tag:blogger.com,1999:blog-6767541251661932312024-02-06T18:29:14.430-08:00MPNEatDIABettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.comBlogger14125tag:blogger.com,1999:blog-676754125166193231.post-73251517672472751192016-04-08T06:09:00.002-07:002016-04-08T14:58:55.939-07:00<h2>
Oxford debate: this house believes that over engineered clinical development has inhibited innovation</h2>
Panellists: Bettina Ryll (Founder of Melanoma Patient Network Europe) Martin Landray (University of Oxford), Mike Ryan (Medidata Solution), Sam Wadsworth (Dimension Therapeutic) -session chair Jullianne Hull<br />
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<b>Mike Ryan</b><br />
innovation -new ideas<br />
new implies taking risks<br />
regulatory authorities are seeing that as a risk<br />
scientific advices -helpful but time consuming<br />
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<b>Martin Landray</b><br />
To engineer- a methodological approach to integrate elements<br />
there is prudence in engineering in the context of CT<br />
-is quality of clinical design, involving all stakeholders including pts and public<br />
a critical thinking<br />
we should stop to do things that we suppose others are expecting from us<br />
over engineering is not the problem, but forget engineering is the problem<br />
CTs (clinical trials) -have to integrate a critical human element,<br />
each element can be subject of engineering<br />
we live in imperfect world<br />
we do need systems to review errors<br />
''I have heard from Pharma that the new technology are inducing undetermined risks '' so better to not go for new technologies..<br />
<i>but..engineering is the necessity for innovation</i><br />
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<b>Sam Wadsworth</b><br />
looking to 20 years clinical trials experience<br />
how we engineer clinical trials now<br />
the number of CTs has increased<br />
but the number of new drug approved has decreased<br />
we are asking pts (patients) to take procedures which are not necessary (?!)<br />
Pts and doctors are finding impossible to run CTs<br />
Interactions with specific IT tools within CTs is difficult for doctors/investigators (!)<br />
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<b>Bettina Ryll</b><br />
Not true<b>-</b>the above assertion confounds symptoms with the cause<br />
Innovation in health improves clinical efficacy, cost effectiveness, pharmacovigilance,<br />
Innovation is there to bring timely access to the good drugs to the patients.<br />
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Innovation has to be accessible in the right time and for all! ..<br />
Clinical trials with a bad design -<i>from the day they start everybody knows that half of our people would die on inferior comparators:</i> this has to stop and we have to ask ourself for what and for whom we are doing this, what are our real goals.<br />
Innovation has to be sustainable<br />
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<b>Regulators comments</b><br />
The most problems come not for engineering the trials but from designing bad protocols<br />
We have to ask yourself if what we do is meaningful for the research -engineering just for engineering is not the way to go<br />
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<b>Statistician view</b> -poor quality of clinical trials in phase II compromises in fact the entire protocol design -should really learn from phase II in order to design properly phase III<br />
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<b>''Just think!...'' </b><span style="font-weight: normal;">(Martin Landray)</span>Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-682299504859252742016-04-08T05:10:00.001-07:002016-04-08T15:20:15.539-07:00IMI2 project ADAPT SMART<br />
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<span style="font-family: "verdana" , sans-serif;">Friday, 14.00- 15.30</span><br />
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<span style="font-family: "verdana" , sans-serif;">Session to give an update on ADAPT SMART is an enabling platform for the coordination of MAPPS (<span style="background-color: white; color: #344248; font-size: 14px;">Medicines Adaptive Pathways to Patients</span><span style="background-color: white; color: #344248; font-size: 14px;"> ) previously called Adaptive LicensingMPA</span></span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>http://adaptsmart.eu</b></span><br />
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<span style="font-family: "verdana" , sans-serif;"><b><i>Definition</i></b></span></h4>
<span style="font-family: "verdana" , sans-serif;">MAPPS are a concept that, based on scientific innovation, procide </span><b style="font-family: Verdana, sans-serif;">timely </b><span style="font-family: "verdana" , sans-serif;">access to specific medicines, with a potential to address </span><b style="font-family: Verdana, sans-serif;">unmet need</b><span style="font-family: "verdana" , sans-serif;">(s) in a specific, well-defined patient populations in a </span><b style="font-family: Verdana, sans-serif;">sustainable way</b><span style="font-family: "verdana" , sans-serif;">.</span><br />
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<span style="font-family: "verdana" , sans-serif;"><a href="http://adaptsmart.eu/" target="_blank">ADAPT SMART</a> is a support tool to MAPPS.</span><br />
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<span style="font-family: "verdana" , sans-serif;">Ad Schuurman- Dutch payer</span></h4>
<span style="font-family: "verdana" , sans-serif;">Very interesting to listen to a payer's perspective on MAPPS and the specific issues a payer faces. Volume control</span><br />
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<span style="font-family: "verdana" , sans-serif;">- prevent decline in quality of evidence</span><br />
