Enacted in 1992, the Prescription Drug User Fee Act (PDUFA) was created to authorize the Food and Drug Administration (FDA) to collect fees from companies that produce certain human drug and biological products.
These fees serve as a secondary source of funding for the FDA and serve to expedite the review process. The FDA establishes specific performance goals regarding the timeframe for review of products.1 Although these timeframes are numerous for new drug products the review period can range from 6 to 10 months depending on the application’s priority of review.
In this publication, the RJ Health Systems Analytics team will present an overview of what to expect from the FDA in 2018 based on established PDUFA dates for identified specialty drugs.
As of the publication of this article, we identified 63 unique specialty products with established PDUFA dates in 2018.
Twenty-five percent of the volume of drugs with PDUFA dates are expected to be reviewed by the end of April with an additional 17% in May. Similar to recent years, the vast majority of products submitted are under review for an oncological diagnosis (53%) (Figure 1).
Sixty six percent of the drugs have a designation of a priority review, therapies that are considered to be a significant advance in the treatment of a specific condition, while the remainder are under standard review.2 Products that are designated with a priority PDUFA review have an established goal of review within 6 months of the filing date, while standard review has a goal of 10 months. Oncology, infectious disease, and metabolic disorders reflect the top therapeutic classes that have greater than one product under review with the greatest proportion of priority designations (Figure 2).
Evaluation of the molecule type reveals the ongoing trend towards greater submissions for biologically based drugs, with monoclonal antibodies representing the greatest proportion of products. Small molecules continue to be an important component of new drugs primarily within the oncology and infectious disease categories (Figure 3).
In regard to the potential benefit of coverage, we reviewed the potential route of administration and found that 53 percent of the products would most likely be covered under the medical benefit, while the remaining 47 percent would receive coverage under the pharmacy benefit (Figure 4). This percentage is consistent with our previous findings that on average 57 percent of FDA actions on specialty drugs are associated with drugs that were highly likely to be covered under the medical benefit (trends-fda-approval-specialty-drugs-1990-q3-2017 ).
Of the applications submitted for review, 65 percent are for a new drug application (NDA or BLA) and 35 percent were for currently approved drugs under review for supplemental indications (sNDA or sBLA) (Figure 5).
Table 1 lists the specialty products with PDUFA dates in 2018 broken out by new and supplemental drug applications. Of interest is that seven products having an established PDUFA in 2018 were evaluated by the end of 2017. Of these seven products, six had a priority review designation and one had a standard review, but the application was for a supplemental new drug application (sNDA) in a previously approved drug (Sutent (Pfizer Inc)).
Table 1 – Specialty drugs with an established PDUFA date sorted by date Pharma Intelligence Center)3
|Drug Name||Company Name||Approval Date||Therapy Area||PDUFA Date|
|New Drug Applications|
|Lutathera (lutetium Lu 177 dotatate)||Advanced Accelerator Applications SA||26-Jan-2018||Oncology||Jan-18|
|Luxturna (voretigene neparvovec-rzyl)||Spark Therapeutics Inc||19-Dec-2017||Genetic Disorders||Jan-18|
|Verzenio (abemaciclib)||Lilly USA LLC||28-Sep-2017||Oncology||Jan-18|
|Biktarvy ((bictegravir sodium + emtricitabine + tenofovir alafenamide))||Gilead Sciences Inc||07-Feb-2018||Infectious Disease||Feb-18|
|Calquence (acalabrutinib)||AstraZeneca Pharmaceuticals LP||31-Oct-2017||Oncology||Feb-18|
|Hemlibra (emicizumab)||Genentech USA Inc||16-Nov-2017||Hematological Disorders||Feb-18|
|Symdeko ((ivacaftor + tezacaftor))||Vertex Pharmaceuticals Inc||12-Feb-2018||Respiratory||Feb-18|
|Blincyto (blinatumomab)||Amgen Inc||Oncology||Mar-18|
|tildrakizumab||Sun Pharma Advanced Research Company Ltd||Immunology||Mar-18|
|YONSA (abiraterone acetate)||Churchill Pharmaceutical LLC||Oncology||Mar-18|
|Azedra (iobenguane I 131)||Progenics Pharmaceuticals Inc||Oncology||Apr-18|
|burosumab||Ultragenyx Pharmaceutical Inc||Nutritional Disorders||Apr-18|
|Erleada (apalutamide [INN])||Johnson & Johnson||14-Feb-2018||Oncology||Apr-18|
|ibalizumab||TaiMed Biologics Inc||Infectious Disease||Apr-18|
|Lynparza (olaparib)||AstraZeneca Pharmaceuticals LP||12-Jan-2018||Oncology||Apr-18|
