage early interactions.Weknow thatearlydialogue,especially through scientificadvice, increases the chancesofa successful marketingauthorisationapplication. Q: Howdoes the ITFensurediscussionson innovative medicinesand technologieswithstakeholdersareshared within thedifferentgroupsat theEMA togeneratenew guidelinesorcontribute to revisionsof the regulatory paradigm? A: Ibelievenetworkingandcollaborationareextremely important, in linewith theoldChineseproverb: “Ifyouwant to go fast,goalone; ifyouwant togo further,go together”.The ITF’smain task iscollaborationand the sharingof information within theagencyandwith the innovationnetwork.Wehave theCommittee forHumanMedicinalProducts (CHMP)monthly meetings inwhichwe shareall requests the ITFhas received andassesshowvaluable these requestsare,what impact they haveandhow canwebest facilitate this information.Once this hasbeen identified,we target specificcommitteesandworking parties thathave the tailoredexpertise tohelpwith requests to the ITF fromapplicants.We circulateallnew relevant topics to

the innovationnetwork for considerationandwill furtherengage with thenetworkviaactivities suchaswebinars,meetings,or workshops.Minutesofmeetingsarealways sharedwith the CHMP, theEMA’sother committees,workingparties,expertsand attendees. It is important to remember that fullconfidentiality is guaranteedduring thisprocess. Regardingnewguidelinesor reflectionpapers,we takea cautiousapproach,because it’s important thatnewguidelines arenot so restrictiveas toprevent innovation.Weask three questions:does somethingneed tobedone,how canwebestdo it,andwhatwill the impactbe?We then reachout to the relevant experts,assesscurrentguidelinesanddiscusswhether these need tobemodified. Ifweneed tomake changes,weuseour network,make recommendationsand runworkshops todiscuss upcoming changes.

30 | REGULATORYRAPPORTEUR | Vol. 17,N03,March2020 Facilitating innovation in medicines

BY JOANBOREN , ConsultantEditor, RegulatoryRapporteur ,and JONATHANMONTAGUE ,RegulatoryAffairsAssociate,BoydConsultants Ltd,UK

Q: Couldyou tellour readersabitaboutyourbackground, whatattractedyou to the regulatoryarenaandhowyoucame join theEuropeanMedicinesAgency (EMA)? A: I’mamedicaldoctorby training,withaPhD inexperimental medicine.Before joiningEMA, I spenta coupleofyearsworking inhospitals, including in intensive careunits. Itbecameapparent during this time thatmyworkwashavingan impactononlya limitednumberofpatients.Then, followinga short stintat the WorldHealthOrganization inGeneva, IbecameawareofEMA in London.Theorganisation interestedmeas itoffered the opportunity towork inamulticulturalenvironmentandmakeuse ofmyexperience inmedicineand time spent treatingpatients. Iwas interested in the ideaofcontributing to researchand development through theagency,as I couldhavean impacton innovationand scientificgrowth.This isagreat job forahungry scientist,ashardlyaweekgoesbyatEMAwithoutadiscussion on cutting-edge science. A: Mymain role involvesmanaging the ITF’sactivities.This is amultidisciplinarygroup comprisedof representatives from throughout theagency, including legal,quality, regulatory, nonclinicalandclinicalexperts. Inmanaging the ITF, Iam the firstcontactpoint for companies. It’s important to remember that, inaddition to innovativemedicines, innovationalso applies to technologiesandmethodologiesused indrug development, sowehave to takeabroadapproach.Once we identify the relevanceandpotentialvalueofa requeston innovation,we involveourEU regulatorynetworkofexpertsand engage in furtherdialogue,organisinga face-to-facemeetingor teleconference tobrainstormon thedevelopmentandaddress issues.Wealso lookatmeetingminutesandanalyse trends to identifyareasof innovation thatare then carried forward into workshopsandother fora. I reallyenjoybeing involved innovel, excitingdevelopments,discussing cutting-edge sciencewith internationalexperts,andworking inamulticulturalenvironment inwhich I can interactwith innovatorsanddevelopersofnew productsand technologies. Q: Howdoes the InnovationTaskForce (ITF)stayaheadof cutting-edge technologiesandemerging techniques? A: The ITFaims tobe thefirstpointofcontact fornewproducts, innovativemethodologiesand technologies.Our role is to Q: Whatdoesyourcurrent role involve,andwhatareyour favouriteaspectsof this role?

