Drug Discovery World

DECEMBER 20, 2022

Thank you to all for the role you have played in helping us reach this regulatory milestone in Europe.” . Concerns about the high price tag have been raised in an article in Nature, particularly whether it will be affordable for low to middle-income countries. . World’s most expensive drug.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Pharmaceutical Technology

NOVEMBER 9, 2022

million price tag. While experts interviewed by Pharmaceutical Technology found the cost-effectiveness estimates valid, the durability of gene therapies in haemophilia remains unclear, particularly in the case of Roctavian, which has faced similar queries from regulatory authorities. appeared first on Pharmaceutical Technology.

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Gene Therapy Gene FDA Approval Medicine 278

Rethinking Clinical Trials

FEBRUARY 16, 2023

When not getting full regulatory consent, the relevant questions are: What should they know? Tags #pctGR, @Collaboratory1 The post Grand Rounds Ethics and Regulatory Series February 10, 2023: Informing and Consenting: What Are the Goals? (P. Participants always know some things and agree to some extent.

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Rethinking Clinical Trials

OCTOBER 4, 2022

CTTI has developed 35 tools to help study teams develop meaningful measures, map individual medical product development, navigate regulatory requirements, and help implement other CTTI recommendations. CTTI wants to increase meaningful novel digital endpoints that are clinically relevant and fit-for-purpose. Discussion Themes.

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Rethinking Clinical Trials

JUNE 29, 2022

Real-world data (RWD) is a term with specific regulatory implications referring to health care data routinely collected from a variety of sources. Big Data, a term first used in the 1990s, leverages modern technology to increase the quantity, forms, speed, and capability to manipulate large-scale data. and the FDA Draft Guidance for RWD/RWE.

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pharmaphorum

SEPTEMBER 15, 2020

“Digitally native brands are typically agile businesses from the ground up, and agility and flexibility are part of their core makeup,” says Joe Holmes, vice president, marketing services, US at Tag. Jeremy Richter, Tag. Joe began his life at Tag in the UK, bringing efficiencies to his clients, working across all conceivable channels.

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Rethinking Clinical Trials

FEBRUARY 7, 2024

Potential strategies: Increase direct-to-consumer education, emphasizing the scientific rigor behind, and the reasons for, FDA’s regulatory processes; collect, analyze, and use information about consumer use of FDA’s website; increase interaction with the media. Observation 2: Information vacuums breed misunderstanding.

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Rethinking Clinical Trials

NOVEMBER 16, 2022

The post Grand Rounds Ethics and Regulatory Series November 11, 2022: Data Sharing and Pragmatic Clinical Trials: Law & Ethics Amidst a Changing Policy Landscape (Stephanie Morain, PhD, MPH; Kayte Spector-Bagdady, JD, MBioethics) appeared first on Rethinking Clinical Trials. pctGR, @Collaboratory1.

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Camargo

AUGUST 22, 2021

The first two were published in December of 2014: Providing Regulatory Submissions in Electronic Format — Submissions Under Section 745A(a) of the Federal Food, Drug, and Cosmetic Act mandates that NDAs, ANDAs, and INDs be submitted in electronic format. The correct STF file-tags are used for datasets and corresponding data definition files.

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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. Professor Bigger said: “This is a next generation stem cell gene therapy approach, which allows transit of the IDS enzyme into the brain.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

DECEMBER 17, 2020

Her videos cover a host of topics from the science behind the immune system, side effects caused by the shots and vaccine hesitancy and the clinical and regulatory path to approval. Additionally, we will soon introduce a new vaccine tag to detect and tag all videos with words and hashtags related to the COVID-19 vaccine,” says Morgan. “We

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Drug Discovery World

JANUARY 30, 2024

Development and Regulatory: Clinical trials: Long and complex clinical trials with stringent regulatory requirements are necessary for safety and efficacy evaluation, but they also contribute significantly to the overall cost. Streamlining trial designs and regulatory pathways can help.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

Intouch Solutions

APRIL 11, 2024

Instead, it will be a transformational process that affects various functions of the organization, triggering considerations related to data privacy and security, regulatory compliance, and change management. Facilitating cost containment and expediting the content approval process in accordance with regulatory compliance.