<span style="font-family: "verdana" , sans-serif;">- cost control</span><br />
<span style="font-family: "verdana" , sans-serif;">- want to restrict MAPPS to special cases:</span><br />
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<li><span style="font-family: "verdana" , sans-serif;">patients who cannot wait</span></li>
<li><span style="font-family: "verdana" , sans-serif;">deteriorate irreversibly or die</span></li>
<li><span style="font-family: "verdana" , sans-serif;">urgent public health protection</span></li>
<li><span style="font-family: "verdana" , sans-serif;">major improvement expected</span></li>
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<span style="font-family: "verdana" , sans-serif;">realistic exit strategy.</span></div>
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<span style="font-family: "verdana" , sans-serif;"><br /></span>Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-84636437888096475512016-04-07T08:26:00.000-07:002016-04-07T08:26:00.250-07:00<h2>
Start-ups with regulatory and industry- small biotech companies potential to develop treatments - Session 0104</h2>
How can idea from academia can be better translated to novel treatment options<br />
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Important to give a chance to thesmall companies- they are the ones developing new compounds/drugs that are later bought by Big Pharma.<br />
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Small companies challenges (finances and innovation)<br />
New treatments paradigms<br />
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Andreas Schmidt CEO AYOXXA Biosystems Germany (35 people)<br />
interest in huge topics<br />
medical decision based on proteomics<br />
company interested in genomics and proteomics<br />
ambition to base clinical trials on proteomics<br />
access to clinical samples<br />
to identify which patients are going to respond (biomarkers)<br />
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Sam Wadsworth, Dimension Therapeutic<br />
2013 -company was funded<br />
partnership with big companies (e g. Bayer)<br />
partnership with academics<br />
partnership is bringing money and/or validation<br />
clinical trials- small trials (phase I, II)<br />
gene therapy -challenge for a small companies, few receive positive recommendation by EMA<br />
focus on one topic is better<br />
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Melanie Carr, EMA -European Medicine Agency<br />
why is important to support small companies<br />
90% -commercialised by big pharma<br />
no product commercialised by academia<br />
but the true is that less than half health technologies are coming from big companies<br />
incentives<br />
Innovation Task Force -early guidance how companies can be embarked in development programs<br />
Difficult to comply with EU regulations<br />
Plan Management Risks has to be included in the projects of small companies<br />
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Dr. . Kalkbrenner, Boehringer Ingelheim<br />
Company Bayer -life science companies -pharma component<br />
Grants4Apps, funding and supporting small companies (?)Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-63891667876830021472016-04-07T07:42:00.001-07:002016-04-07T08:12:05.811-07:00Adaptive Pathways and conditional approval<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Thursday, 16.00</span><br />
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<span style="font-family: "verdana" , sans-serif;">Francois Houyez</span><br />
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<span style="font-family: "verdana" , sans-serif;">Valid point</span><br />
<span style="font-family: "verdana" , sans-serif;">differences in compassionate usage programs between countries effectively create disparity in access across Europe, even between countries with similarly efficient healthcare systems. Francois mentioned 4 years- but need to verify in which setting.</span><br />
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<span style="font-family: "verdana" , sans-serif;">Advantages</span><br />
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<span style="font-family: "verdana" , sans-serif;">Disadvantages</span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>Tomas Salmonson</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">Comment-</span><br />
<span style="font-family: "verdana" , sans-serif;">conditional earlier approval can mak</span><span style="font-family: "verdana" , sans-serif;">e it difficult to recruit for the registration trials. </span><i style="font-family: Verdana, sans-serif;">And I wonder why that is!!! Guess the trial design is not attractive for patients? </i><br />
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<span style="font-family: "verdana" , sans-serif;"><b>Susan Forda</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">comparison between US and EU approvals of innovative therapies-</span><br />
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<span style="font-family: "verdana" , sans-serif;">for indications that let to special pathways for both sides.</span><br />
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<span style="font-family: "verdana" , sans-serif;">US has more fast access designations than the EU</span><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjUUQTMfhB6-4Dcs5wd0PaTsc2xioi0s7hWTkI79jWT96gP2E-221uFhyhcSt4-iWPAr0vg3D6W6zYbFeiRJ9ljXei6x7k0NNhV7IAiR5aX7MaadTgugf-lLHVPh87FX_ntdZxJ7qs3hGoS/s1600/IMG_2413.JPG" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" height="197" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjUUQTMfhB6-4Dcs5wd0PaTsc2xioi0s7hWTkI79jWT96gP2E-221uFhyhcSt4-iWPAr0vg3D6W6zYbFeiRJ9ljXei6x7k0NNhV7IAiR5aX7MaadTgugf-lLHVPh87FX_ntdZxJ7qs3hGoS/s320/IMG_2413.JPG" width="320" /></a></div>
