BEGIN:VCALENDAR VERSION:2.0 METHOD:PUBLISH PRODID:-//Telerik Inc.//Sitefinity CMS 15.4//EN BEGIN:VEVENT DESCRIPTION:Immunotherapy treatments use parts of a person&rsquo\;s immune system to fight disease. In the recent past\, immunotherapy has become an important part of treating some types of cancer e.g. melanoma\, NSCLC. Stu dies of these treatments have resulted in a number of observations that ha ve implications for the statistician e.g. delayed treatment effects\, long term survivors etc. This joint Basel Biometric Society / PSI one day meet ing will present an overview of the science and potential statistical chal lenges across a range of topics covering early and late phases of developm ent\, regulatory and health technology assessments. The speakers from Acad emia\, Regulatory Bodies and Pharma will share their thoughts\, ideas and experiences\, including case studies. There will be plenty of time for que stions and interactions with colleagues. The meeting will take place on Ju ne 15th \;2017 in Basel at the Roche IT Centre. To view the agenda\, p lease click here.\nIf you have any questions please feel free to contact o ne of the organisers from the BBS or PSI Scientific Committee: david.lawre nce@astrazeneca.com\, dominik.heinzmann@roche.com or emanuela.pozzi@roche. com \n​​Registration is now closed. Presentation Slides Session 1 - Andrew Stone Session 1- Jorge Martinalbo Session 2 - Daniel Sabanes Bove Session 2 - Matt Whiley Session 3 - Claude Berge Session 3 - Dominik Heinzmann Se ssion 3 - Sergio Fracchia Session 4 - Fred Sorenson Session 4 - Nicholas L atimer Speakers and Abstracts THE SCIENCE OF IMMUNOTHERAPY \nAndrew Stone Managing director Stone Biostatistics Biography A consultant biostatisti cian with Stone Biostatistics who has 24 years of experience in the pharma ceutical industry and held a senior leadership or management position in t he oncology TA at AstraZeneca for nearly 10 years. \; Andrew has signi ficant regulatory experience\, leading biostatistics teams to the submissi on or approval of six oncology products. \; Furthermore\, Andrew sat o n governance committees that approved the design of >\; 50 pivotal trial s. \; \; In addition\, Andrew led the Statistical Innovation team at AZ for 9 months before his departure from AZ\, due to a site closure\, in May 2016. Presentation Statistical issues in the development of cancer immunotherapy With the advent of immunotherapy (IO)\, which seems to be co ntributing to a golden age in oncology\, a lot has been discussed about no n-proportional hazards (NPH). \; Some of this has the potential to lea d to unwise alternatives\, whereby increasing the survival of better progn osis patients is weighted as more important. \; \; In the presence of NPH\, the hazard ratio (HR) produced by conventional analysis approach es is equal to the average HR\, which remains a meaningful measure of over all benefit. \; \; We should though consider alternative\, not rep lacement\, measures of absolute benefit to better the describe any benefit . \; \; The emerging profile of IO questions whether we should gra pple with the challenges of assessing cure rates\, or long term survival r ates\, and also re-consider the role of non-inferiority in assessing the o verall benefit/risk of therapies. \; \; Currently we maybe making it more difficult to make available better tolerated\, equally efficacious alternatives most difficult in situations where there is the greatest nee d. \; Finally\, broader issues will be discussed such as requirements for demonstration of contribution of components when seeking approval for combinations of unapproved therapies\, and less reliance on single-arm tri als that provide a rapid but an ultimately unreliable approach to assessin g likely benefit. \nEARLY DEVELOPMENT CHALLENGES \nDaniel Sabanes Bove Senior Statistical Scientist\, Roche Biography Daniel joined Roche in Octo ber 2013\, and has supported multiple early phase Oncology projects from t he Basel headquarters\, where he is currently Senior Statistical Scientist . Before that\, he received a Master of Science in Statistics from the Lud wig-Maximilians-Universitä\;t Mü\;nchen in 2009 and a PhD in Stati stics from the University of Zurich in 2013. Daniel received the Bernd-Str eitberg young researcher award from the German Region of the International Biometrical Society\, and co-authored the book "Applied Statistical Infer ence" (Springer\, 2014). He developed the R-package &ldquo\;crmPack&rdquo\ ; for model-based dose escalation designs and gave Roche-internal and also external (ICTMC 2017) tutorials. Other current research interests compris e endpoints in cancer immunotherapy and associated decision making. (For p ublications please see Google Scholar) Presentation Bayesian Learning in E arly Phase Cancer Immunotherapy: A Case Study The early clinical stage of drug development is a learning phase: we are learning continuously about t he drug&rsquo\;s safety\, pharmacokinetics\, pharmacodynamics and efficacy \, building on our current knowledge. Therefore Bayesian inference\, with its coherent concept of updating prior information with observed data to o btain the posterior information about quantities of interest\, is a perfec t match to early phase study designs and to broader clinical development q uestions. This case study on a new cancer immunotherapeutic agent starts with the entry-into-human phase I dose escalation study. It is shown how t he modified Continual Reassessment Method (CRM) design incorporated reason able