Compliance 52

Compliance Marketing Drugs Pharma Companies 52

XTalks

FEBRUARY 27, 2024

Despite high price tags of a couple of million dollars, gene therapy makers say they are working to ensure patients are able to access the treatments through patient assistance programs developed in conjunction with the support of governments. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

JANUARY 5, 2022

Other drugmakers have been looking at Met as a target in cancer, including Novartis, Merck KgAA and Hutchmed, which have all bagged regulatory approvals for oral therapies that target NSCLC harbouring a specific mutation in Met known as exon 14 skipping which affects about 3% to 4% of NSCLC patients.

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Pharmaceutical Technology

MAY 2, 2023

Chimeric antigen receptor T-cell (CAR-T) therapies are the only genetically modified cell therapies to have received regulatory approval, and they are currently utilised in relapsed/refractory settings. Cost-related unmet needs also scored highly.

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Rethinking Clinical Trials

MARCH 21, 2023

Regulatory authorities have become interested in PROs and it is thought of as a fundamental part of drug development. FDA and other regulatory authorities want to think about symptoms separately from adverse events. Now almost any physician has heard of patient-reported outcomes. You will want a dedicated tool for adverse events.

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Clinical Trials Clinical Trials Trials Research 130

Rethinking Clinical Trials

FEBRUARY 28, 2024

But there are concerns to developing DCTs including lack of standardization and validation, regulatory and ethical uncertainties, engagement vs. coercion, data security and privacy issues, technological literacy and access, resistance to change and adoption, and lack of “safe” sharing. from 2021 to 2026.

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The Pharma Data

AUGUST 18, 2020

The $475 million price tag on the deal will be made by BMS in near-term upfront payments, and Dragonfly will have access to further milestone payments contingent on regulatory and commercial successes of drugs under the partnership, as well as up to 24% royalties on net sales worldwide. .

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Rethinking Clinical Trials

AUGUST 23, 2022

There is a national priority to increase diversity in clinical trials, from regulatory, funding agencies, industry, and others, so that the participant population reflects the U.S. Assistant Professor of Medicine. Duke University School of Medicine. Diversity, Inclusion. Key Points. Learn more. pctGR, @Collaboratory1.

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Clinical Trials Clinical Trials Trials Medicine 130

Rethinking Clinical Trials

JULY 6, 2023

During the COVID-19 pandemic, several regulatory authorities around the world introduced guidance on DCTs. There has been a growing sense of hesitancy about whether regulators will remain equally receptive to these decentralized methods when the pandemic recedes.

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pharmaphorum

NOVEMBER 19, 2021

Evrysdi was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in May, a couple of months after the drug also got a green light in the EU. million price tag make it is the most expensive treatment ever approved for NHS funding.

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Drugs Gene Therapy Genetic Disease Gene 95

Rethinking Clinical Trials

DECEMBER 14, 2022

The post Grand Rounds Ethics and Regulatory Series December 9, 2022: The Stepped Wedge Cluster Randomized Trial: Friend or Foe? It’s still important to look at how effective it is and is the juice worth the squeeze in this context? You should use the best statistical methods to answer the question you are trying to answer.

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pharmaphorum

AUGUST 25, 2022

Johnson & Johnson has its first worldwide regulatory approval – in the EU – for Tecvayli, one of two much-anticipated bispecific antibodies the drugmaker has developed for patients with multiple myeloma. Tecvayli previously scored a priority medicines (PRiME) designation from the EMA and a breakthrough-therapy tag from the FDA.

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Drug Discovery World

NOVEMBER 7, 2022

CELMoDs are designed to attack cancer by binding to a regulatory protein called cereblon, which then triggers the degradation of key cancer-driving proteins. . Cereblon, the researchers determined, has a default ‘open’ conformation, but must be switched to a particular ‘closed’ conformation for the ubiquitin-tagging of target proteins. .

