The FDA Law Blog

DECEMBER 13, 2023

Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.” Members for this advisory committee would be selected “from among authorities knowledgeable and experienced in rare disease research and development.”

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FDA 59

PQA

FEBRUARY 29, 2024

The economic burden of rare disease reached nearly $1 trillion in 2019, and the average drug approved under the Orphan Drug Act costs nearly $120,000 per year. A measure developer, researcher, educator and convener, PQA’s quality initiatives support better medication use and high-quality care.

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Specialty Pharmacies Communication Patient Care 45

IDStewardship

SEPTEMBER 2, 2022

Additionally, the Centers for Medicaid & Medicare Service (CMS) Center for Clinical Standards and Quality/Quality, Safety & Oversight Group released updates to interpretive guidance for hospital requirements, making their antimicrobial stewardship regulations live now.

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Hospitals Documentation Formulary Information Medication Guidelines 130

pharmaphorum

OCTOBER 13, 2022

However, there is a problem facing the treatment of osteoporosis, and it is the lack of research into new therapies, leading to a limited pipeline of new treatments coming through. Emerging research and biosimilars. This raises the question of why investment has been slow in the area, and are there signs of change on the horizon?

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The FDA Law Blog

JANUARY 31, 2023

Sasinowski — In early 1984, a year after President Reagan signed the Orphan Drug Act into law, the FDA was not seeing the anticipated avalanche of requests for orphan drug designation. At that time, new Federal laws on drugs were far and few between.

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FDA 59

PharmaShots

JANUARY 31, 2023

Shots: Immutable capital demands and high-spending drug development plans are the most common drivers in encouraging fully-fledged private healthcare companies to go public. The lead program of the company, THIO, is an investigational dual mechanism of action drug incorporating telomere targeting and immunogenicity in patients with NSCLC.

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Vaccines Immunization FDA 40

PharmaShots

MARCH 14, 2023

Also known as orphan diseases, the segment accounts for around 7000 types of diseases that affect 300M patients worldwide, of which most patients are young aged. The article focuses on challenges faced in drug development, government incentive programs, grants, and various therapies used to diagnose or treat rare diseases.

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FDA Immunization Compounding Vaccines 40

pharmaphorum

OCTOBER 7, 2021

The answer to the problem, of course, lays in making medical research more effective. Prior to that he was a pharmacist at the Pharmaceutical Establishment division of the Paris hospitals group AP-HP, where he was a project manager for the development of an orphan drug and a pharmaceutical contact for publicly-funded clinical studies.

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Hospitals 98

Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. Cablivi stops platelets from clumping together, which prevents small clots, and can be used along with other drugs that suppress the immune system.

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Documentation Immunization Pharmaceutical Companies Pharmaceutical Companies 52

pharmaphorum

NOVEMBER 29, 2022

Headquarters: Rockville, MD, USA (2400 Research Blvd., Locations: 3 (US HQ, Korea office, Korea R&D center). Dr Yang has created several intellectual-property protected inventions, including the hyFc® platform technology and NT-I7 drug candidate. NeoImmuneTech, Inc. CEO: Dr Se Hwan Yang, PhD. Suite 250, Rockville, MD 20850).

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Immunization Chemotherapy Compounding FDA 105

European Pharmaceutical Review

OCTOBER 31, 2022

1 Within these programmes, the ability to identify and solve challenges in the early stages with agility and flexibility is critical to successful early clinical trials that will help advance drugs from late stage through product launch. Addressing bioavailability challenges for small molecule drugs.

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Compounding Pharmaceutical Companies Pharmaceutical Companies FDA 104

European Pharmaceutical Review

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has approved bluebird bio’s Zynteglo (betibeglogene autotemcel; beti-cel), the first cell-based gene therapy for the treatment of adult and paediatric patients with beta-thalassemia who require regular red blood cell transfusions. .

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FDA Adverse Reactions 81

Pharmaceutical Technology

FEBRUARY 3, 2023

Researchers have relentlessly worked through the barriers to create lifechanging treatments for patients in need. “As As opposed to other therapies or drugs that basically help you manage a condition, gene therapy has the potential to cure a disease,” says Nicole Faust, PhD, General Manager, Cell Line Development, Cytiva. 1] BioNews.

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Communication Packaging Dosage Vaccines 97

The FDA Law Blog

OCTOBER 11, 2022

Valentine — As we begin the final quarter of 2022 and the leaves here on the east coast begin to turn and fall, it seems the clock may be running out on FDA and the Center for Drug Evaluation and Research (CDER) to meet its goal of publishing a draft guidance on confirmatory evidence this year.

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FDA Documentation Labelling 61