|rituximab biosimilar||Celltrion Inc||Oncology||Apr-18|
|Ryplazim (plasminogen (human))||ProMetic Life Sciences Inc||Genetic Disorders||Apr-18|
|Tavalisse (fostamatinib disodium)||Rigel Pharmaceuticals Inc||Hematological Disorders||Apr-18|
|trastuzumab biosimilar||Celltrion Inc||Oncology||Apr-18|
|trastuzumab biosimilar||Pfizer Inc||Oncology||Apr-18|
|Verzenio (abemaciclib)||Eli Lilly and Co||Oncology||Apr-18|
|AndexXa (andexanet alfa)||Portola Pharmaceuticals Inc||Hematological Disorders||May-18|
|avatrombopag||Dova Pharmaceuticals Inc||Hematological Disorders||May-18|
|erenumab||Amgen Inc||Central Nervous System||May-18|
|Lenvima (lenvatinib mesylate)||Eisai Inc||Oncology||May-18|
|pegvaliase [INN]||BioMarin Pharmaceutical Inc||Metabolic Disorders||May-18|
|trastuzumab biosimilar||Amgen Inc||Oncology||May-18|
|COMBO450 (binimetinib + encorafenib)||Array BioPharma Inc||Oncology||Jun-18|
|fremanezumab [INN]||Teva Pharmaceutical Industries Ltd||Central Nervous System||Jun-18|
|mogamulizumab||Kyowa Hakko Kirin Co Ltd||Oncology||Jun-18|
|cobicistat + darunavir + emtricitabine + tenofovir alafenamide||Johnson & Johnson||Infectious Disease||Jul-18|
|filgrastim||Adello Biologics LLC||Hematological Disorders||Jul-18|
|inotersen sodium||Ionis Pharmaceuticals Inc||Metabolic Disorders||Jul-18|
|rituximab biosimilar||Sandoz International GmbH||Oncology||Jul-18|
|ivosidenib||Agios Pharmaceuticals Inc||Oncology||Aug-18|
|migalastat hydrochloride||Amicus Therapeutics Inc||Genetic Disorders||Aug-18|
|patisiran [INN]||Alnylam Pharmaceuticals Inc||Metabolic Disorders||Aug-18|
|volanesorsen sodium||Akcea Therapeutics Inc||Metabolic Disorders||Aug-18|
|doravirine||Merck & Co Inc||Infectious Disease||Oct-18|
|doravirine + lamivudine + tenofovir disoproxil fumarate||Merck & Co Inc||Infectious Disease||Oct-18|
|Supplemental Indication Applications|
|Perjeta (pertuzumab)||Genentech Inc||20-Dec-2017||Oncology||Jan-18|
|Sutent (sunitinib malate)||Pfizer Inc||16-Nov-2017||Oncology||Jan-18|
|Cabometyx (cabozantinib s-malate)||Exelixis Inc||19-Dec-2017||Oncology||Feb-18|
|Makena (hydroxyprogesterone caproate)||AMAG Pharmaceuticals Inc||Women’s Health||Feb-18|
|Leukine (sargramostim)||Genzyme Corp||Toxicology||Mar-18|
|Trisenox (arsenic trioxide)||Teva Pharmaceutical Industries Ltd||12-Jan-2018||Oncology||Mar-18|
|Imfinzi (durvalumab)||MedImmune LLC||Oncology||Apr-18|
|ipilimumab + nivolumab||Bristol-Myers Squibb Co||Oncology||Apr-18|
|Keytruda (pembrolizumab)||Merck & Co Inc||Oncology||Apr-18|
|Opdivo (nivolumab)||Bristol-Myers Squibb Co||Oncology||Apr-18|
|Rubraca (rucaparib camsylate)||Clovis Oncology Inc||Oncology||Apr-18|
|Adcetris (brentuximab vedotin)||Seattle Genetics Inc||Oncology||May-18|
|Darzalex (daratumumab)||Genmab A/S||Oncology||May-18|
|Prolia (denosumab)||Amgen Inc||Metabolic Disorders||May-18|
|Tafinlar + Mekinist (dabrafenib mesylate + trametinib dimethyl sulfoxide)||Novartis AG||Oncology||May-18|
|Tagrisso (osimertinib mesylate)||AstraZeneca Plc||Oncology||May-18|
|Cinryze (C1 esterase inhibitor (human))||Shire Plc||Immunology||Jun-18|
|Xeljanz (tofacitinib citrate)||Pfizer Inc||Gastrointestinal||Jun-18|
|Kymriah (tisagenlecleucel)||Novartis AG||Oncology||Jul-18|
|Rituxan (rituximab)||Genentech Inc||Immunology||Jul-18|
|Eylea (aflibercept)||Regeneron Pharmaceuticals Inc||Ophthalmology||Aug-18|
|Ruconest (C1 esterase inhibitor (recombinant))||Pharming Group NV||Immunology||Sep-18|
Evaluation of the products slated for review by the FDA in 2018 shows another robust year for new products and supplemental indications for existing products. Oncology will continue to dominate the reviews and biologics will continue to be a significant component of the reviews. In the coming months we will be publishing information about the products covered in this article along with other relevant information to assist you in managing these products. Below are the publications so far for 2018:
- LUTATHERA ® (lutetium Lu 177 dotatate): Drug Alert – FDA Approval – lutetium Lu 177 dotatate injection, for intravenous use ( LUTATHERA ®)
- Hemlibra ® (emicizumab-kxwh): Hemlibra (emicizumab-kxwh) introductory pricing
- Rituxan ® (Rituximab) Biosimilars: Rituxan (Rituximab) Biosimilars – 2018 Drug Pipeline Review
Stay up to date on the latest drug news
1. PDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2018 THROUGH 2022. Available at: https://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm511438.pdf . Last accessed: February 20, 2018.
2. U.S. Food & Drug Administration. Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review. https://www.fda.gov/ForPatients/Approvals/Fast/default.htm Accessed February 23, 2018.
3. Pharma Intelligence Center, available at: https://pharma.globaldata.com/index.aspx . Last accessed: February 20, 2018.