prior assumptions about the expected safety profile\, and ensured max imum flexibility for study conduct. A separate dose escalation was then pl anned for the combination with another new drug\, with the design building on the two compound&rsquo\;s information. As during the phase I it became apparent that a large proportion of patients developed anti-drug antibodi es against the molecule\, a small proof-of-concept study with a pretreatme nt aiming to diminish the immune response against the biologic was designe d. Finally\, the information gathered so far can be used to setup the entr y-into-human phase I study for another molecule from the same platform. T he clinical development questions and Bayesian answers to them will be pre sented\, with a focus on the decision making and practical considerations in developing a new cancer immunotherapy. \nYolanda Barbachano Senior Stat istical Assessor\, Licensing Division\, MHRA Presentation Development of I mmunotherapies \; &ndash\; challenges encountered at MHRA Immunothera pies work very differently to other cancer therapies but this is often not taken into account when designing clinical trials to investigate their ef ficacy and safety. This talk will describe some of the issues this can lea d to\, from phase 1 to phase 3\, by using anonymised examples of trials se en at MHRA. LATE DEVELOPMENT: STATISTICAL AND REGULATORY CHALLENGES Domi nik Heinzmann Associate Director Biostatistics\, Roche Biography Dr. Domi nik Heinzmann is an Associate Director and Manager of Biostatistics in Roc he Basel for Cancer Immunotherapy and HER2+ targeted therapies. Dominik is also acting as a Global Development Team Leader in HER2+ breast cancer. D ominik has about 7 years of experience in Roche in medical oncology and ha s a broad experience in interactions with regulators including multiple su bmissions of different oncology products. Prior to joining Roche\, Dominik received a PhD in Biomathematics from the University of Zurich\, and hold s a M.Sc. in Mathematics &\; Statistics from the Swiss Federal Institut e of Technology. He authored more than 20 publications in peer-reviewed jo urnals in statistical\, epidemiological and medical journals. Presentation Statistical\, clinical and ethical considerations when minimizing confoun ding for overall survival in cancer immunotherapy trials Recent data on ca ncer immunotherapy (CIT) monotherapy suggest that PFS may not be an approp riate endpoint. If one considers overall survival (OS)\, a risk with this endpoint is that of confounding\, due to cross-over\, i.e. treatment switc hing from control patients to the experimental arm within the trial\, enro lling into a subsequent trial with a similar agent as the experimental arm \, or because a similar agent has become commercially available. \nIn this talk\, we will discuss statistical measures to minimize confounding for O S and their implications for trial participants as well as the broader und erlying population from a clinical and ethical perspective. Sergio Fracc hia Associated Director Biologics Cell and Gene Therapy\, Novartis Biogr aphy Sergio presently works as Associate Director RegCMC &ndash\; Cell and Gene Therapy at Novartis (Basel). \; He joined Novartis in 2016. Prev iously\, he covered the position of Regulatory Affairs Manager in Molmed ( milan)\, where he followed the global development through the entire life cycle from early clinical trial application to filing of different cell an d gene therapy products intended to address highly unmet medical need in t he area of oncohaematology\, primary immunodeficiencies and. neurodegenera tive diseases. He started his professional life with 1 year post-doctoral research in biochemistry\, followed by 15 years experience at Merck/Serono where he worked in a laboratory focussed on cell bank characterization. F ormal education includes a MSc in Biology and a PhD in Biotechnology.  \; Presentation Challenges in development and approval: the case of cell based therapeutic The presentation covers the issues and challenges in the development of cell and gene therapy products\, still representing a spec ific niche\, still increasing in relevance\, in the broader pharmaceutical arena. \; Pharmaceutical based on cells have been in the last decade an area of intense investigation and in the last year the first have been approved for commerce. \; These products differ from large and small molecules in a number of features because of the extreme species specifici ty and often because a specific batch is produced for a single patient.&nb sp\; In addition\, manufacturing process and product characterization are non-conventional. \; Therefore\, the approach and paradigms adopted fo r overall manufacturing\, non-clinical and clinical development leading to registration is different from the one usually used large and small molec ules in terms of the stud(ies) design and endpoint for safety and efficacy assessment\, timing for execution during development and size.