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<span style="font-family: "verdana" , sans-serif;">US takes less time to approval than EU</span>
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<span style="font-family: "verdana" , sans-serif;"><span style="font-family: verdana, sans-serif;">BUT less post-marketing studies in the EU than in the US. </span></span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;"><b>And- faster approval does not lead to more withdrawls</b></span><br />
<span style="font-family: "verdana" , sans-serif;">So far, no withdrawl after accelerated approval/ conditional approval. In the US, none on break-through and one withdrawl for a priority review!</span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>Niklas Hedberg, TLV</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">used a great analogy of a stream to describe how new therapies come down after a damm (the regulators) and how the downstream tries to cope.</span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;"><b>Hans-Georg Eichler</b></span><br />
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<span style="font-family: "verdana", sans-serif;">Use of real-world data in the Adaptive Pathways pilots</span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>and again the very valid point that IF registries, they need to have the right quality</b></span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>Discussion</b></span><br />
<span style="font-family: "verdana" , sans-serif;">interesting enough, this is an adaptive pathway discussion and the first comment goes directly to drug prices. Niklas made the point that there is the worry that adaptive licensing effectively out-sources part of the development cost to society (instead of the company paying for it). </span><br />
<span style="font-family: "verdana" , sans-serif;">Very valid point by Tomas Salmonson- what is the point if we approve drugs for a wide indication (like HepC) but then only give them to a few patients because of price? That makes no sense from a public health perspective!</span><br />
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<span style="font-family: "verdana", sans-serif;">Niklas, TLV, suggestion to discuss conditions with a consortium of different Pharma working in that space to discuss the specific challenges and needs way before a single products hits the market.</span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com2tag:blogger.com,1999:blog-676754125166193231.post-6743514751542523642016-04-07T06:24:00.002-07:002016-04-08T12:50:06.095-07:00<h2>
<b>Why involve patients in Rare Disease Research</b></h2>
<div style="text-align: right;">
<b>The Genetic Alliance</b></div>
<div style="text-align: right;">
<b>Patient voice</b></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtzukfuGwz1cHIMc7TxEJebLWEQRGZth9rOgR01HH9tLiu8Bh-fLlkNycMWJ-K3aV7BcjfzSSlTDakOsfS5-QY5K7r4tXqVJAzm8Q7gHxP5G4MkHmeUSaAFML-oNOOPYRCLSC78yA4Wvs/s1600/Genetic+Alliance.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="180" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtzukfuGwz1cHIMc7TxEJebLWEQRGZth9rOgR01HH9tLiu8Bh-fLlkNycMWJ-K3aV7BcjfzSSlTDakOsfS5-QY5K7r4tXqVJAzm8Q7gHxP5G4MkHmeUSaAFML-oNOOPYRCLSC78yA4Wvs/s320/Genetic+Alliance.jpg" width="320" /></a></div>
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<b>Reasoning </b><br />
Rare disease condition<br />
understand the condition<br />
define the question<br />
plan the strategy<br />
recruit the volunteers<br />
validate the outcomes<br />
help secure implementation<br />
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we move in a era of full scientific advances<br />
with advance of personalised medicine-small and careful defined populations are much more likely to get benefits from the drug that is administered <br />
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Clinical data is based on number-are important-but what that numbers means in real life?<br />
Numerical changes have impact of quality of life for pts* and their families<br />
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we have NO interest in poor quality science<br />
Involvement in judging research proposal<br />
is this a relevant question for us?<br />
working with James Lind Alliance-involvement of pts pushed-up the quality of research<br />
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<b>How pts can help?</b><br />
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Research Priority Settings<br />
Donors -have interest to see where the money goes<br />
Registries<br />
Recruitment<br />
Realism -tangible deliverable related to our experience, to things that matter t us<br />
Sustainability<br />
Results -Eurogentest-what patients want to know, but not what clinicians think is the ought to go<br />
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Citizen jury<br />
Close relationship with the pts and their families<br />
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About EUPATI- expertise is needed; skills to have to conversation with regulators, clinicians, payers..etc.<br />
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<b>''We are not experts in everything but neither are you!''</b><br />
<i>We can tell you what is important for us, but not how to use the statistical tools -this is the job you are paid to do..</i><br />
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pts*-patients<br />
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<br />Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-60154503976150951432016-04-07T05:29:00.000-07:002016-04-07T05:29:00.606-07:00<h2>
Innovative ways of involving patients in RD</h2>
<div style="text-align: left;">
The industry view </div>
<div style="text-align: right;">
Tony Hoos, Amgen</div>
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<div>