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Scientist Drugs Protein Research 52

XTalks

AUGUST 19, 2022

Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more. Zynteglo’s hefty price tag of $2.8

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Rethinking Clinical Trials

SEPTEMBER 16, 2022

The post Grand Rounds Ethics and Regulatory Series September 9, 2022: Building An Academic Learning Health System: Why Is It So Hard? It is incumbent on all of us, and it should be a central part of the goal for all of us to deliver care to patients and families. We should all think about how our organizations can move in this direction.

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The Pharma Data

MAY 5, 2021

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. . AbbVie’s Humira regains top pharma TV spender spot, while sibling brands Rinvoq and Skyrizi tag along ( Fierce ). Government & Regulatory. Remote Regulatory Assessments ( FDAatty ).

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Vaccination Vaccine Gene Therapy Contract Packaging 52

pharmaphorum

AUGUST 17, 2020

The financial world, like the pharmaceutical industry, must operate within a tightly-defined regulatory environment, and in doing so has proved adept at connecting with its customers. Joanna Carlish, managing director, financial services, Tag Americas. Joanna began her career at Tag as an Account Manager with Banco Santander in London.

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Regulation Marketing Sales Branding 84

World of DTC Marketing

JUNE 23, 2021

The story of how a drug that’s never even been proven to work is now poised to rake in billions of dollars for its manufacturer, impose needless suffering on millions of families, and destabilize Medicare and Medicaid in the process, goes well beyond the failures of what should be the toughest regulatory body of the U.S. government.

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Drugs FDA Approval Doctors Doctor 218

pharmaphorum

AUGUST 21, 2022

There’s no word yet on its pricing, but for comparison Spravato was launched with an annual price tag of around $32,400, which the Institute for Clinical and Economic Review (ICER) concluded was around 25% to 52% too high to be cost effective.

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Rethinking Clinical Trials

JANUARY 19, 2023

Tags #pctGR, @Collaboratory1 The post Grand Rounds Ethics and Regulatory Series January 13, 2023: Ethical Considerations When Vulnerable Populations are Subjects in Pragmatic Trials (Emily A. That’s difficult. Take the individual’s consent as word; however, if they are dependent on the surrogate for transportation, etc.,

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Trials Clinical Trials Clinical Trials Research 130

Rethinking Clinical Trials

JUNE 14, 2023

Tags #pctGR, @Collaboratory1 The post Grand Rounds June 9, 2023: Emulating Randomized Clinical Trials with Non-randomized Real-world Evidence Studies: Results From The RCT DUPLICATE Initiative (Shirley V. The goal with this aim is to determine if the same conclusion can be drawn from a database study and a trial.

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Rethinking Clinical Trials

AUGUST 28, 2023

In terms of administrative and regulatory lessons learned, the team reflected that the integration of PT/DC at the forefront of the patient experience is often different from many typical processes. There have also been hiring challenges related to the current job market, as well as regulatory challenges related to consent waivers and IRBs.

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Rethinking Clinical Trials

OCTOBER 19, 2022

The post Grand Rounds Ethics and Regulatory Series October 14, 2022: Responding (or Not) to Signals of Potential Clinical Significance in Pragmatic Clinical Trials (Joseph Ali, JD; Tanya Matthews, PhD; Leslie J. The timeliness of the response is also important; if the trial is monitoring something they need to be able to respond timely.

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Clinical Trials Clinical Trials Trials Medicine 130

The Pharma Data

NOVEMBER 15, 2020

Peng Liang , Founder and Chief Scientific Officer of Clover and inventor of the Trimer-Tag© technology, says, “A successful vaccine that can truly modulate the course of the COVID-19 pandemic must have four essential characteristics: safety, efficacy, scalability of manufacturing, and speed. .”

Vaccination 52

Vaccination Vaccine Manufacturing Life Science 52

pharmaphorum

APRIL 20, 2021

NICE’s final appraisal document for Kesimpta will be followed by technology appraisal guidance (TAG) that will pave the way for NHS prescribing in England and Wales. The Scottish Medicines Consortium is expected to publish its final advice on the drug later this year.

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