Rethinking Clinical Trials

SEPTEMBER 30, 2022

Randomized controlled trial. This trial took place in South Carolina at a time when Medicare was thinking about how to improve outcomes and if expanding home nursing services could improve health outcomes. The primary evaluation question for the trial was what is the impact of NFP on health outcomes when delivered at scale?

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XTalks

AUGUST 31, 2022

Hear more about the approval, including clinical trial data showing the treatment can take effect in as little as one week, and how it is the first antidepressant approved in over 60 years that has a new mechanism of action. Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

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Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The post First gene therapy trial for Hunter syndrome opens in Manchester, UK appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 20, 2022

The positive opinion is based on findings from the pivotal HOPE-B trial, which showed that patients treated with etranacogene dezaparvovec demonstrated durable increases in mean Factor IX activity levels which led to an adjusted annualised bleed rate (ABR) reduction of 64%. . World’s most expensive drug.

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pharmaphorum

AUGUST 21, 2022

There is still a pressing need for antidepressant therapies that can start to work more quickly than current tricyclic or selective serotonin reuptake inhibitor drugs can take weeks to elevate mood, placing severely affected patients at risk of self harm before they kick in.

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XTalks

JULY 12, 2021

“Based on our ongoing conversations with prescribing physicians, FDA and patient advocates, we submitted this label update with the goal to further clarify the patient population that was studied across the three Aduhelm clinical trials that supported approval. This week, Rep.

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XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

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XTalks

OCTOBER 12, 2022

Magnetic resonance imaging (MRI) has had a part in clinical trials for more than three decades now. However, the role of MRI in new drug clinical trials can involve diagnosis of lesions and determination of their severity, early identification of therapeutic responders to drug treatment and treatment monitoring and follow-up.

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Drug Discovery World

JANUARY 30, 2024

Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr Streamlining trial designs and regulatory pathways can help. Increased clinical data and evidence of cost-effectiveness can improve coverage.

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The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

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XTalks

OCTOBER 26, 2021

The Phase III trial results show that Givlaari offers sustainable benefit to AIP patients and confirm its favorable safety profile. The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. Clinical trial results have shown that Givlaari reduces severe AIP symptoms by 74 percent.

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pharmaphorum

MAY 17, 2021

Empaveli’s label covers treatment-naïve PNH patients as well as those who want to switch from Soliris or Ultomiris, and is based on the phase 3 PEGASUS trial in 80 patients which compared the drug to Soliris over a 16-week period. In that study, patients in the Empaveli group saw an average increase of 2.4 billion and $1.1

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pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. With a price tag of over $2.5 Zolgensma Gene Therapy: START Trial. Why is Zolgensma So Expensive?

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pharmaphorum

NOVEMBER 24, 2022

Johnson & Johnson’s Spravato therapy for treatment-resistant depression (TRD) hasn’t made the impact the company hoped for in the market, and it is looking to new clinical results to accelerate its take-up. The post J&J builds case for antidepressant Spravato with head-to-head trial appeared first on.

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pharmaphorum

JULY 25, 2022

The CHMP has backed approval of Tecvayli for adults with relapsed and refractory multiple myeloma who have received at least three prior therapies – including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody – setting up a full approval in the coming weeks.

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XTalks

JUNE 9, 2021

However, despite the welcome news, there are some within the scientific community that remain aducanumab skeptics owing to conflicting results from previous clinical trials. Under the FDA’s accelerated approval pathway , Biogen has several years to conduct a required confirmatory post-approval clinical trial.

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Rethinking Clinical Trials

MARCH 21, 2023

Goldberg Distinguished Professor and Chief of Oncology Physician-in-Chief, North Carolina Cancer Hospital Director, Cancer Outcomes Research Program University of North Carolina Slides Keywords Patient-reported outcomes, Oncology, Clinical Trials Key Points Symptoms are common in cancer care.

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XTalks

AUGUST 19, 2022

Patients with the severe form can develop microcytic anemia (characterized by small red blood cells due to reduced amounts of hemoglobin beta chains) which may necessitate the need for regular blood transfusions (administered every two to five weeks). XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research.

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pharmaphorum

MARCH 27, 2022

Johnson & Johnson is in course for approval of its multiple myeloma CAR-T therapy Carvykti in the EU the next few weeks, after getting a green light from the EMA’s human medicines committee. In the US, Abecma has a list price of around $420,00, while J&J has set its therapy a little higher at $465,000.

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XTalks

APRIL 29, 2022

Eli Lilly announced that its obesity drug tirzepatide has scored favorably in a late-stage clinical trial, with results showing that people who took the drug lost an average of 50 pounds, or 21 percent, of their body weight compared to placebo. Wegovy had a price tag of $1,297 a month when it was first launched. percent and 22.5

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pharmaphorum

AUGUST 10, 2020

While this is not cheap by anybody’s reckoning, Roche hopes that the less intimidating price tag, plus the patient-friendly oral administration method will give it a competitive edge. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

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XTalks

MARCH 22, 2023

However, this consolidation of power hasn’t been isolated to the drug companies themselves; contract research organizations (CROs) have also been coming together to form clinical trial powerhouses over the last decade. In 2015, LabCorp acquired Covance , and Quintiles and IMS Health formed a joint venture in 2016, dubbed IQVIA.

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The Pharma Data

MAY 4, 2021

In addition, we have achieved important clinical, regulatory and commercial milestones across our pipeline and portfolio while also continuing to increase our capacity to supply urgently-needed doses of BNT162b2 to the world. Clinical and Research Developments. Hospital products, which grew 10% operationally to $2.3

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Pharmaceutical Technology

NOVEMBER 30, 2022

“Bariatric surgeries show 30% weight loss in the first year and 25% weight loss in the long term,” says Papamargaritis, while that the clinical data for Mounjaro shows that the drug leads to a 16–22.5% One highly anticipated study is Mounjaro’s SURMOUNT-MMO Phase III clinical trial. compared to a 5.1%

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Pharmaceutical Technology

NOVEMBER 30, 2022

“Bariatric surgeries show 30% weight loss in the first year and 25% weight loss in the long term,” says Papamargaritis, while that the clinical data for Mounjaro shows that the drug leads to a 16–22.5% One highly anticipated study is Mounjaro’s SURMOUNT-MMO Phase III clinical trial. compared to a 5.1%

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Pharma Marketing Network

JUNE 8, 2020

I mean, you know, there has obviously been a lot of breakthrough biologics and different therapeutics like around cancers and things like that, that have high price tags because maybe they don’t have as big a market as you know, something’s wrong, you know blood pressure medication. So yeah, there is plenty to talk about there.

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