<b>We all serve patients& society (?)</b></div>
Patient -consumer/Peer<br />
Patient involvement consideration<br />
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strategic document<br />
strategic purpose<br />
R-D. Life cycle (R_D, Life cycle management)<br />
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Where there opportunities for patients involvement<br />
insert slide (red)<br />
meaningful pts involvement -which stages<br />
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enhance pts access<br />
embrace pts centric healthcare<br />
facilitate coordination and integration of pts data -un-bias info<br />
acquire and develop talent<br />
do we have the right talent to interact with pts organisation?<br />
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evolution of pts engagement<br />
number of pts involved increased<br />
FDA strategic priorities<br />
<br />Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-28119917794811785982016-04-07T05:16:00.007-07:002016-04-07T06:29:17.353-07:00Shall HTA depend on RCT (randomised controlled trilas) or RWD (real world data) or both?<br />
<span style="font-family: "verdana" , sans-serif;"><b>Thursday, 14.00- 15.30</b></span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Chris Chinn, Head of Real World Investigations, Sanofi and <a href="https://www.imi-getreal.eu/" target="_blank">GetReal</a> </span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>
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<span style="font-family: "verdana" , sans-serif;">outcome- comparator- patients- intervention</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Outcomes are affected by patient behaviour, physician behaviour and the healthcare system and not all of these can be measured in a clinical trial.</span><br />
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<span style="font-family: "verdana" , sans-serif;">C</span><span style="font-family: "verdana" , sans-serif;">urrent- </span><br />
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<span style="font-family: "verdana" , sans-serif;"><b><span style="color: #cc0000;">RCT</span></b> to inform <b><span style="color: #cc0000;">clinical effectiveness</span></b></span><br />
<span style="font-family: "verdana" , sans-serif;"><b><span style="color: blue;">Observational study</span></b> for <b><span style="color: blue;">budget impact</span></b></span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Now</span><br />
<span style="font-family: "verdana" , sans-serif;">integrate both data sets using network meta analysis (<i>NESTA involved in this- worth checking out!</i>)</span><br />
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<span style="font-family: "verdana" , sans-serif;">TWiCS- run a randomised trial within an observational cohort, using the registry as a whole as control. <i>group in Manchester, failed to capture name</i></span><br />
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<span style="font-family: "verdana" , sans-serif;">Discussion-</span><br />
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<span style="font-family: "verdana" , sans-serif;">In the end, patients make <b>personal risk- benefit assessments</b>: are the benefits of the drug out-weighing the risks? That then affects the <b>effectiveness of the treatment</b>.</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;"><i>So in my mind, we would have to worry less about the gap between effectiveness (drug in the real world) and efficacy (drug in a clinical trial) if we paid more to those personal-risk assessments EARLY ON.</i></span>
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<h3>
<span style="font-family: "verdana" , sans-serif;">PAES- Post-Authorisation Efficacy Studies - applying RCT standards to RWD</span></h3>
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<span style="font-family: "verdana" , sans-serif;">Giovanni Tafuri, EMA</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">uncertainties about drug efficacy stem from:</span><br />
<ul>
<li><span style="font-family: "verdana" , sans-serif;">(sub-)populations studies</span></li>
<li><span style="font-family: "verdana" , sans-serif;">endpoints</span></li>
<li><span style="font-family: "verdana" , sans-serif;">long term use of a product</span></li>
<li><span style="font-family: "verdana" , sans-serif;">co-treatment with other products</span></li>
<li><span style="font-family: "verdana" , sans-serif;">real life usage</span></li>
<li><span style="font-family: "verdana" , sans-serif;">change in the understanding of a disease or a drug</span></li>
<li><span style="font-family: "verdana" , sans-serif;">change in scientific factors for previous efficacy </span></li>
</ul>
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<span style="font-family: "verdana" , sans-serif;">choice of study design depends on the need and can include:</span></div>
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<ul>
<li><span style="font-family: "verdana" , sans-serif;">randomised trials</span></li>
<li><span style="font-family: "verdana" , sans-serif;">non-randomised trials</span></li>
<li><span style="font-family: "verdana" , sans-serif;">explanatory trials</span></li>
<li><span style="font-family: "verdana" , sans-serif;">pragmatic trials</span></li>
<li><span style="font-family: "verdana" , sans-serif;">observational trials</span></li>
</ul>
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<span style="font-family: "verdana" , sans-serif;"><b>Data sources-</b></span></div>
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<span style="font-family: "verdana" , sans-serif;"><br /></span></div>
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<span style="font-family: "verdana" , sans-serif;">1. can be collected for the purpose: clinical trials</span></div>