\nThe impac t of cell based pharmaceutical peculiarities in terms manufacturing and in -vivo behavior are considered and compared with large and small molecules. BEYOND APPROVAL (HTA) Fred Sorensen Assistant Director\, Global HEOR and Market Access\, Xcenda®\; Biography Fred Sorenson is an Assistant D irector for Global Health Economics\, Outcomes Research\, and Market Acces s at Xcenda. His work includes comparative effectiveness research\, retros pective database studies\, prospective studies and chart reviews\, systema tic reviews\, global value dossiers\, and contributing to posters and publ ications. Before joining Xcenda\, Mr. Sorenson led various teams responsib le for research in health economics as well as heading the department of b iostatistics at a clinical research organization and health care consultan cy in Switzerland for more than 10 years. He remains active in biostatisti cs as an Executive Board Member of the Basel Biometric Society and formerl y as representatives to the European Federation of Statisticians in the Ph armaceutical Industry (EFSPI). Mr. Sorenson received Bachelor of Science degrees in both Psychology and Philosophy from the University of Southern Colorado and did post-graduate studies in Sociology and Economics at the U niversity of Basel in Switzerland. Presentation Cancer immunotherapy from the Health Technology Assessment (HTA) and payer perspective Assessment of value by Health Technology Assessment (HTA) bodies for reimbursement of I mmuno-oncology drugs is complex as few of this class of drugs have single indications\, and divergence in clinical value by indication complicates a ssessments by payers. This is further complicated by the fact that only on e EU market authorization submission is required\, whereas HTA bodies adhe re to their own health reimbursement policy\, and therefore are not always in agreement. More recently\, the development by different organizations of &ldquo\;Value Frameworks&rdquo\;\, especially in Oncology and employing different algorithms for assessing value have entered the scene. \;&n bsp\; \; \nThis presentation will provide some background concerning w hat statisticians need to know and the issues surrounding these developmen ts\, and more importantly\, possible ways that statisticians can contribut e to improving methods and processes for evaluating value to meet the need s of payers and reimbursement. Nicholas R. Latimer Senior Research Fel low in Health Economics\, NIHR Post-Doctoral Fellow\, University of Sheffi eld Biography Nicholas Latimer is a Senior Research Fellow in Health Econo mics at the School of Health and Related Research (ScHARR)\, University of Sheffield. He joined ScHARR in 2008\, having previously worked as a healt h economist at NERA Economic Consulting\, Queen Mary University of London (QMUL)\, and Roche Products Ltd. His research expertise is in the area of survival analysis in economic evaluations &ndash\; particularly the use o f survival modelling techniques to extrapolate beyond clinical trial data\ , and the use of statistical methods for adjusting survival estimates in t he presence of treatment switching. In 2012 Nick completed an National Ins titute for Health Research (NIHR) Doctoral Research Fellowship that focuss ed upon these topics and in 2015 he was awarded an NIHR Post-Doctoral Fell owship to continue this research. Nick has authored two National Institute for Health and Care Excellence (NICE) Decision Support Unit technical sup port documents\, on survival analysis (TSD 14) and the use of treatment sw itching adjustment methods (TSD16). Nick has considerable experience of an alysing clinical trial data\, and of conducting model-based and trial-base d economic evaluation. He has led the Evidence Review Group (ERG) on NICE Technology Appraisals\, has led the economic analysis on NICE Clinical Gui delines\, has contributed to NICE Public Health guidelines\, and has been the principal investigator on several research and consultancy projects. N ick drafted sections on extrapolation and treatment switching for the 2013 NICE Methods Guide and is an invited expert on the NICE Scientific Advice Programme. Nick collaborates internationally\, and has been involved in t he development of technical guidance on survival analysis methods by the P harmaceutical Benefits Advisory Committee (PBAC) in Australia. He has link s with IQWiG (Germany)\, the Canadian Agency for Drugs and Technologies in Health (CADTH)\, the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). Presentation Estimating survival benefit for he alth technology assessment: new challenges presented by immuno-oncology tr eatments? Several new immuno-oncology (I-O) treatments appear to &lsquo\;c ure&rsquo\; a proportion of patients: survival for this group often contin ues beyond the trial observation period and a plateau is observed in the s urvival curve. Hazard functions appear to be non-proportional and complex. Standard parametric models which have commonly been used to estimate long -term survival for use in economic evaluations undertaken within health te chnology assessments may not be appropriate for modelling such data. Novel survival modeling methods\, such as mixture cure models and flexible para metric models\, have emerged as potentially useful alternative modelling a pproaches\, and using these models can fundamentally change estimates of e ffectiveness and cost&ndash\;effectiveness. In this session\, standard mod elling approaches will be summarised\, as will their limitations given the apparent characteristics of new I-O treatments. Alternative modelling met hods will be introduced and discussion will consider whether the issues ra ised are specific to I-O treatments\, and whether HTA agencies are prepare d to appraise the application of more complex survival models. \; DTEND;VALUE=DATE:20170616 DTSTAMP:20261014T081355Z DTSTART;VALUE=DATE:20170615 LOCATION:Basel\,Roche IT Centre SEQUENCE:0 SUMMARY:BBS / PSI One-day Event on Cancer Immunotherapy UID:RFCALITEM639275624357558354 X-ALT-DESC;FMTTYPE=text/html:

Immunotherapy treatments use parts of a per son&rsquo\;s immune system to fight disease. In the recent past\, immunoth erapy has become an important part of treating some types of cancer e.g. m elanoma\, NSCLC. Studies of these treatments have resulted in a number of observations that have implications for the statistician e.g. delayed trea tment effects\, long term survivors etc. This joint Basel Biometric Societ y / PSI one day meeting will present an overview of the science and potent ial statistical challenges across a range of topics covering early and lat e phases of development\, regulatory and health technology assessments. Th e speakers from Academia\, Regulatory Bodies and Pharma will share their t houghts\, ideas and experiences\, including case studies. There will be pl enty of time for questions and interactions with colleagues.

The me eting will take place on June 15th \;2017 in Basel at the R oche IT Centre. To view the agenda\, please click here.

\nIf you have any questions please f eel free to contact one of the organisers from the BBS or PSI Scientific C ommittee: david.lawrence@a strazeneca.com\, dominik.heinzmann@roche.com or emanuela.pozzi@roche.com

\n​​Re gistration is now closed.

Presentation Slides

Session 1 - Andrew Stone
Session 1- Jorge Martinalbo
Se ssion 2 - Daniel Sabanes Bove
Session 2 - Matt Whiley
Session 3 - Claude B erge
Session 3 - Dominik Heinzmann
Session 3 - Sergio Fr acchia
Session 4 - Fred Sorenson
Session 4 - Nicholas Latimer< /a>

Speakers and Abstracts

THE SCIENCE OF IMM UNOTHERAPY

\nAndrew Stone
Andrew Stone

Managing director Stone Biostatistics

Biography

A consultant biostatistician with S tone Biostatistics who has 24 years of experience in the pharmaceutical in dustry and held a senior leadership or management position in the oncology TA at AstraZeneca for nearly 10 years. \; Andrew has significant regu latory experience\, leading biostatistics teams to the submission or appro val of six oncology products. \; Furthermore\, Andrew sat on governanc e committees that approved the design of >\; 50 pivotal trials. \;&n bsp\; In addition\, Andrew led the Statistical Innovation team at AZ for 9 months before his departure from AZ\, due to a site closure\, in May 2016 .

