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tag treatment 
Pharmaceutical Technology
NOVEMBER 20, 2023
NeoImmuneTech is developing NT-17, an IL-7 treatment, to promote rapid T-cell recovery in patients with acute radiation syndrome.
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pharmaphorum
MARCH 20, 2024
Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment
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Bio Pharma Dive
FEBRUARY 25, 2021
Amondys 45 is now cleared for the estimated 8% of Duchenne patients eligible for treatment. Like other Sarepta drugs, it comes with a high price tag and its clinical benefits haven't been confirmed.
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Pharmaceutical Technology
SEPTEMBER 30, 2022
Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. bluebird has also proposed an outcomes-based agreement to make covering this treatment more attractive for payers.
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Pharmaceutical Commerce
MARCH 6, 2024
Navigating the complex landscape of healthcare coverage can be an intimidating task, especially for patients with rare diseases for which treatments often come with a high price tag.
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Fierce Pharma
NOVEMBER 7, 2023
Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Vertex and CRISPR Therapeutics are up first with a Dec. |
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BioSpace
NOVEMBER 7, 2023
Cell and gene therapy represents the most promising breakthrough in cancer treatment for decades, but it comes with a hefty price tag. Here’s how to drive costs down.
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Pharmaceutical Technology
MAY 19, 2023
Currently, most patients face the prospect of life-long treatment with MS medications, as the main objectives of available disease-modifying therapies (DMTs) are to reduce the frequency of relapses and to delay the accumulation of disability. DMTs for MS have a high price tag, particularly in the US.
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World of DTC Marketing
JUNE 1, 2021
Still, Alzheimer’s is a nasty disease, and people are desperate for treatment even if it only provided a slim chance of partial recovery. If the drug is approved you can bet that it’s going to carry a huge price tag. Will they follow the science or will they put a price tag on hope?
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STAT News
JANUARY 6, 2023
Food and Drug Administration has granted accelerated approval to the widely anticipated Alzheimer’s drug from Eisai and Biogen, a key question is the extent to which payers — private and public — will cover the treatment. Such decisions are based on myriad factors, starting with the average $26,500 price tag.
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Medical Xpress
NOVEMBER 23, 2022
People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.
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Pharmaceutical Technology
NOVEMBER 9, 2022
Despite their potential multi-million-dollar sticker price, if haemophilia treatments by BioMarin and CSL Behring prove durable, they could help patients save money compared to current treatments, experts note. A fair price for CSL Behring’s haemophilia B treatment etranacogene dezaparvovec would be between $2.93–2.96
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STAT News
OCTOBER 27, 2022
Amid sporadic shortages of a drug that is essential in preparing patients for lifesaving, cancer-fighting treatments, one manufacturer has returned to the market — but is selling its medicine for 10 to 20 times the prices offered by the only other companies with available supplies. Continue to STAT+ to read the full story…
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STAT News
JULY 14, 2022
These treatments constitute a highly lucrative market because they are used to help the body fight cancer, infection, and a wide array of other diseases — and often carry considerable price tags. Continue to STAT+ to read the full story…
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Pharmaceutical Technology
JANUARY 17, 2023
Furthermore, bimagrumab is a long-acting therapy, thus significantly reducing the number of treatment days in comparison to many currently marketed and late-stage pipeline therapies. However, its intravenous route of administration means that the treatment can only be administered in a hospital.
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Rethinking Clinical Trials
DECEMBER 6, 2023
Each of the clusters receive the control and treatment, and each cluster is sequenced to the intervention at different times. How will the treatment effect the outcome? But this assumption may not be true in all studies or for all types of treatment. Classic stepped-wedge analyses assume an instantaneous treatment effect.
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Drug Discovery World
OCTOBER 26, 2022
Biomarkers are valuable tools in diagnosing disease or predicting treatment outcomes, but they are challenging to find. . The new method involves adding chemical tags to sugar molecules which are added to cells. When the cells make these proteins, they remain marked with the chemical tag, meaning that researchers can identify them.
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Rethinking Clinical Trials
MARCH 15, 2023
An estimand is a precise description of the treatment effect that a researcher aims to estimate from the trial. Informative cluster size refers to situations in which outcomes or treatment effects differ in large clusters versus smaller clusters. and “How much would standard errors really change?”
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Pharmaceutical Technology
FEBRUARY 10, 2023
On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.
263 Rethinking Clinical Trials
JANUARY 23, 2024
RCT design will vary depending on which CAD manifestation(s) are reflected in the information provided by the imaging test being studied, which affects the treatment target(s) being evaluated in a therapeutic trial. Tags #pctGR, @Collaboratory1 The post Grand Rounds January 19, 2024: Why Are Imaging RCTs Different?
152 Rethinking Clinical Trials
JANUARY 17, 2024
The first monoclonal antibody (mABs) treatment for treatment of COVID-19 was approved in November 2020. Patients from disadvantaged communities had increased odds of receiving treatment. UPMC built upon the success of the Remdesivir lottery and in December 2020, UPMC began infusing monoclonal antibodies across 45 UPMC locations.