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<span style="font-family: "verdana" , sans-serif;">2. analyze existing data- e.g. registries, electronic Health records</span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Importance to agree on a design prior to starting and the place of real world data during the life cycle of the product: worth noting the <b>registries</b>.</span><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhHqFeQ9pC_2Bo-6H3MGYgyzvBUABalTMkGLSrU257IUHJjPw0vwPCKX3-GATrU-k1V5dEuzLkdi6yHLkDFfhJBwnZEVi-EXlDzbB2yf3pQ9XEiLTSpEBrl8f3SKE6GdD46UqsN1ugy2SUj/s1600/IMG_2389.JPG" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" height="206" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhHqFeQ9pC_2Bo-6H3MGYgyzvBUABalTMkGLSrU257IUHJjPw0vwPCKX3-GATrU-k1V5dEuzLkdi6yHLkDFfhJBwnZEVi-EXlDzbB2yf3pQ9XEiLTSpEBrl8f3SKE6GdD46UqsN1ugy2SUj/s320/IMG_2389.JPG" width="320" /></a></div>
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;">The <a href="http://patientregistries.eu/" target="_blank">PARENT joint action</a> <b><span style="color: #45818e;">pa</span>tient <span style="color: #45818e;">re</span>gistry <span style="color: #45818e;">i</span>nitiative</b>: purpose to reduce duplication between registries. <i>Worth looking up!</i></span><br />
<span style="font-family: "verdana" , sans-serif;"><i><br /></i></span>
<span style="font-family: "verdana" , sans-serif;"><i><br /></i></span>
<span style="color: #cc0000; font-family: "verdana" , sans-serif;"><b>And important- who sets the standards for the registries?</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">Comment of Guido Rasi, EMA- we<b> need to define what real world data means</b>. </span></div>
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<h3>
<span style="font-family: "verdana" , sans-serif;">Conducting HTA using RWD (real world data)</span></h3>
<span style="font-family: "verdana" , sans-serif;">Francois Meyer, HAS</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Achievements of<a href="http://www.eunethta.eu/" target="_blank"> EUnetHTA</a></span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">1. Database EVIDENT - HTA bodies can find out what others have collected</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">2.</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">3. position papers on research recommendations</span><br />
<br />
<ul>
<li><span style="font-family: "verdana" , sans-serif;">how to best formulate research recommendations</span></li>
<li><span style="font-family: "verdana" , sans-serif;">how to decide on the appropriate study design to answer those questions</span></li>
</ul>
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<span style="font-family: "verdana" , sans-serif;">4. core protocol pilot for AEG (additional evidence generation)</span></div>
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<span style="font-family: "verdana" , sans-serif;">first pilot of a joint HTA process</span></div>
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<span style="font-family: "verdana" , sans-serif;"><i>would be worth looking this up- and see whether we agree or not</i></span><br />
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<span style="font-family: "verdana" , sans-serif;"><i><br /></i></span></div>
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<span style="font-family: "verdana", sans-serif;"><b>Discussion</b></span><br />
<span style="font-family: "verdana", sans-serif;"><br /></span>
<span style="font-family: "verdana", sans-serif;">Comment from <b>Chris Chin</b></span><br />
<span style="font-family: "verdana", sans-serif;">When to set up a registry? At launch of a product is too late- one should start earlier, so that there is a good quality registry in place at launch and one can focus on answering questions!</span><br />
<span style="font-family: "verdana", sans-serif;"><br /></span>
<span style="font-family: "verdana", sans-serif;"><b>Luca Pani, AIFA</b></span><br />
<span style="font-family: "verdana", sans-serif;">differential MEA in place in Italy with the existing registry - a different MEA (management entry agreement) for every indication of the same drug</span><br />
<span style="font-family: "verdana", sans-serif;">but</span><br />
<span style="font-family: "verdana", sans-serif;">it has been a major investment for the healthcare system to have a registry of this granularity and quality standards but he thinks it has paid of!</span><br />
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<br />Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com2tag:blogger.com,1999:blog-676754125166193231.post-67549171441329278722016-04-07T03:12:00.000-07:002016-04-07T05:07:08.476-07:00<h2>
What are the challenges of conducting pragmatic trials (PCTs)?</h2>
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<b>Iris Goetz</b></div>
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Global Health Outcomes</div>
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Eli Lilly, UK</div>
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<b>Get REAL PRoject </b><br />
<b>IMI Work Package WP3 </b></div>
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<b>Focus on randomisation</b></div>
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RCT in real world !</div>
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<b>Aim</b></div>
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consequences of design</div>
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consequences of design choice</div>
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<b>key activities</b></div>
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ensure feasibility while keeping validity</div>
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literature review</div>
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practical solutions</div>
