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Gerry Smith at Bloomberg writes on how self-insured employers are valuing the lives of employees when it comes to gene therapy for rare diseases. https://lnkd.in/dnMB4SST We took this up last week, and the problem of forecasting the duration of therapeutic benefits in the real world. If you didn’t catch it then, see our ISPOR—The Professional Society for Health Economics and Outcomes Research Europe poster - Generalizing the Distribution and Dynamics of Health Outcomes in Clinical Trials for Novel Treatment of Rare Disease https://lnkd.in/e5Qbuh4A from Devin Incerti, Darius Lakdawalla, Patricia Dorling, PharmD, MS, PhD, Partha Deb, Edward Vytlacil, Andrew Morris, and Joe Cappelleri. (thanks Brian Reid for sharing Gerry’s piece earlier, and if you don’t follow Brian, you should) #GeneTherapy

55 days until the election. Regardless of the outcome Neal Masia says the thing to watch is “this question around what counts as value. And are we really going to think seriously about patient preferences, and caregiver preferences, and the long run path. Or are we going to think about this year’s budget.” Watch the most recent Demy-Colton session here with Greg Simon, Karen Deschaine, Evonne Sepsis and Peter L. Rubin - https://lnkd.in/g_C3uBjN. Jump to 36:55 to hear about how this “fork in the road from a patient perspective is really about what counts as value.”

James M. Wilson is launching Gemma Biotherapeutics and Franklin Biolabs with the ambition of seeing a better commercialization model for gene therapy take shape. One problem is forecasting the duration of therapeutic benefits in the real world. We took a look at that in our ISPOR—The Professional Society for Health Economics and Outcomes Research Europe poster - Generalizing the Distribution and Dynamics of Health Outcomes in Clinical Trials for Novel Treatment of Rare Disease https://lnkd.in/e5Qbuh4A. Devin Incerti, Darius Lakdawalla, Patricia D., Partha Deb, Edward Vytlacil, Andrew Morris, and Joe Cappelleri found – The prediction framework detailed captures structural uncertainty. It generalizes to the real-world population. We can generate the full distribution of predicted outcomes, rather than just means. We can enable extrapolation of baseline health risk and treatment effects into the future. We provide evidence to inform outcomes-based contracting and parameterize disease models for cost effectiveness analysis. And this can be applied to a wide variety of other rare disease estimation problems. #genetherapy

Our CEO Neal Masia and one of our favorite customers/thought partners Peter L. Rubin of No Patient Left Behind will be discussing how the upcoming election will shape the health policy landscape this Wednesday. For free! Join them for what will surely be an entertaining and informative hour.

Insightful analysis from Jared Hopkins and colleages. It is an open question whether patients will save a penny - in fact, the follow on implications for formulary placement of competitors make it unclear whether weighted average prices will go up or down for those who may not want to switch therapy, for example. Add to this the opaque nature of the proceedings and it’s hard to imagine a stranger approach. The only certainty is that this has added to uncertainty for investors in the space. Our #gcea and #heor tools can help #marketaccess teams make better decisions in a very uncertain environment.

This nice work by Lou Garrison, Boshen Jiao, Jens Grueger and other smart people shows that even using traditional QALYs, society and consumers realized the vast majority of benefits from Hep-C treatments. At the time of launch 10 years ago, industry critics could not catch their breath worrying about the potential for these new drugs to "bust" state budgets, Medicare Part D, etc. Sound familiar? https://lnkd.in/egKapnr6

Recent signals about biotech fundraising have been mixed. While the amount raised in Q1, for example, was bigger than the average quarterly fundraise last year, the number of deals, especially early-stage deals, was the lowest in more than five years.  In an insightful analysis, the Financial Times lists rising biotech stock prices, increasing sentiment that interest rates will gradually decrease, and an uptick of M&A activity in the sector among the factors contributing to optimism. Yet we are still far from the “heady days” during the pandemic when investing in biotech stocks would rake in profits “regardless of the quality of a company’s pipeline or balance sheet,” Andy Acker and Dan Lyons of Janus Henderson Investors told the FT. Much of the M&A enthusiasm, for example, may be influenced by a single deal, Amgen’s acquisition of Horizon Therapeutics last year, they added.  Check out the full FT story here: https://lnkd.in/dkZpKSYN  And reach out to learn more about how we can help you can model biotech uncertainty better.  #HEOR #Innovation #BioPharma #HealthEconomics #DataScience 

