01:05:58 Michael: As an engineer I’ll take advantage of Ben’s reference to sewers b/c it’s my understanding that it’s considered the greatest contribution to health in all of history. I am surprised at the use of terms like “safe.” Engineers use something called “safety cases” to determine whether technologies are “safe ENOUGH.” I would encourage that a lot of energy be put into this. I am familiar with (originally) British standards called EN 50126, 50128 and 50129. Also, safety cases are live documents these days. They are continuously updated. The idea that medications like antidepressants would be understood to be “safe” by clinicians based on 6 week trials done in the 1980s and 1990s is surprising. Certain truths about the reality of withdrawal effects are considered obtained in the patient community, but there continue to be epic wars conducted in social media which damage everyone. 01:06:10 David Colquhoun: There is huge pressure to use non-randomised evidence. That would lead to huge expense for little gain (DAGs won't prevent that) 01:07:43 Anja Schiel: The problem is that drug developers only want to provide evidence for their product, the strength of RWD/RWE lies in generating knowledge 01:09:06 Francois Maignen: I love the expression "real world" evidence … I am desperately trying to find intersideral or black holes evidence … (only joking). 01:09:47 Michael: “Ww are just beginning to consider real world evidence” - respectfully, if engineers behaved this way, the whole world would be on fire. 01:11:18 Eric Low: RWE has a role, but it shouldn't just be used to bridge evidence gaps as a result of poorly designed trials or a trial designed to answer only regulatory questions for the FDA/US market. I like Ken's take on it, that it should be used to figure out if a new medicine really works in a real world setting, not just in a single arm trial with ~80 arefully selected patients and 6 months follow up etc. 01:11:28 David Colquhoun: The term "real world evidence" is loved by the trillion dollar "wellness" and supplement industries -because they have a financial interest in poor evidence :-( 01:12:49 Francois Maignen: True ... 01:13:34 Eric Low: I agree , Ken. Good points. 01:15:54 Anja Schiel: To me it seems that orphan drugs have become more interesting because science allowed to development with less investment and the opportunity to as fantasy prices and possibly get away with it 01:18:34 Francois Maignen: I believe that with the MHRA rubber stamping some authorisations from the FDA as from January next year that NICE might have to reconsider its decision making processes and may be consider not reimbursing products with marginal added clinical value ... 01:19:15 David Colquhoun: It would take 20 years to get evidence only if the effect size were small 01:20:07 Ben Purchase: Replying to "If we want to have b..." This might negate the point of a threshold. A counter would be that most clinical benefits of wonder drug would drive it being under a threshold, and so that in itself will allow a company to charge a higher price (the reward?) to push their wonder drug closer to threshold. 01:20:37 Francois Maignen: I know ... 01:21:24 Michael: Replying to "The term "real world..." I’m a trained teacher of mindfulness and am in total agreement that there is a deep amount of nonsense there. I no longer teach it. But please understand that some advocates of what the term RWE means at face value is supported by people who have forgotten more math than seems ever to have been discussed at HTA conferences. HTA in its current form is not recognizable to people who are serious about functional and system safety. 01:24:14 Ben Purchase: Does the panel consider that there are genuine logistical and practical challenges for MedTech developers in terms of conducting RCTs versus RWE and that if still maintaining hierarchy of evidence, there's a risk NICE goes behind curve as these tech are being adopted anyway regardless of what NICE says 01:25:57 Eric Low: Good point, Richard. I would take money from the CDF and IMF and give it to MHRA and NICE to ensure they have the resources and capacity needed. I think the billions spent by NIHR, CRUK and AMRC members on clinical research should be leveraged to help partner with industry to generate in-country data ahead of HTA to reduce uncertainties and de-risk the FDA study. 01:27:16 seren phillips: New technologies such as gene therapies may open up the possibility to treat many rare diseases - many of which affect children. there will be many evidence uncertainties: evidence generation hard, , heterogenous population, generating utilities in children etc. It won’t usually be the case that dramatic differences can be shown since many patients will often be treated at later stages in their disease as diagnosis is usually delayed . does this mean that many rare disease treatment will never be commercially viable ? 01:27:59 Francois Maignen: It was a fantastic discussion … Thxs to the panel!!!