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toolbox</div>
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stakeholders interviews</div>
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<b>Definition of pragmatic trials -various- </b></div>
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mainly trials comparing different health interventions -effectiveness</div>
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<b>Comparing RCT (randomised clinical trials) with PCT (pragmatic clinical trials)</b></div>
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<b>Which design criteria important in designing a PCT?</b></div>
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<b>Comparison of Treatments Arms -questions to be answered</b></div>
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Baseline-do we need wash-out period?</div>
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link hospital data with the GP data</div>
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Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-50737098365488815062016-04-07T02:49:00.003-07:002016-04-07T02:49:44.479-07:00<h2>
How well can the pre-launch study design provide information on the relative effectiveness of a new medicine </h2>
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<b>François Mayer</b></div>
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HAS France </div>
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<b>Assessment model of drugs in (HTA) -</b></div>
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<b>study conducted in France</b></div>
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SMR- service medical <i>rendu </i></div>
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Drug eligibility reimbursement</div>
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Added therapeutic benefit </div>
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5 ASMR levels model</div>
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major</div>
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important </div>
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minor</div>
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no improvement </div>
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post launch of observational studies</div>
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OS results are considered </div>
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comparable</div>
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un-interpretable</div>
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not comparable</div>
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<b>Reassessment SMR/ASMR</b></div>
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Clinical effectiveness</div>
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Safety</div>
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Quality of life </div>
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Improvement of is quite rare post launch! </div>
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Many cases when results do not confirm what was observed during RCTs!</div>
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Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-61773108373776701382016-04-07T02:25:00.000-07:002016-04-07T02:53:14.832-07:00<h2>
Real World Evidence in Drug Development</h2>
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Chris Chinn -Sanofi </div>
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Acceptability of real world evidence<br />
R-D decision anticipate the HTA decision<br />
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phase 3 optimise<br />
phase 3b supplement<br />
conditional licensing<br />
conditional access<br />
phase IV<br />
''commit'<br />
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Would we accept the uncertain for a period of time while waiting for the study to complete?<br />
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<b>R-D decision</b><br />
to what extend generate the study plan generate data for effectiveness?<br />
the concern of pharma/producer of health technology : will the study be accepted by regulators and HTA agencies?<br />
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<b>IMI Get real Work Packages</b><br />
acceptability<br />
understading the efficacy-effectiveness gap<br />
clinical design barriers<br />
best practices-predictive models<br />
practical feasibility<br />
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<b>Questions asked at workshop of Pragmatic Design of Clinical Trials</b><br />
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<b>Get Real deliverables</b><br />
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<b>Raising acceptability</b><br />
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Violeta Astratineihttp://www.blogger.com/profile/12687227276797631227noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-48211753357085966002016-04-07T02:10:00.003-07:002016-04-07T03:26:55.545-07:00The needs of the payers shape the evidence for market access<br />
<span style="font-family: "verdana" , sans-serif;"><b>Thursday, 11.00- 12.30</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">How Companies fit all evidence requirements into one development plan</span></h3>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiIqBMHcy2LKd75OadPR9E8uHqp8cMqShKfo2y7CDeQZE7W8Xk0tFHjmHOb-4HjrkvGwoJEbF87jt3yNzYm3a48HvlIxmnngZ7K4S5dPu_ZFkRipFNioeuaww-LBQtBQEf5Fy1aSLj1siOF/s1600/Screen+Shot+2016-04-07+at+11.18.24.png" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" height="137" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiIqBMHcy2LKd75OadPR9E8uHqp8cMqShKfo2y7CDeQZE7W8Xk0tFHjmHOb-4HjrkvGwoJEbF87jt3yNzYm3a48HvlIxmnngZ7K4S5dPu_ZFkRipFNioeuaww-LBQtBQEf5Fy1aSLj1siOF/s320/Screen+Shot+2016-04-07+at+11.18.24.png" width="320" /></a></div>
