Specialty Drug 2018 Prescription Drug User Fee Act (PDUFA) Schedule

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 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).

Figure 1- Number of drugs with established PDUFA dates by month in 2018

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).

Figure 2 – Type of PDUFA review by therapeutic class

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).

Figure 3 – Molecular type by therapeutic class for drugs with established PDUFA dates in 2018

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 ).

Figure 4 – Route of Administration of 2018 products

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).

Figure 5 – Drug application type by Month

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
lorlatinib Pfizer 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:

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.