Presentation

Statistical issues in the development of cancer immunotherapy

With the advent of immun otherapy (IO)\, which seems to be contributing to a golden age in oncology \, a lot has been discussed about non-proportional hazards (NPH). \; S ome of this has the potential to lead to unwise alternatives\, whereby inc reasing the survival of better prognosis patients is weighted as more impo rtant. \; \; In the presence of NPH\, the hazard ratio (HR) produc ed by conventional analysis approaches is equal to the average HR\, which remains a meaningful measure of overall benefit. \; \; We should t hough consider alternative\, not replacement\, measures of absolute benefi t to better the describe any benefit. \; \; The emerging profile o f IO questions whether we should grapple with the challenges of assessing cure rates\, or long term survival rates\, and also re-consider the role o f non-inferiority in assessing the overall benefit/risk of therapies.  \; \; Currently we maybe making it more difficult to make available be tter tolerated\, equally efficacious alternatives most difficult in situat ions where there is the greatest need. \; Finally\, broader issues wil l be discussed such as requirements for demonstration of contribution of c omponents when seeking approval for combinations of unapproved therapies\, and less reliance on single-arm trials that provide a rapid but an ultima tely unreliable approach to assessing likely benefit.


\nEARLY DEVELOPMENT CHALLENGES

\nDaniel Saba nes Bove

Daniel Sabanes Bove

Senior Statistical Scientist\, Roche

Biography

Daniel joined Roche in October 2013\, and has supported multiple early phase Oncology projects from the Basel headq uarters\, where he is currently Senior Statistical Scientist. Before that\ , he received a Master of Science in Statistics from the Ludwig-Maximilian s-Universitä\;t Mü\;nchen in 2009 and a PhD in Statistics from the University of Zurich in 2013. Daniel received the Bernd-Streitberg young researcher award from the German Region of the International Biometrical S ociety\, and co-authored the book "Applied Statistical Inference" (Springe r\, 2014). He developed the R-package &ldquo\;crmPack&rdquo\; for model-ba sed dose escalation designs and gave Roche-internal and also external (ICT MC 2017) tutorials. Other current research interests comprise endpoints in cancer immunotherapy and associated decision making. (For publications pl ease see Google Scholar)

Presentation

Bayesian Learning in Early Phase Cancer Immunotherapy: A Case Study

The early clinical stage of drug development is a learning phase: we are learning continuously about the drug&rsquo\;s safety\, pharmacokinetic s\, pharmacodynamics and efficacy\, building on our current knowledge. The refore Bayesian inference\, with its coherent concept of updating prior in formation with observed data to obtain the posterior information about qua ntities of interest\, is a perfect match to early phase study designs and to broader clinical development questions.

This case study on a ne w cancer immunotherapeutic agent starts with the entry-into-human phase I dose escalation study. It is shown how the modified Continual Reassessment Method (CRM) design incorporated reasonable prior assumptions about the e xpected safety profile\, and ensured maximum flexibility for study conduct . A separate dose escalation was then planned for the combination with ano ther new drug\, with the design building on the two compound&rsquo\;s info rmation. As during the phase I it became apparent that a large proportion of patients developed anti-drug antibodies against the molecule\, a small proof-of-concept study with a pretreatment aiming to diminish the immune r esponse against the biologic was designed. Finally\, the information gathe red so far can be used to setup the entry-into-human phase I study for ano ther molecule from the same platform.

The clinical development que stions and Bayesian answers to them will be presented\, with a focus on th e decision making and practical considerations in developing a new cancer immunotherapy.


\nYolanda Barbachano

S enior Statistical Assessor\, Licensing Division\, MHRA

Presentation

Development of Immunotherapies \; &n dash\; challenges encountered at MHRA

Immunotherapies work very di fferently to other cancer therapies but this is often not taken into accou nt when designing clinical trials to investigate their efficacy and safety . This talk will describe some of the issues this can lead to\, from phase 1 to phase 3\, by using anonymised examples of trials seen at MHRA.