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<span style="font-family: "verdana" , sans-serif;">How all evidence requirements (regulators, HTA, payers) fits into one development plan for one product- first talk. So this decides what type of clinical trials we get to see!</span><br />
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<span style="font-family: "verdana" , sans-serif;">Problem is: requirements for regulatory approval- EMA- are different from what HTA bodies (like NICE/ UK or ZIN/ NL) or payers ( like health insurers) want. </span><br />
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<span style="font-family: "verdana" , sans-serif;">It has happened that HTAs rejected regulatory assessment = no access despite drug is approved.</span><br />
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<span style="font-family: "verdana" , sans-serif;">Marlene Gyldmark/ Roche is speaking how the company attempts to integrate how the generate evidence that fulfils BOTH regulatory AND HTA requirements. </span><br />
<span style="font-family: "verdana" , sans-serif;"><i>Personally, I would always assumed that this was done already...? Why would you want to risk to invest so much and then get stuck so late in the process? But then I'm sure there is something I missed- in any case, NOT good for patients either....</i></span><br />
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<span style="font-family: "verdana" , sans-serif;">Trying to come up with ONE strategy to fulfil differing HTA requirements- referring to the <a href="http://www.eunethta.eu/hta-core-model" target="_blank">HTA Core Model </a>® from <a href="http://www.eunethta.eu/" target="_blank">EUnetHTA</a>- <i>so it is good to see that harmonisation efforts on the HTA side are showing effect! </i></span><br />
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<span style="font-family: "verdana" , sans-serif;">Interesting how to combine different types of evidence: clinical trials, real world data and modelling to have an integrated evidence plan. The challenge is integrating three different evidence needs from regulatory, HTA and medical</span><br />
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<span style="font-family: "verdana" , sans-serif;">How will payers react to the future of Drug Development?</span></h3>
<span style="font-family: "verdana" , sans-serif;">Steffen Thirstrup, UK</span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>Health care systems</b> are now the main customers for regulatory and HTA evaluation </span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>RWD</b> has replaced clinical trials as the main source for evidence about effectiveness (and safety)</span><br />
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<span style="font-family: "verdana" , sans-serif;">problem- regulators and HTA haven't developed methods how to use this RWD for evidence-generation</span><br />
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<span style="font-family: "verdana" , sans-serif;">Development of early HTA advice: </span><br />
<span style="font-family: "verdana" , sans-serif;">competition among reimbursement and payer agencies, so where to turn?</span><br />
<span style="font-family: "verdana" , sans-serif;"><i>Hopefully, EUnetHTA will change some of that!!</i></span><br />
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<span style="font-family: "verdana" , sans-serif;"><b><a href="http://www.eunethta.eu/seed" target="_blank">SEED Consortium</a></b> (14 EU HTA agencies, coordinated by HAS)- early dialogue processes- worth looking up</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;"><b><a href="https://www.imi-getreal.eu/" target="_blank">GetREAL</a> </b></span><br />
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<span style="color: #6aa84f; font-family: "verdana" , sans-serif;"><b>Very nice summary slide on HTA collaboration :-) </b></span><br />
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<span style="font-family: "verdana" , sans-serif;">problem remains that health and finances are under national authority.</span><br />
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<span style="font-family: "verdana" , sans-serif;">Interesting suggestion to have <b>conditional reimbursement</b>- <i>something worth thinking about!</i></span><br />
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<h3>
<span style="font-family: "verdana" , sans-serif;">How can a joint regulatory-HTA scientific advice process help deliver the right evidence?</span></h3>
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<span style="font-family: "verdana" , sans-serif;">Jane Moseley, EMA</span></div>
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<span style="font-family: "verdana" , sans-serif;">Interesting case study from joint advice: large company, small indication, unmet need-</span></div>
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<span style="font-family: "verdana" , sans-serif;">original recommendation was a placebo-controlled study designed.</span></div>
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<span style="font-family: "verdana" , sans-serif;">After multi-stakeholder discussion including EMA, 4 HTA bodies, clinicians, patients and the Pharma let to the adaptation of a different design.</span></div>
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<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;"><a href="https://embed.topra.org/sites/default/files/regrapart/1/6026/parallel_eta-hta_advice-moseley-warner-sibal.pdf" target="_blank">Interview</a> with Jane Moseley on the topic</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;">- Very valid comments</span><br />
<span style="font-family: "verdana" , sans-serif;">- shared EMA-HTA advice is in patients' interest</span><br />
