Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected
On the path to U.S. commercialization, every pharmaceutical product makes a stop at the Food & Drug Administration (FDA). Whether that stop becomes a waystation or the end of the line may depend on the degree to which the presenters share the FDA’s understanding of
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of the advancements included in PDUFA
This is the final installment in a four-part blog series highlighting FDA designations for products with rare disease indications: orphan drug designation (ODD), rare pediatric disease designation (RPDD), and humanitarian use device (HUD) designation. In this post, we examine the humanitarian use designation. The HUD designation
Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. These therapies frequently target
In 2020, CDER approved 68 NDAs that used the 505(b)(2) pathway, representing important advances in patient care across a wide range of therapeutic areas. 505(b)(2) Drug Improvement Approvals by Year In spite of an unusual year due to the COVID-19 pandemic, NDA approvals via the
Rare Disease Clinical Trials Most Often Terminated Due to Regulatory and Recruitment Issues The difficulty associated with successfully completing rare disease clinical trials is well-known. Most people believe that recruitment is the primary reason because, by definition, the number of potential trial participants is extremely
This is the third installment in a four-part blog series highlighting FDA designations for products with rare disease indications: orphan drug designation (ODD), rare pediatric disease designation (RPDD), and humanitarian use device (HUD) designation. Part 2 examined orphan drugs, and in Part 3, we take a detailed
This is the second installment in a four-part blog series highlighting FDA designations for products with rare disease indications: orphan drug designation (ODD), rare pediatric disease designation (RPDD), and humanitarian use device (HUD) designation. Part 1 compared these designations, and in Part 2, we provide a
Comparing Orphan Drug, Rare Pediatric Disease, and Humanitarian Use Device Designations The FDA offers sponsors a variety of special programs to incentivize development of therapies to treat unmet patient needs, including fast track, breakthrough therapy, and qualified infectious disease product designations. This blog post initiates a four-part series
As a sponsor designs clinical studies, the respective comparison control groups become a critical factor to consider. Often, to gain clinical trial design insights, a sponsor reviews the physician package inserts from approved New Drug Applications (NDAs) and Biologics License Applications (BLAs) with similar indications
Throughout the life-cycle of a drug development program, sponsors are confronted with a range of key questions. They must decide on the correct… Regulatory pathway, FDA center, Key Opinion Leaders (KOLs), Target Product Profile (TPP) inclusions, Data to rely on, Studies to conduct, Indication and
Panel Summary from the World Orphan Drug Congress 2020 Last month, executives from leading international biopharma companies came together at the World Orphan Drug Congress 2020 to discuss how they are evaluating new opportunities in the rare disease space. The panel discussion was moderated by
Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.1 The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area
A pharmaceutical company is granted orphan designation for a medicine that treats Hunter Syndrome. The company goes on to receive marketing approval for the medicine. The approved medicine is administered through intravenous infusion. This route of administration, while effective for some, does not address the
It has recently been reported that drugmakers have argued against broad orphan exclusivity for Eagle Pharmaceuticals, Inc.’s Bendeka® product. This was in response to the FDA’s invitation to applicants of certain products containing bendamustine to share how broad of a scope they believe Eagle’s seven-year
2017 was a big year for orphan designations and approvals. Sixteen of the sixty-three (25%) products approved via the 505(b)(2) pathway in 2017 received orphan designation. Importantly, more than half of the approved 505(b)(2) products with orphan designation received seven years of orphan exclusivity. That’s
The Benefits of 505(b)(2) for Orphan Drug Development The Orphan Drug Designation Program, created by the Orphan Drug Act of 1983, provides significant financial incentives for the development of drugs for rare diseases. The potential incentives include tax credits for clinical trial costs, waiver of
A 505(b)(2) may rely on the FDA’s previous findings of safety and efficacy of an approved drug product. It is possible to rely on more than one approved drug product. It is also possible that a 505(b)(2) applicant does not have to rely on any approved
Orphan drugs, defined in the Orphan Drug Act as drugs developed to treat rare diseases that affect fewer than 200,000 people in the U.S., have begun to make their mark for patients and drug companies. As the number of orphan drugs has increased over the
Last week at the Generic Pharmaceutical Association (GPhA) Annual Meeting, the 21st Century Cures Act, a proposed bill with bipartisan support, was a topic of discussion. Specifically, subtitle L—Dormant Therapies, which would offer 15 years of exclusivity for drugs and biologics approved as dormant therapies.
Until now, if a Sponsor intended to request orphan designation with 7 years of marketing exclusivity for a drug that has already been granted orphan designation, FDA has followed the Code of Federal Regulations (CFR), including the condition described in 21CFR §316.34(c): “If a drug
Yesterday, Depomed filed suit against the FDA requesting the Court to order FDA to grant their product Gralise (gabapentin) seven years of exclusivity since it was granted Orphan status; upon approval, Gralise was granted three years of exclusivity. Depomed licensed the product to Solvay which became
As part of the PDUFA V reauthorization discussions, the FDA and industry are talking about better approaches to rare disease drug development and orphan drugs. Public interests and Congress have mandated that the FDA develop new guidances on the required studies needed for NDA approval.
IP attorney Stephen Albainy-Jenai and I just concluded a webinar hosted by DIA entitled 505(b)(2) Patent & Exclusivity. 23 different companies attended, showing the increasing interest in 505(b)(2) issues. Earlier this year, DIA hosted my overview of the 505(b)(2) drug development process where the attendees had many questions asking