LATE DEVELOPMENT: STATISTICAL AND REGULATORY CHALLENGES

Dominik Heinzmann< /p> Dominik Heinzmann

Associate Director Biostatistics\, Roche

Biography

Dr. Dominik Heinzm ann is an Associate Director and Manager of Biostatistics in Roche Basel f or Cancer Immunotherapy and HER2+ targeted therapies. Dominik is also acti ng as a Global Development Team Leader in HER2+ breast cancer. Dominik has about 7 years of experience in Roche in medical oncology and has a broad experience in interactions with regulators including multiple submissions of different oncology products. Prior to joining Roche\, Dominik received a PhD in Biomathematics from the University of Zurich\, and holds a M.Sc. in Mathematics &\; Statistics from the Swiss Federal Institute of Techn ology. He authored more than 20 publications in peer-reviewed journals in statistical\, epidemiological and medical journals.

Pre sentation

Statistical\, clinical and ethical consider ations when minimizing confounding for overall survival in cancer immunoth erapy trials

Recent data on cancer immunotherapy (CIT) monotherapy suggest that PFS may not be an appropriate endpoint. If one considers over all survival (OS)\, a risk with this endpoint is that of confounding\, due to cross-over\, i.e. treatment switching from control patients to the exp erimental arm within the trial\, enrolling into a subsequent trial with a similar agent as the experimental arm\, or because a similar agent has bec ome commercially available.

\nIn this talk\, we will discuss statistic al measures to minimize confounding for OS and their implications for tria l participants as well as the broader underlying population from a clinica l and ethical perspective.


Sergio Fracchia< /strong>

Sergio Fracchia

Associated Director Biologics C ell and Gene Therapy\, Novartis

Biography

Sergio presently works as Associate Director RegCMC &ndash\; Cell and Gene Therapy at Novartis (Basel). \; He joined Novartis in 2016.

Previously\, he covered the position of Regulatory Affairs Manager i n Molmed (milan)\, where he followed the global development through the en tire life cycle from early clinical trial application to filing of differe nt cell and gene therapy products intended to address highly unmet medical need in the area of oncohaematology\, primary immunodeficiencies and. neu rodegenerative diseases.

He started his professional life with 1 ye ar post-doctoral research in biochemistry\, followed by 15 years experienc e at Merck/Serono where he worked in a laboratory focussed on cell bank ch aracterization.

Formal education includes a MSc in Biology and a Ph D in Biotechnology. \;

Presentation< /p>

Challenges in development and approval: the case of cell based ther apeutic

The presentation covers the issues and challenges in the de velopment of cell and gene therapy products\, still representing a specifi c niche\, still increasing in relevance\, in the broader pharmaceutical ar ena. \;

Pharmaceutical based on cells have been in the last de cade an area of intense investigation and in the last year the first have been approved for commerce. \; These products differ from large and sm all molecules in a number of features because of the extreme species speci ficity and often because a specific batch is produced for a single patient . \; In addition\, manufacturing process and product characterization are non-conventional. \; Therefore\, the approach and paradigms adopte d for overall manufacturing\, non-clinical and clinical development leadin g to registration is different from the one usually used large and small m olecules in terms of the stud(ies) design and endpoint for safety and effi cacy assessment\, timing for execution during development and size.

\nT he impact of cell based pharmaceutical peculiarities in terms manufacturin g and in-vivo behavior are considered and compared with large and small mo lecules.


BEYOND APPROVAL (HTA)

Fred Sorensen

Fred Sorensen

Assistant D irector\, Global HEOR and Market Access\, Xcenda®\;

Biography

Fred Sorenson is an Assistant Director for Global Health Economics\, Outcomes Research\, and Market Access at Xcenda. His work includes comparative effectiveness research\, retrospective data base studies\, prospective studies and chart reviews\, systematic reviews\ , global value dossiers\, and contributing to posters and publications.

Before joining Xcenda\, Mr. Sorenson led various teams responsible fo r research in health economics as well as heading the department of biosta tistics at a clinical research organization and health care consultancy in Switzerland for more than 10 years. He remains active in biostatistics as an Executive Board Member of the Basel Biometric Society and formerly as representatives to the European Federation of Statisticians in the Pharmac eutical Industry (EFSPI).

