Xena....here from Janet Woodcock's report on 2018 Drug Approvals is the part about CDER's expediting promising drugs. Given the visibility and the expected political pressure to address possibly the world's most pressing medical need, IMHO CDER will expedite A-273 with great vigor if the Australian trial is successful.It could possibly set a record as Dr Woodcock would love to have the first effective AZ drug approved on her watch.
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Innovation: Frequent Use of Expedited Development and Review Pathways
CDER used several regulatory pathways to enhance efficiency and expedite the development and approval of novel drugs in 2018. These pathways use a range of approaches, including more interactions between CDER staff and drug developers, greater program design flexibility, and shortened timelines for review of applications.
Fast Track
Fast Track-designated drugs have the potential to address unmet medical needs. CDER designated 24 of the 59 novel drugs (41%) in 2018 as Fast Track. Fast Track speeds new drug development and review by increasing the level of communication between FDA and drug developers, and by enabling CDER to review portions of a drug application ahead of the submission of the complete application.
Drugs designated with Fast Track status were: Aemcolo, Copiktra, Crysvita, Epidiolex, Erleada, Galafold, Lucemyra, Lumoxiti, Lutathera, Mulpleta, Nuzyra, Omegaven, Onpattro, Oxervate, Palynziq, Revcovi, Symdeko, Takhzyro, Tegsedi, Tibsovo, Tpoxx, Trogarzo, Xerava, and Xospata.
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Breakthrough Therapy
Breakthrough therapies are drugs for serious or life-threatening diseases for which there is unmet medical need and for which there is preliminary clinical evidence demonstrating that the drug may result in substantial improvement on a clinically significant endpoint (usually an endpoint that reflects how the patient feels, functions or survives) over other available therapies. CDER designated 14 of the 59 novel drugs (24%) in 2018 as breakthrough therapies. A breakthrough therapy designation includes all the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough therapy designation is designed to help shorten the development time of a potential new therapy.
Drugs designated with Breakthrough therapy status were: Crysvita, Elzonris, Firdapse, Gamifant, Krintafel, Libtayo, Lorbrena, Onpattro, Oxervate, Poteligeo, Symdeko, Takhzyro, Trogarzo, and Vitrakvi.
Priority Review
A drug receives a Priority Review if CDER determines that the drug could potentially provide a significant advance in medical care. The drug is reviewed in an expedited time line: within eight months instead of the standard 12 months. Forty-three of the 59 novel drugs approved in 2018 (73%) were designated Priority Review. Note, in some instances, priority review is assigned as a result of the sponsor redeeming a voucher for priority review under CDER’s Priority Review Voucher program, which may mean the drug does not potentially provide a significant advance. Such drugs are not included in the list below.
Drugs designated Priority Review were: Aemcolo*, Ajovy, Biktarvy, Copiktra, Crysvita, Daurismo, Diacomit, Doptelet, Elzonris, Epidiolex, Erleada, Firdapse, Galafold, Gamifant, Krintafel, Libtayo, Lorbrena, Lucemyra, Lumoxiti, Lutathera, moxidectin, Mulpleta, Nuzyra*, Omegaven, Onpattro, Orilissa, Oxervate, Palynziq, Poteligeo, Revcovi, Symdeko, Takhzyro, Talzenna , Tegsedi, Tibsovo, Tpoxx, Trogarzo, Vitrakvi, Vizimpro, Xerava*, Xofluza, Xospata, and Zemdri.*
* Aemcolo, Nuzyra, Xerava, and Zemdri received Priority Review as Qualified Infectious Disease Products (QIDPs) as authorized by the Generating Antibiotics Incentives Now Act (GAIN Act), which provides incentives to help bring new antibiotics and other antimicrobials to market. These products may or may not have otherwise received the priority review designation.
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Accelerated Approval
The Accelerated Approval program allows FDA more flexibility in what endpoints can be used to approve a drug that offers a benefit over current treatments for a serious or life-threatening illness. These accelerated approval endpoints may include ones that show benefits over a shorter duration of treatment (where longer term demonstration of benefit is needed for full approval) or are considered as “reasonably likely” to predict that an important clinical benefit will be seen. Subsequent confirmatory trials must be conducted to support full approval. CDER approved four of the 59 novel drugs (7%) in 2018 under the Accelerated Approval program. The application of accelerated approval brings drugs that can provide important advances to patients sooner than with traditional approvals.