<span style="font-family: "verdana" , sans-serif;">- advice needs to happen EARLY to be efficient and guidelines are not sufficient</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;">And yet another case where there is a willingness to alter trial design to a more patient-friendly design: no placebo as the indication was small with high unmet need 'to reduce patient suffering'. </span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;"><i>I can't get my head around that argument- in a large indication with high unmet need (where we can get the data), we insist in placebos. In a small indication with high unmet need (where we can't get the data), we are worried about the patient impact. </i></span><br />
<span style="font-family: "verdana" , sans-serif;"><i>That means that the suffering of a patient with in a small indication is worse than the one of a patient with a large indication. This makes no sense whatsoever!!!! </i></span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;">Comment in the discussion-</span><br />
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<span style="font-family: "verdana" , sans-serif;">we should not only discuss market entry but also <b>market exit</b> strategies</span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span>Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-51569063236564356792016-04-06T07:29:00.003-07:002016-04-07T02:00:11.972-07:00Opening Plenary Session INNovation- do you win by being IN?<br />
<span style="font-family: "verdana" , sans-serif;">Discussion about the Value of Innovation and impact on our society!</span><br />
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<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;">1. How <b>global innovation</b> </span><br />
<span style="font-family: "verdana" , sans-serif;">will be <b>financially sustained </b></span><br />
<span style="font-family: "verdana" , sans-serif;">for <b>future decades</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">Discussants</span><br />
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<span style="font-family: "verdana" , sans-serif;">Mads Krogsgaard Thomson - Novo Nordisk</span><br />
<span style="font-family: "verdana" , sans-serif;">Sarah Garner- NICE</span><br />
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<span style="font-family: "verdana" , sans-serif;">Mads makes the argument that YES- innovation is a long-lasting process and happens in waves and with newer therapies coming up in richer countries, previous waves spread to less wealthy countries so that over time, global society profits.</span><br />
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<span style="font-family: "verdana" , sans-serif;">Sarah Garner is making a long list of very valid points - I will get her notes as it's too much to listen and type!!</span><br />
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<span style="font-family: "verdana" , sans-serif;"><b>2 .True cost of innovation</b></span><br />
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<span style="font-family: "verdana" , sans-serif;">Karl Broich- president BfArM</span><br />
<span style="font-family: "verdana" , sans-serif;">Nicola Bedlington, EPF</span><br />
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<span style="font-family: "verdana" , sans-serif;">Nicola makes the point that innovation has to be sustainable. Meaningful patient engagement requires educational resources for patients! </span><br />
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<span style="font-family: "verdana" , sans-serif;">3. Use of <b>patient data</b> for <b>innovation</b>, <b>challenges</b> and <b>ethical dilemmas</b>.</span><br />
<span style="font-family: "verdana" , sans-serif;"><br /></span><span style="font-family: "verdana" , sans-serif;">Ritva Halila, ministry of social affairs and health, Finland</span><br />
<span style="font-family: "verdana" , sans-serif;">Kemal Malik, head of innovation, Bayer </span><br />
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<span style="font-family: "verdana" , sans-serif;">Kemal on the role of patient in clinical development: </span><br />
<span style="font-family: "verdana" , sans-serif;">'patient engagement seen as pre-marketing exercise' </span><br />
<span style="font-family: "verdana" , sans-serif;">'lack of clear of evidence of patient engagement'</span><br />
<span style="font-family: "verdana" , sans-serif;">a key future trend will be to show real life outcomes</span><br />
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<br />Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com1tag:blogger.com,1999:blog-676754125166193231.post-76566760492709062892016-04-06T07:16:00.001-07:002016-04-06T07:16:24.665-07:00Regulatory Town Hall meeting<span style="font-family: Verdana, sans-serif;"><br /></span>
<span style="font-family: Verdana, sans-serif;">Wednesday, 5th April</span><br />
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<span style="font-family: Verdana, sans-serif;">A town hall meeting on the topic of the Network Strategy 2020. </span><br />
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<span style="font-family: Verdana, sans-serif;"><br /></span>Bettinahttp://www.blogger.com/profile/02103075738345492061noreply@blogger.com0tag:blogger.com,1999:blog-676754125166193231.post-79894877299318503952016-04-06T07:08:00.002-07:002016-04-06T07:14:02.088-07:00DIA2016<span style="font-family: "verdana" , sans-serif;"><br /></span>
<span style="font-family: "verdana" , sans-serif;"><a href="http://www.diaglobal.org/en/flagship/euromeeting-2016" target="_blank">DIA Europe</a> is taking place in Hamburg 6- 8th April 2016 and we have just arrived! </span><br />
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<span style="font-family: "verdana" , sans-serif;">Violeta and Bettina </span><br />
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