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01:05:58 Michael: As an engineer I’ll take advantage of Ben’s reference to sewers b/c it’s my understanding that it’s considered the greatest contribution to health in all of history.
I am surprised at the use of terms like “safe.” Engineers use something called “safety cases” to determine whether technologies are “safe ENOUGH.” I would encourage that a lot of energy be put into this. I am familiar with (originally) British standards called EN 50126, 50128 and 50129.
Also, safety cases are live documents these days. They are continuously updated. The idea that medications like antidepressants would be understood to be “safe” by clinicians based on 6 week trials done in the 1980s and 1990s is surprising. Certain truths about the reality of withdrawal effects are considered obtained in the patient community, but there continue to be epic wars conducted in social media which damage everyone.
01:06:10 David Colquhoun: There is huge pressure to use non-randomised evidence. That would lead to huge expense for little gain (DAGs won't prevent that)
01:07:43 Anja Schiel: The problem is that drug developers only want to provide evidence for their product, the strength of RWD/RWE lies in generating knowledge
01:09:06 Francois Maignen: I love the expression "real world" evidence … I am desperately trying to find intersideral or black holes evidence … (only joking).
01:09:47 Michael: “Ww are just beginning to consider real world evidence” - respectfully, if engineers behaved this way, the whole world would be on fire.
01:11:18 Eric Low: RWE has a role, but it shouldn't just be used to bridge evidence gaps as a result of poorly designed trials or a trial designed to answer only regulatory questions for the FDA/US market. I like Ken's take on it, that it should be used to figure out if a new medicine really works in a real world setting, not just in a single arm trial with ~80 arefully selected patients and 6 months follow up etc.
01:11:28 David Colquhoun: The term "real world evidence" is loved by the trillion dollar "wellness" and supplement industries -because they have a financial interest in poor evidence :-(
01:12:49 Francois Maignen: True ...
01:13:34 Eric Low: I agree , Ken. Good points.
01:15:54 Anja Schiel: To me it seems that orphan drugs have become more interesting because science allowed to development with less investment and the opportunity to as fantasy prices and possibly get away with it
01:18:34 Francois Maignen: I believe that with the MHRA rubber stamping some authorisations from the FDA as from January next year that NICE might have to reconsider its decision making processes and may be consider not reimbursing products with marginal added clinical value ...
01:19:15 David Colquhoun: It would take 20 years to get evidence only if the effect size were small
01:20:07 Ben Purchase: Replying to "If we want to have b..."
This might negate the point of a threshold. A counter would be that most clinical benefits of wonder drug would drive it being under a threshold, and so that in itself will allow a company to charge a higher price (the reward?) to push their wonder drug closer to threshold.
01:20:37 Francois Maignen: I know ...
01:21:24 Michael: Replying to "The term "real world..."
I’m a trained teacher of mindfulness and am in total agreement that there is a deep amount of nonsense there. I no longer teach it.
But please understand that some advocates of what the term RWE means at face value is supported by people who have forgotten more math than seems ever to have been discussed at HTA conferences. HTA in its current form is not recognizable to people who are serious about functional and system safety.
01:24:14 Ben Purchase: Does the panel consider that there are genuine logistical and practical challenges for MedTech developers in terms of conducting RCTs versus RWE and that if still maintaining hierarchy of evidence, there's a risk NICE goes behind curve as these tech are being adopted anyway regardless of what NICE says
01:25:57 Eric Low: Good point, Richard. I would take money from the CDF and IMF and give it to MHRA and NICE to ensure they have the resources and capacity needed. I think the billions spent by NIHR, CRUK and AMRC members on clinical research should be leveraged to help partner with industry to generate in-country data ahead of HTA to reduce uncertainties and de-risk the FDA study.
01:27:16 seren phillips: New technologies such as gene therapies may open up the possibility to treat many rare diseases - many of which affect children. there will be many evidence uncertainties: evidence generation hard, , heterogenous population, generating utilities in children etc. It won’t usually be the case that dramatic differences can be shown since many patients will often be treated at later stages in their disease as diagnosis is usually delayed . does this mean that many rare disease treatment will never be commercially viable ?
01:27:59 Francois Maignen: It was a fantastic discussion … Thxs to the panel!!!