Benjamin Pierce’s Post

Many thanks to Betsy Goodfellow at Pharmafile.com for featuring an article by Dr Dan Williams. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. The full story is here: https://lnkd.in/e_58twVs

Important article in Pharmafile.com by our client Dr Dan Williams at SynaptixBio Ltd. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. Many thanks to Betsy Goodfellow. The full article is here: https://lnkd.in/e_58twVs.

#DiagnosticEfficiency👩🏼🔬 The KDIGO 2024 Clinical Practice Guideline for Chronic Kidney Disease acknowledges cystatin C advantages looking into patient groups benefitting from combined testing of #cystatinC and #creatinine. Learn more: 

'Severity of disease modifiers' are a focus for questions about access to medicines, but are we missing the bigger picture? NICE committees define the acceptable ICER between £20k and £30k, taking account of uncertainty in the ICER and uncaptured benefits. I estimate 76 published HTAs have used these rules since 2022. 17 were CCAs and in 19 an acceptable ICER was not stated. For the other 40, in 22 the acceptable ICER was set at the lower end of the range, 6 were around the middle and 10 were at the upper end (three were explicitly at £30k). The remaining two were below £20k. Using the values stated and assuming 'lower end of the range' means £22.5k, etc: The average acceptable ICER was £23.5k (median £22.5k). For cancer HTAs £24k and for others £22.8k. For guidance published in 2024 it was £24.1k and for 2022-3 it was £22.8k. Whether the SoD modifer is 1.2, 1.4 or 1.7 probably makes less difference than whether the decision is based on an acceptable ICER of £20k (or lower) or £30k.

A new analysis of phase 3 GRIPHON study data evaluated changes in risk status for pulmonary arterial hypertension (PAH) patients using the REVEAL 2.0 risk calculator from the Registry to Evaluate Early and Long-Term PAH Disease Management. Read more: https://lnkd.in/eWmGVm2W #RareDisease

Exciting news for the Immunoglobulin Industry! 🌟 With a projected CAGR of 7.4%, this sector is set for steady growth, driven by the rising prevalence of immunodeficiency diseases and the growing adoption of immunoglobulin therapies. 💉 Stay tuned for advancements improving immune system support and patient outcomes! #Immunoglobulin #Immunodeficiency #MedicalTherapies

Patients with seronegative IgG4-related disease (IgG4-RD) exhibit distinct clinical patterns compared to other patients with IgG4-RD, including lower serum IgG levels and eosinophil counts, and fewer affected organs, according to a study. Read the full article here ⤵️ #AutoimmuneDisease #Autoimmune #ChronicDisease

Our week starts with a promising piece of news for the #DINcommunity #CIDP #dysimmuneinflammatoryneuropathies #cureDINs #treatDINs #peoplelivingwithDINs #DINpeople #clinicaltrials #medicalresearch

A Phase 3 trial evaluated the use of bulevirtide in patients with chronic hepatitis D. The primary combined end-point response at week 48, defined as an undetectable HDV RNA level or a level that decreased by at least 2 log10 IU per milliliter from baseline, and normalization of the ALT level, occurred in a significantly greater percentage of patients in the bulevirtide treatment groups compared to the control group. Bulevirtide treatment led to reductions in HDV RNA and ALT levels, with a dose-dependent effect observed. Adverse events associated with bulevirtide treatment were generally mild to moderate in severity and did not lead to drug discontinuation. Further studies are ongoing to assess the long-term efficacy and safety of bulevirtide therapy. #KeyTakeaways => Significantly more patients in the bulevirtide groups showed a combined virologic and biochemical response at week 48 compared to the control group. => HDV RNA levels and liver enzyme levels were reduced in patients receiving bulevirtide. => The treatment was well-tolerated, with mild to moderate side effects reported. #JoinTheDiscussion: What do you think this breakthrough in the treatment of chronic hepatitis D means for the future of liver disease management and patient care? Share your thoughts below! #Comment, #Like, #Share, & #Follow @Dr. David Mwin #Source: https://lnkd.in/gcjDHqkE #HepatitisResearch #MedicalAdvancements #LiverDiseaseTreatment #LiverHealth

Data from an integrated pooled analysis of finerenone across three Phase III trials involving heart failure, chronic kidney disease (CKD), and Type 2 diabetes (T2D) was presented at the European Association for the Study of Diabetes e.V. (EASD) Congress 2024. This analysis was conducted with data from the FIDELIO-DKD2 and FIGARO-DKD3 trials in patients with chronic kidney disease (CKD) and type 2 diabetes and the FINEARTS-HF4 trial in patients with heart failure and mildly reduced or preserved ejection fraction. For more detailed insights, click: https://lnkd.in/gh_AGMCq