A student-run non-profit whose mission is to empower the next generation of medical innovators is attracting increasing attention in the biotech industry. In the five years since its founding, Nucleate has spawned hundreds of successful collaborations which have raised more than $300M in early-stage funding and created over 200 jobs.  “What we started noticing is that in biotech entrepreneurship [...] you have the need for risk-tolerant capital [...] but then there was really this gap missing in social capital,” the organization’s then-VP of Partnerships, Sonia Maryam Setayesh, MS, PhD, told a recent episode of the National Cancer Institute (NCI) Institute’s Inside Cancer Careers podcast. “So once we saw that there were really not many resources that people could go to get equity-free education on social capital, this is where Nucleate was born out of.”  Setayesh herself went on to become an Investment Partner at the $9.7B VC fund Civilization Ventures after completing her Ph.D. in Molecular Biology from the University of Southern California last year. During her studies, she discovered a blood-based liquid biopsy platform for the early detection of breast cancer (and worked on several other exciting projects), which is how she was first introduced to Nucleate. We highly recommend the podcast and the insightful input from the other speakers, Manifold Bio’s Co-Founder and CEO Gleb Kuznetsov, Persephoni BioPartners’ Senior Associate (and VanguardSTEM’s Director of Funding) Geraldine Ezeka, PhD, and the Global Head of Nucleate’s Activator, Alex Kim.  Check out the full episode here: https://lnkd.in/gk-bFX-b  #HEOR #Innovation #DrugPricing #BioPharma #HealthEconomics 

There are ways to reconcile the need for affordable healthcare now with the future needs of patients who would benefit from medical innovation. The key is having a consistent and scientifically sound method to determine the value of drugs.     Those are some of the big takeaways for us from a panel of legislators and scientists in which our Co-Founder and Scientific Advisor Dana P Goldman took part.  "While patients may see a lot of benefits at the counter today, long-term access to treatment will be hindered," Dana said with reference to criticism of the Inflation Reduction Act and the unprecedented Medicare drug price negotiations.  Some aspects of the law may need to be revised to protect innovation and keep drugmakers in the US, legislators who participated in the event admitted.  Check out Fierce Healthcare’s story about the debate here: https://lnkd.in/gYsTD8Nw #HEOR #Innovation #DrugPricing #BioPharma #HealthEconomics

A little over a month ago, news that a bispecific antibody—ivonescimab, produced by Summit Therapeutics and Akeso—had performed well in a Phase 3 non-small cell lung cancer clinical trial, causing a big stir in the biotech community. Celebrated as a success for cancer immunotherapy, Keytruda, which was initially approved by the FDA in 2014 for the treatment of melanoma (and has since been used for the treatment of a host of other cancers), is well known. Luba Greenwood, J.D., the Managing Partner of the Dana Farber Cancer Institute Venture Fund, Binney Street Capital, captured the moment in Episode 103 of the Biotech Hangout podcast.  “I would say probably in the last two-three years there hasn’t been that much interest in investment, where people are turning up their nose,” and doubted the incumbent therapy could be surpassed, Greenwood said.   Greenwood then pointed the conversation toward patient needs and the value of new therapies, which is one of the drivers for our model, for knowing uncertainty in biotech development and investing wisely. Listen to the full podcast, which features also Daphne Zohar, Josh Schimmer, Yaron Werber, and Dawn Bell, PharmD.    Check it out here if you haven’t already: https://lnkd.in/g7R8te5r    #HEOR #Innovation #DrugPricing #BioPharma #HealthEconomics