Q: Does the ITFpropose theconductofworkshopsonkey topics forcross-discussionamong regulators,academiaand industry? A: Yes,absolutely.We recommendworkshopsbutwedon’t conduct themourselvesasabroadernetworkneeds tobe involved.Wehighlight trendsand these flow into thework programmesof the committeesandworkingparties.TheEMA workshops takeabout sixmonths toplan,andconducting them canbequite resource-intensive.Hence, the ITF is the initiating spark for furtherdiscussions,butwedon’tusually takean organising roleata later stage.Recentexamples include the EMA’sexpertmeetingongenome-editing technologies, the discussiononnanomedicines, (theEMA is thefirst regulatory agency toaddress this topic)and,more recently, theuseof digital toolsand technologies indrugdevelopment. A: Mybestadvice is tonothesitate to reachoutasearlyas possible.Weappreciateapplicantsmay think it’s “too soon” for interactionsbutwepreferearlyengagementanddiscussion,even witha telephone call.Weaim toofferan informal interaction so ITF interactions shouldbeviewedasa low-threshold,planning zone.Toooften,we’re consulted too late,whichmakes itmore difficult forus to influenceandadvise.Please reachoutearly for greater impactand input. groups,academicgroups, healthcareprofessionals, individualsandotherparties Wemustnot forget that innovation isnot justan industryactivity, theEMAalsohasastakeholder divisionand liaiseswithpatient

developments and anticipate likely impact. Regulatory science resources

Q: Whatadvicewouldyougive toorganisationsseeking input from the ITF? Innovation:newtechnologyplus anoldproblemandabig idea BY JOANBOREN , Director,RgulatoryAffairs ,AstraZeneca,UKand JULIEWARNER , VicePresident,RegulatoryAffairs ,AlanBoydConsultants Ltd,UK



Regulatory science related articles have always featured regularly in our peer-reviewed journal, evenmore so recently reflecting the increasing pace of innovation within

10 | REGULATORYRAPPORTEUR | Vol. 17,N02, February2020

Vol. 17,N02, February2020 | REGULATORYRAPPORTEUR | 11

reduced.Wemust recognise thatmost true innovations have their origins in academia, a settingnot traditionally familiarwith regulatoryexpectations or requirements andusuallywithbudget limitations.Could thisbebecause that environment supports free thinkingwithout the constraints of industry timelines and commercial pressures? Thinking of Henry Ford’s quote (“If I had asked the public what they wanted, they would have said a faster horse”) reallydoesdrivehome themessage that it isagifted fewwhohave

when the natural properties of red blood cellswould result in theiruse asnoveldrug deliverysystems,or that theywouldevenbe artificially created, dried and administered topatients? The introduction of novel approaches to the treatment of human and veterinary disease is moving at a rate never seen before, largely owing to the fast-paced advancements in scientific knowledge and improvements in technologies that are used throughout the product development spectrum, from drug discovery to new methodologies toassess clinicalendpoints. Considering that thefirstexperimentalgene therapy was administered to humans just short of 30 years ago, significant progress has been made and the field continues to evolve at blistering speed. Of course,

sufficient numbers of ideas for some to fall by the wayside and the true diamonds – really innovative concepts – to emerge. However, this is definitely not without its challenges froma chemistry,manufacturing and controlsperspective, aswehear in this issue. Although such innovators often have the technical knowledge around a concept orproduct, theymaybesomewhatdetached from the regulatory reality– inotherwords, what are the next logical steps on the path todevelopmentandwhatevidenceneeds to be generatedorplansput inplace tomake thishappen,andwhatare thedependables?

regulatory affairs. Regulatory Rapporteur continues to be a valued resource across the wider regulatory community for addressing cutting edge topics. Focus articles include real world data/real world evidence (RWD/RWE) and the implications of big data; artificial intelligence (AI) and how it can be applied in regulatory data management; digital health (eHealth, mHealth); advancedmedicinal therapy products (ATMPs); and COVID-19 related content.