Novel drugs approved in 2018 that received the Accelerated Approval designation were: Copiktra, Galafold, Lorbrena, and Vitrakvi.
Overall Use of Expedited Development and Review Methods
CDER used at least one expedited development and review method to speed approval for 73% of all novel drugs approved in 2018.
Forty-three of the 59 novel drug approvals of 2018 (73%) were designated in one or more expedited categories of Fast Track, Breakthrough, Priority Review, and/or Accelerated Approval.
Novel drugs approved in 2018 using at least one expedited approval method were: Aemcolo, Ajovy, Biktarvy, Copiktra, Crysvita, Daurismo, Diacomit, Doptelet, Elzonris, Epidiolex, Erleada, Firdapse, Galafold, Gamifant, Krintafel, Libtayo, Lorbrena, Lucemyra, Lumoxiti, Lutathera, moxidectin, Mulpleta, Nuzyra, Omegaven, Onpattro, Orilissa, Oxervate, Palynziq, Poteligeo, Revcovi, Symdeko, Takhzyro, Talzenna, Tegsedi, Tibsovo, Tpoxx, Trogarzo, Vitrakvi, Vizimpro, Xerava, Xofluza, Xospata, and Zemdri.
Predictability: Meeting PDUFA Goals
CDER met its target date for 100% of 2018’s novel drug approvals.
Under the Prescription Drug User Fee Act (PDUFA), sponsors are assessed user fees that provide FDA with the additional resources needed to maintain an efficient and effective review process. Throughout the year, CDER met or exceeded every PDUFA goal date for application review agreed to with the pharmaceutical industry and approved by Congress. In 2018, CDER met its PDUFA goal dates for 100% of the novel drugs approved (59 of 59).
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Access: First Cycle Approval and Approvals Compared to Other Countries
First Cycle Approval
First cycle approval prevents delays in bringing valuable new therapies to market.
CDER approved 56 of the 59 novel drugs of 2018 (95%) on the “first cycle” of review, meaning without a “complete response” letter from FDA that requires re-submission with additional information, resulting in more time before the drug can be approved. From 2011 through 2017, CDER approved 250 novel drugs, of which 205 (82%) were approved on the first cycle. This high proportion of first-cycle approval reflects the extent to which CDER staff and drug developers work together to ensure that the application contains the information CDER needs to be able to fully review, and if appropriate, approve an application.
Novel drugs approved in 2018 on the first cycle were: Aemcolo, Aimovig, Ajovy, Akynzeo, Annovera, Asparlas, Biktarvy, Braftovi, Copiktra, Crysvita, Daurismo, Diacomit, Doptelet, Elzonris, Emgality, Epidiolex, Erleada, Firdapse, Galafold, Gamifant, Ilumya, Krintafel, Libtayo, Lorbrena, Lucemyra, Lumoxiti, Mektovi, Motegrity, moxidectin, Mulpleta, Nuzyra, Omegaven, Onpattro, Orilissa, Oxervate, Palynziq, Pifeltro, Poteligeo, Revcovi, Seysara, Symdeko, Takhzyro, Talzenna, Tavalisse, Tegsedi, Tibsovo, Tpoxx, Trogarzo, Ultomiris, Vitrakvi, Vizimpro, Xerava, Xofluza, Xospata, Yupelri, and Zemdri.
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Approval in the United States Before Other Countries:
Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 42 of the 59 novel drugs approved in 2018 (71%) were approved in the United States before receiving approval in any other country.
71% of the novel drugs approved in 2018 were approved in the United States before any other country.
Novel drugs of 2018 approved first in the United States were: Aemcolo, Aimovig, Ajovy, Akynzeo, Annovera, Asparlas, Biktarvy, Braftovi, Copiktra, Daurismo, Doptelet, Elzonris, Emgality, Epidiolex, Erleada, Gamifant, Ilumya, Krintafel, Libtayo, Lumoxiti, Mektovi, moxidectin, Nuzyra, Onpattro, Orilissa, Palynziq, Pifeltro, Revcovi, Seysara, Symdeko, Takhzyro, Talzenna, Tavalisse, Tibsovo, Tpoxx, Trogarzo, Ultomiris, Vitrakvi, Vizimpro, Xerava, Yupelri, and Z
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