What is the Pediatric Drug Development Approach for Rare Diseases and Orphan Drugs?
As part of the PDUFA V reauthorization discussions, the FDA and industry are talking about…
Results for "orphan drugs"
How to Get Orphan Status for 505(b)(2) Drugs
2017 was a big year for orphan designations and approvals. Sixteen of the sixty-three (25%)…
Orphan Drug Designation: When Is It Appropriate, and How Does It Apply to Orphan Subsets?
It is an unfortunate reality that many diseases and conditions affect such small numbers of…
FDA Designations for Rare Disease Products, Part 2: Orphan Drug Designation
This is the second installment in a four-part blog series highlighting FDA designations for products…
Rare and Orphan Drug Development in the EU
A pharmaceutical company is granted orphan designation for a medicine that treats Hunter Syndrome. The…
FDA Firsts and Updates: Competitive Generics, Complex Generics, SiRNA approval, and Closing the Orphan Loophole
We decided to mention some noteworthy firsts from the FDA during August 2018 and a…
Orphan Exclusivity for ‘Same Drug’: What Has Changed Since FDARA 2017/ PDUFA VI?
Last month the US District Court for the District of Columbia ordered the FDA to…
Orphan Drug Development: Ensuring Best Time to Market
The Benefits of 505(b)(2) for Orphan Drug Development The Orphan Drug Designation Program, created by…
Opportunities in Orphan Drug Development for Investors, Pharma, and CROs
Orphan drugs, defined in the Orphan Drug Act as drugs developed to treat rare diseases…
The Potential Unveiling or Unraveling of Dormant Therapies – 15-Year Data Exclusivity for Drugs for Unmet Needs
Last week the “Dormant Therapies Act” (DTA), a companion piece to the Modernizing our Drug…
Orphan Drug Exclusivity for a Previously Approved Drug: a 505(b)(2) Conundrum
Until now, if a Sponsor intended to request orphan designation with 7 years of marketing…
The Expanding Role of RWE in Rare Studies
Thursday, September 29, 2022 | 2pm AET / 1pm KST / 12pm SGT | 60...
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2020 505(b)(2) NDA Approvals: A Year in Review, and What a Year It Was!
In 2020, CDER approved 68 NDAs that used the 505(b)(2) pathway, representing important advances in…
FDA Designations for Rare Disease Products, Part 3: Rare Pediatric Disease Designation
This is the third installment in a four-part blog series highlighting FDA designations for products…
Advancing Patient Healthcare: Clarifying the 2019 Changes in India’s Drug and Clinical Trial Rules
In 2019, only 1.2 percent of global clinical studies included India, even though the country…
FDA Designations for Rare Disease Products, Part 1
Comparing Orphan Drug, Rare Pediatric Disease, and Humanitarian Use Device Designations The FDA offers sponsors…
How Are Biopharma Companies Evaluating Partnerships in the Rare Disease Space?
Panel Summary from the World Orphan Drug Congress 2020 Last month, executives from leading international…
What is 505(b)(2)?
The 505(b)(2) new drug application (NDA) is one of three U.S. Food and Drug Administration...
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Product Ideation: Who Wants to Develop a Successful Product?
As Sponsors become more aware of the benefits and risks of developing products in the…
Deuterization: Is it Enough to Get 5- or 7-Year Exclusivity for a 505(b)(2) Product?
As the 505(b)(2) experts, Premier Consulting has received several enquiries about developing deuterated drugs as…
Extending Exclusivity: How Long Will It Really Last?
Last week at the Generic Pharmaceutical Association (GPhA) Annual Meeting, the 21st Century Cures Act,…
505(b)(2) Patent & Marketing Exclusivity
IP attorney Stephen Albainy-Jenai and I just concluded a webinar hosted by DIA entitled 505(b)(2) Patent…
Premier Research and InSilicoTrials Partner to Leverage In Silico Modeling and Simulation and Optimize Regulatory Pathways for Rare Disease Therapies
MORRISVILLE, N.C. and MILAN and TRIESTE, Italy, March 13, 2023 /PRNewswire/ — Premier Research, whose mission is to help the most innovative…
PREA and Fixed-Dose Combinations: When Things Get Complicated
The Pediatric Research Equity Act (PREA) was signed into law in 2003 to increase pediatric…
New Drug Development: Succeeding With the FDA – Nov. 24, 2022
Date: November 24, 8am EST | 6:30pm IST Speaker: Kristi Miller, PhD, Vice President, Regulatory...