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Rethinking Clinical Trials
APRIL 18, 2023
RECOVER has used cohort data to identify major symptom clusters and develop 5 platform protocols that will investigator priority symptom clusters and their causes; test known and novel interventions across domains; and evaluate treatments to improve Long COVID symptoms.
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STAT News
OCTOBER 6, 2022
… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even
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pharmaphorum
DECEMBER 24, 2021
The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of early-stage Alzheimer’s disease. The $56,000-a-year price tag for the drug, which has now been cut in half in the US, also hasn’t helped the drugmakers make a case for Aduhelm with US payers.
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Rethinking Clinical Trials
AUGUST 4, 2022
TOGETHER is a randomized adaptive platform trial that was initiated in June 2020 to investigate the efficacy of repurposed treatments for COVID-19 disease among high-risk adult outpatients. It was a big treatment effect observed in a predominantly vaccinated population. TOGETHER, COVID-19, Peginterferon Lambda. Key Points.
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XTalks
AUGUST 31, 2022
In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Bluebird’s $2.8M
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Pharmaceutical Technology
APRIL 13, 2023
Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. As per ICER, if a treatment improves quality of life or extends a patient’s life, then this can be represented in additional QALYs provided by the therapy.
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Rethinking Clinical Trials
FEBRUARY 28, 2024
Did participants receive the assigned treatment and did they stay on the treatment? Baseline state, treatment, outcome, and safety data are critical to understanding treatment benefits and risks. Was there complete ascertainment of primary and secondary efficacy data? Were there any major GCP-related issues?
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Pharmaceutical Technology
DECEMBER 5, 2022
Cannabinoid receptors are a popular therapeutic target for cannabinoid-based drugs in the treatment of pain, neurological disorders and inflammation, according to GlobalData’s Pharma Intelligence Centre Drugs database. This is closely followed by CB2 receptors in second place.
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Rethinking Clinical Trials
OCTOBER 26, 2023
There is a lack of point-of-care diagnostics; it can take up to 3 steps to treatment initiation. There is high cost of treatment and insurance prior-authorization requirements. The treatment is not a routine part of primary care, and there is an underserved and hard-to-reach population.
XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Current treatments for sickle cell disease include blood transfusions but patients often have episodes of intense pain and an increased risk of stroke.
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Pharmaceutical Technology
MAY 1, 2023
CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression. Shunxi will also hold an option to negotiate rights to other CAR-T products, which include the licenced IP.
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pharmaphorum
OCTOBER 3, 2022
Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.
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pharmaphorum
JANUARY 9, 2023
Since 2020, the global SMA treatment landscape has progressed, with three treatments now approved, according to Novartis. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 The post Free access to Zolgensma curbed, says Novartis appeared first on.
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pharmaphorum
JANUARY 5, 2022
The FDA thinks an antibody-drug conjugate (ADC) developed by AbbVie could set new standards in treatment for certain patients with lung cancer, awarding the drug breakthrough status. The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on. Preliminary results from that study showed a 53.8%
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World of DTC Marketing
SEPTEMBER 7, 2022
In pharma, growth depends on new products with hefty price tags when over 80% of voters want lower costs for their prescription drugs. DTC commercials will be replaced by unbranded ads that look to engage patients and caregivers by forming a relationship based on making them more intelligent about their treatments.
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XTalks
AUGUST 19, 2022
Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8
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Drug Discovery World
DECEMBER 20, 2022
The CHMP has recommended conditional marketing authorisation of CSL’s (etranacogene dezaparvovec), a one-time gene therapy for the treatment of appropriate adults with the disease. . A one-time treatment with etranacogene dezaparvovec costs US$3.5 The therapy was also recently approved by the FDA for patients in the US. .
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Rethinking Clinical Trials
MARCH 1, 2023
For the standard of care arm, treatment is determined by the treating investigator and participant, with the recommendation that the choice of drug is based on NCCN guidelines and dosing administration based on the participant’s previous therapy and disease. – What was the rationale for the choice of study treatments (i.e.,
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pharmaphorum
NOVEMBER 19, 2021
Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.
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Drug Discovery World
MARCH 2, 2023
New understanding of epigenetics It has been known for more than 20 years that the enzymes placing H3K4me3, a chemical tag added to DNA, are crucial for normal cell development, as well as being linked to leukaemia, breast, bowel and pancreatic cancers.
Rethinking Clinical Trials
FEBRUARY 2, 2023
In clinical care, patients are receiving treatments that are ineffective (or harmful). Without RCTs we cannot know which treatments are helpful and which are not. Emergency research has largely focused on a small number of conditions and neglected many common treatments, such as intubation.
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Drug Discovery World
OCTOBER 31, 2022
The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.
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Rethinking Clinical Trials
NOVEMBER 6, 2023
Cook, PhD Senior Biostatistics Investigator, Kaiser Permanente Washington Health Research Institute Slides Keywords Individually randomized group treatments; Intervention; Randomization; Clinical trials Key Points In individually randomized trials (IRTs), individuals are randomized to either control or intervention arms.
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