By Kurt R. Karst – Now that the dust from 2015 has settled, we’re able to take a look at and evaluate the year that was in orphan drug designations and approvals. Our annual review has become quite popular. We often see the numbers we provide …
Menu
Orphan Drugs
- Orphan Drug Approvals Dipped in 2015, While Designations and Designation Requests Continue Upward TrendFebruary 9th, 2016
- Orphan Drugs Under Fire: Recent Reports Reject Call for Additive Exclusivity & Urge Orphan Drug Act ReformsDecember 8th, 2015
By Kurt R. Karst – Earlier this week, Public Citizen announced the release of a report, titled “House Orphan Drug Proposal: A Windfall for Pharma, False ‘Cure’ for Patients,” that takes aim at a particular provision in the 21st Century Cures Act (H.R. 6) that the …
- HRSA Loses the Battle, and Maybe the War, Over the Orphan Drug RuleOctober 16th, 2015
By Jennifer M. Thomas & Michelle L. Butler – The U.S. District Court for the District of Columbia ruled decidedly in favor of PhRMA and against the government earlier this week in PhRMA v. HHS, No. 14-1685 (Oct. 14, 2015), potentially concluding a protracted fight between the Health …
- How Effective is a “Depomed Threat” at Resolving an Orphan Drug Clinical Superiority Dispute?September 27th, 2015
By Kurt R. Karst – The legal world is chock-full of tests, doctrines, and processes named after a particular case or court decision. It’s kind of a legal short-hand; a lexicon of legal eponyms. There’s the “Winter Standard” for preliminary injunctions in federal courts; the “Lemon …
- FDA Gives a “Nudge Nudge Wink Wink” in Denial of NORD Petition on Special Treatment of Orphan DrugsSeptember 16th, 2015
By Kurt R. Karst – In a September 10, 2015 Response to a September 2, 2011 Citizen Petition (Docket No. FDA-2011-P-0657) submitted by the National Organization for Rare Disorders (“NORD”), FDA refuses to add a statement to guidance documents concerning orphan drugs that the Agency’s official …
- FDA Releases Primer on Rare Disease Drug Development, Discusses Utility of Natural History StudiesAugust 26th, 2015
By Alexander J. Varond & James E. Valentine* – FDA recently released a draft guidance, titled “Rare Diseases: Common Issues in Drug Development.” The fourteen-page document amounts to more of a primer than a focused discussion of technical issues. It serves as a useful reminder of the …
- BIO & NORD Report Explores a World With and Without the Orphan Drug Tax CreditJuly 7th, 2015
By Kurt R. Karst – The Orphan Drug Tax Credit (“ODTC”) is not an incentive you hear about all too often, but it’s been around for quite some time – since the enactment of the Orphan Drug Act of 1983 – and is a strong incentive …
- Update to Seminal Report on Orphan Drugs Co-Authored By HP&M Attorneys Shows FDA’s Continued “Extraordinarily Reasonable Flexibility” in ApprovalMay 1st, 2015
On April 27, 2015 the Drug Information Association journal, Therapeutic Innovation and Regulatory Science, published the findings of a study conducted by HP&M Attorneys Frank J. Sasinowski and James E. Valentine*, along with co-author, Erika B. Panico, RAC, head of U.S. Regulatory Affairs for Chiesi …
- Otsuka Sues FDA Over “Corrected” ABILIFY Approval; Alleges Violation of the Most Fundamental Precept of FDA’s Statutory Drug Approval AuthorityMarch 25th, 2015
By Kurt R. Karst – Does FDA has the authority to approve a new indication for an approved drug when that new indication has not been shown to be safe and effective by adequate and well-controlled studies submitted by the sponsor of the application? And under …
- How Orphan Drugs Came to Be Called “Orphan”February 22nd, 2015
By Frank J. Sasinowski & Andrew J. Hull* – Even though we often blog on orphan drug developments (see two of our most recent posts here and here), many may be unaware of the origin of the term “orphan.” Today, we may think that the term …
- The 2014 Numbers Are In: FDA’s Orphan Drug Program Shatters RecordsFebruary 16th, 2015
By Kurt R. Karst – Earlier this month in a post concerning the Orange Book we mentioned our love of data and discussed how solid figures can reveal some interesting conclusions. After all, it’s rather difficult – and dangerous – to come to a conclusion without …
- House Energy & Commerce Committee Releases 21st Century Cures Act Discussion Draft; The Nearly 400-Page Bill Includes More Proposals Than You Can Shake a Stick At!January 28th, 2015
By Kurt R. Karst – For months we have been waiting with bated breath for the promised January 2015 release of a discussion draft of legislation to implement the 21st Century Cures Initiative, which was launched in April 2014 by now-Chairman of the House Energy and …
- FDA Issues Post-Depomed Policy Statement; Agency Doubles Down on Clinical Superiority RequirementDecember 22nd, 2014
By Kurt R. Karst & Michelle L. Butler – Ever since FDA withdrew a notice of appeal in Depomed Inc. v. U.S. Department of Health and Human Services et al., Case No. 1:12-cv-01592, to the U.S. Court of Appeals for the District of Columbia Circuit (see …
- HRSA’s 340B “Mega-Rule” Becomes Casualty of PhRMA Lawsuit Against Orphan Drug RuleNovember 18th, 2014
By Michelle L. Butler & Alan M. Kirschenbaum – On November 13, 2014, the Health Resources and Services Administration (“HRSA”) withdrew the 340B program “mega-rule” it had submitted for review to the Office of Management and Budget. In a previous blog post we reported on a …
- FDA Withdraws Appeal in the Depomed CaseNovember 11th, 2014
By Michelle L. Butler – We previously reported that FDA had filed a notice of appeal in Depomed Inc. v. U.S. Department of Health and Human Services et al., Case No. 1:12-cv-01592. In another turn of events, on November 6, 2014, FDA filed an unopposed motion to dismiss …