It is clear that close communication betweenacademia, industryanddevelopers is required. Speaking from an industry perspective, it is our responsibility, as regulators, to increase awareness, within academia and in sponsor organisations, of the need for and value of early regulatory interactions. This is a clear goal of the European Medicines Agency’s Innovation Task Force, as you will read in this issue, andwe clearly need to help overcome the reticence ofmany organisations to engage

regulation has had to keep pace with this rate of progress and, as a result, there is now a growing portfolio of regulations and guidance covering themore prevalent advanced therapies and medical device legislation,whichhas recentlybeenupdated in theEU. However, there remains a need for this portfolio to continue to expand to cover emerging science such as gene editing, microbiome applications or the potential roleofartificial intelligence indevelopment,

Innovation isa progressivepath,andone whichbuildsupasnew technologiesdevelop

earlywith regulators.Thisearlyengagement offers an opportunity to lead the field of novel therapies and the EMA’s Regulatory Science to2025strategyalso recognises the need for early engagement, as presented

asexistingguidelinesonly just touch the surfaceof thepotential challenges in these areas. So, in the absence of guidance, the question is how dowe lead thefield? Industryoftenapplauds itselfforworkingatthecuttingedgeof innovation. However,by the timewe regulatoryprofessionalsseea late-stageconceptor earlydevelopment stageproduct, the technology is already a few yearsold and the capacity tomould it to complywith regulatory requirementsmaybe

in thismonth’smeeting report. It also offers a transparent approach for all stakeholders and enables regulators to perform horizon scanning which, ultimately, speeds access both for patients to ground-breaking therapies and, fordevelopers, to the targetmarket (it isawin-winsituation). The clear theme emerging at the start of this new decade is thatwe all need tocommunicate,educate,andcollaborate, to innovate.The framework todo this isout there–weall justneed toembrace it.


Vol. 17,N02, February2020 | REGULATORYRAPPORTEUR | 3

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CASE STUDY Future therapies realised today

Regulatory scientists like Michela Gabaldo are at the forefront of delivering the potential of gene therapies. After graduating from Padua University, Italy, with a degree in pharmaceutical chemistry and technology she joined GlaxoSmithKline in 2001 and worked in clinical operations and CMC (chemistry manufacturing controls) regulatory affairs.

Ten years later, she was managing gene therapy projects at Alliance Management and Regulatory Affairs at Fondazione Telethon, Milan, Italy. She made a key contribution to the clinical development and EU marketing approval of Strimvelis (GSK), an autologous gene therapy for the treatment of an ultra-rare severe combined immunodeficiency (ADA-SCID), which was the first ex vivo gene therapy approved in the world. She is also a winner of the Futures category of the TOPRA Awards for Regulatory Excellence, in recognition of her contribution of ‘innovative ideas and solutions to overcome the regulatory hurdles that are typically associated with a highly innovative medicine as gene therapy for a rare disorder’. Dr Gabaldo is now Head Alliance Management & Regulatory Affairs Manager at SR-TIGET San Rafaele Telethon Institute for gene therapy, Milan, Italy and a member of the International Rare Diseases Research Consortium (IRDiRC) Therapies Scientific Committee.

Michela Gabaldo

Preparing regulatory professionals for expanded roles

There are nowmany different career routes and specialties open to the modern regulatory professional; expectations for skills, knowledge and competencies have changed radically in the 21st century. TOPRA offers relevant training and professional development to enable regulatory professionals at all levels to pursue these options and achieve their potential. Academic preparation For more than 30 years, TOPRA has offered a postgraduate specialist regulatory qualification. It continues to adapt to regulatory needs and expectations. In 2017, the TOPRA MSc Regulatory Affairs programme was radically updated and expanded with the University of Hertfordshire, offering two pathways – Medicines andMedical Devices. Students on either pathway can now choose up to two modules from the other – an innovation that reflects the increasing number of professionals working with combination products or needing in-depth knowledge of a particular area,

eg, data management and digitisation. Newmodules and Masterclasses are being considered and developed, eg, the regulatory aspects of developing advancedmedicinal therapy products (ATMPs). A number of regulatory professionals are studying for a PhD with TOPRA. Adding to the body of knowledge, their research will help to underpin future development of regulatory science. Soft skills Regulatory knowledge and expertise are not the only requirements to build a successful career. To be a truly effective regulatory professional, individuals need to draw on a toolbox of practical, soft skills to manage their expanded roles. Communications and project management are key areas for professional development. Such needs are being met by an increasing range of CRED and online courses and webinars, covering document writing, regulatory operations, digitisation, etc.