Mr. Sorenson received Bachelor of Scienc e degrees in both Psychology and Philosophy from the University of Souther n Colorado and did post-graduate studies in Sociology and Economics at the University of Basel in Switzerland.

Presentation< /strong>

Cancer immunotherapy from the Health Technology Assessment (HTA) and payer perspective

Assessment of value by Health Technolo gy Assessment (HTA) bodies for reimbursement of Immuno-oncology drugs is c omplex as few of this class of drugs have single indications\, and diverge nce in clinical value by indication complicates assessments by payers. Thi s is further complicated by the fact that only one EU market authorization submission is required\, whereas HTA bodies adhere to their own health re imbursement policy\, and therefore are not always in agreement. More recen tly\, the development by different organizations of &ldquo\;Value Framewor ks&rdquo\;\, especially in Oncology and employing different algorithms for assessing value have entered the scene. \; \; \;

\nThis p resentation will provide some background concerning what statisticians nee d to know and the issues surrounding these developments\, and more importa ntly\, possible ways that statisticians can contribute to improving method s and processes for evaluating value to meet the needs of payers and reimb ursement.


Nicholas R. Latimer

Nicholas R. Latimer

Senior Research Fellow in Health Economics\, NIHR Post-Doctoral Fellow\, University of Sheffield

Biography

Nicholas Latimer is a Senior Resea rch Fellow in Health Economics at the School of Health and Related Researc h (ScHARR)\, University of Sheffield. He joined ScHARR in 2008\, having pr eviously worked as a health economist at NERA Economic Consulting\, Queen Mary University of London (QMUL)\, and Roche Products Ltd.

His res earch expertise is in the area of survival analysis in economic evaluation s &ndash\; particularly the use of survival modelling techniques to extrap olate beyond clinical trial data\, and the use of statistical methods for adjusting survival estimates in the presence of treatment switching. In 20 12 Nick completed an National Institute for Health Research (NIHR) Doctora l Research Fellowship that focussed upon these topics and in 2015 he was a warded an NIHR Post-Doctoral Fellowship to continue this research. Nick ha s authored two National Institute for Health and Care Excellence (NICE) De cision Support Unit technical support documents\, on survival analysis (TS D 14) and the use of treatment switching adjustment methods (TSD16).

< p>Nick has considerable experience of analysing clinical trial data\, and of conducting model-based and trial-based economic evaluation. He has led the Evidence Review Group (ERG) on NICE Technology Appraisals\, has led th e economic analysis on NICE Clinical Guidelines\, has contributed to NICE Public Health guidelines\, and has been the principal investigator on seve ral research and consultancy projects. Nick drafted sections on extrapolat ion and treatment switching for the 2013 NICE Methods Guide and is an invi ted expert on the NICE Scientific Advice Programme.

Nick collaborat es internationally\, and has been involved in the development of technical guidance on survival analysis methods by the Pharmaceutical Benefits Advi sory Committee (PBAC) in Australia. He has links with IQWiG (Germany)\, th e Canadian Agency for Drugs and Technologies in Health (CADTH)\, the Europ ean Medicines Agency (EMA) and the US Food and Drug Administration (FDA).< /p>

Presentation

Estimating survival b enefit for health technology assessment: new challenges presented by immun o-oncology treatments?

Several new immuno-oncology (I-O) treatments appear to &lsquo\;cure&rsquo\; a proportion of patients: survival for thi s group often continues beyond the trial observation period and a plateau is observed in the survival curve. Hazard functions appear to be non-propo rtional and complex. Standard parametric models which have commonly been u sed to estimate long-term survival for use in economic evaluations underta ken within health technology assessments may not be appropriate for modell ing such data. Novel survival modeling methods\, such as mixture cure mode ls and flexible parametric models\, have emerged as potentially useful alt ernative modelling approaches\, and using these models can fundamentally c hange estimates of effectiveness and cost&ndash\;effectiveness. In this se ssion\, standard modelling approaches will be summarised\, as will their l imitations given the apparent characteristics of new I-O treatments. Alter native modelling methods will be introduced and discussion will consider w hether the issues raised are specific to I-O treatments\, and whether HTA agencies are prepared to appraise the application of more complex survival models. \;

END:VEVENT END:VCALENDAR