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New Drug Development: Succeeding With the FDA
New Drug Development: Succeeding With the FDA March 17, 2022 | 45 min Speaker: Kristi...
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FDA Designations for Rare Disease Products, Part 4: Humanitarian Use Device
This is the final installment in a four-part blog series highlighting FDA designations for products…
The FDA Is Ending Its Rare Pediatric Disease Priority Review Vouchers – and Time Is Running Out to Get One
Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA…
Understanding New FDA Guidance For Pediatric Oncology Studies: A Sponsor’s Guide to the FDA Research to Accelerate Cures and Equity for Children Act
Understanding New FDA Guidance For Pediatric Oncology Studies A Sponsor’s Guide to the FDA Research...
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2019 505(b)(2) NDA Approvals in Review
In 2019, CDER approved 64 NDAs that used the 505(b)(2) pathway, representing important advances in…
A Vitamin Approved as a Drug(!): Forget what you think you know to be true
Dogma says that a compound used for a nutrition, such as a vitamin, cannot be…
Totality of Evidence and 505(b)(2): Are Two Phase III Studies Too Many for a Well-Known Already-Approved Drug?
As the 505(b)(2) expert, Premier Consulting is exposed to many projects in which sponsors have…
The Value of a Strategic Assessment: Aligning for Success from the Start
The first step for every wise drug developer beginning a drug development program is to…
505(b)(2) Approval Times: The Real Scoop
The Approval Time for 505(b)(2) and 505(b)(1) NME Products Is Similar A recent article by…
Real-World Evidence: Can it Really Be Used For Drug Approvals?
With the signing of the 21st Century Cures Act, the US Congress tasked the FDA…
Unforced Errors: FDA Refusal to File or Receive Letters
Few things can be more damaging to a pharmaceutical company than the refusal by the…
Effects on Combination Products: The Biologics Price Competition and Innovation Act of 2009
On March 23, 2010, the U.S. FDA enacted the Biologics Price Competition and Innovation Act…
Effects on Exclusivity: The Biologics Price Competition and Innovation Act of 2009
On March 23, 2010, the U.S. FDA enacted the Biologics Price Competition and Innovation Act…
Improving Drug Development ROI in 2017
Time to Pick the Low-Hanging Fruit: Improving Drug Development ROI in 2017 With forecasts of…
Protein Product 505(b)(2)s Face a Looming Application “Dead Zone”
The Biologics Price Competition and Innovation Act of 2009 (BPCIA) was enacted on March 23,…
The Prodrug Benefit of Utilizing the 505(b)(2) Pathway
Designing a new drug from scratch is costly and time-consuming. One attractive option to differentiate…
Tropical or Rare Pediatric Disease Priority Review Vouchers: Update and Use of the 505(b)(2) Pathway
While the United States market for drugs and biologics targeting tropical or rare pediatric diseases…
Back to Basics: 505(b)(2) FAQs Part 3: Regulatory Strategies
As the 505(b)(2) expert, Premier Consulting is frequently asked questions about how to get a…
Pediatric Applicability or Not–This Revised Guidance Is for You
Since 1994, the statutory and regulatory requirements for drug product labeling for pediatric populations have…
21st Century Cures Act – Provisions Impacting Pharmaceutical Regulation
On July 2, 2015 the House passed the 21st Century Cures Act . The focus…
ANDA but No NDA – What to Rely on?
Premier Consulting participates in multiple pre-IND (PIND) meetings each month and one thing we notice…
505(b)(2): Freedom from Generic Competition and Exclusivity Issues
Well, freedom from generic competition for a while at least. Generally, most companies’ business plans…