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Engagement and Connectivity

Our commitment • Build on our digital platform and channels to disseminate content, share experiences and collaborate remotely • Promote a culture of equality, diversity and inclusion across the regulatory profession • Reach out to other networks, groups and organisations sharing our vision and values to offer access to complementary resources and opportunities • Build on our volunteer programme to help every member contribute to our community to enrich the sense of belonging Making connections Our success in readily adapting to the challenges of the COVID-19 global pandemic underlines the importance of enabling our membership and wider stakeholder networks to connect by many and variedmeans. Sharing of regulatory knowledge and experience within the regulatory community is fundamental to helping develop individuals, educational activities and informing professional practice at an organisational, national and international level. Within the TOPRA community Access to a wide range of local groups and Special Interest Networks (SPINs) provides a much valued resource for members to access and participate in developing the group’s activities through volunteering. National and regional networks Regional and country-specific groups offer individuals the opportunity to get together to networkwith colleagues to learn and discuss issues related to their geography.

Contribute and grow Building a network of regulatory knowledge and experience

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Many SPINs engage in sub-group projects, eg, preparing articles for publication, presenting parallel sessions for Symposia. The Regulatory Intelligence SPINmaintains an online RI Knowledge Hub for members. Beyond the TOPRA community Social media has radically changed how people network personally and professionally. TOPRA’s increased presence on LinkedIn, Twitter and Facebook, reaches a global audience of tens of thousands of stakeholders with a common interest in the regulatory affairs community.

Apart from face-to-face educational focused meetings with a social aspect, members can interact with regional online communities and access webinars. As TOPRA’s global membership expands, so will its local groups – more recent additions include TOPRA In India and TOPRA in Central and Eastern Europe . Special interest networks SPINmembership provides access to discussion of topics with common interest –mainly through online communities, supplemented with relevant webinars and occasional face-to- face meetings.

We established our TOPRA in Sweden local group in order to network and share information on regulatory news and topics affecting us in our day-to-day roles. Meetings take place in the evening five times a year – three of them at the Swedish MPA in Uppsala, the other two are in Malmo and Gothenburg respectively. Everyone enjoys getting together regularly to discuss popular and valued topics, such as annual regulatory update news for medicinal products and medical devices, drug/device combination products, regulatory support to start-up companies, clinical trials, HTA/market access and product information. Margareth Jorvid Senior Partner, LSM group (Life Science Management), Uppsala, Sweden

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Attracting a broad range of talents and backgrounds TOPRA proactively promotes a culture of equality, diversity and inclusion; encouraging members to engage with expertise, experience and wider networks of colleagues from any and all backgrounds. We are a signatory to the Science Council’s Diversity Declaration . Enhancing the depth of knowledge and breadth of reach available to the TOPRA community and our stakeholder networks increases the value of membership and raises the overall profile of the regulatory affairs profession. To encourage wider access, w e support a range of routes into the profession – in particular by encouraging student membership and developing apprenticeships. Students Undergraduates can access free membership, which enables them to experience what regulatory affairs and TOPRA have to offer. Every year TOPRA hosts Regulatory Careers Fair Live, which draws in many students and professionals at the outset of their careers. Apprentices The Regulatory Affairs Apprenticeship Standard is a crucial route for new entrants to the profession. We now have a growing number of apprentices from Medicines, Medical Devices and ATMPs (advanced therapy medicinal products) taking part in the programme with the opportunity to graduate with a TOPRA MSc Regulatory Affairs (Medicines/Medical Devices) and achieve Registered Scientist status.

At this stage of my life, it would not be suitable for me to quit my job in order to start a full-time MSc... [The apprenticeship] allows me to become qualified in two-and-a-half years while earning a living. I will have an MSc degree and will be able to work as a regulatory affairs specialist. The apprenticeship is not all study and includes great networking opportunities. Since I enrolled, I have met many people from other organisations on the same courses and

events as me. Ksenia Sitara

Senior QC Officer, Analytical Services Group at Oxford Biomedica, is a Regulatory Affairs Specialist Apprentice (ATMPs)

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Global Reach and Value

Our commitment • Provide relevant support to respond to the needs of the global regulatory community • Extend access to regulatory education, particularly in emerging markets, through further development of our online services and virtual engagement • Reach out to and workwith partners to help deliver initiatives that advance health equity across the world • With partners help create environments where biomedical science, public health and clinical research can flourish in developing countries through improved regulatory oversight TOPRA is uniquely positioned to capitalise on the increasing trend for convergence of regulatory practice worldwide. Our ethos and outlook remain international, offering regulatory support that is relevant and responsive to specific geographic needs. We are furthering our use of technology to increase digital outreach and virtual engagement across our growing community. Promoting shared professional standards and driving best practice globally are core to our values. We are well placed to offer developing countries support with creating environments where biomedical science, population health, clinical research and medical innovation flourish because of effective regulatory oversight.

Breaking barriers and boundaries

Bringing regulatory affairs together across the world

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