As Humanity succed on “making Pandemy History ” How to Strike Balance between Drug Approvals and Public Safety?

by Yavuz Selim SILAY M.D. , M.B.A

As we transition to a new digital era of “making the pandemy history ” policymakers will have to decide whether to retain these changes and watch for the top 11 trends in healthcare for 2022 & beyond https://opinyu.com/drysilay/trends-in-healthcare/ (1) which has succesfully expedited access to new pharmaceuticals and innovative medical products , potentially improved health outcomes for patients & caregivers , less expensive physical and digital healthcare products ; which also means decreased cost to the overall healthcare system.

While Most industry executives remain upbeat about the hunt for new medicines , medical devices and digital healthcare solutions, it remains challenging to get new drugs through the approval process and to secure a decent financial return once they are launched, given resistance from healthcare insurers & governments to the rising cost of medical treatments.

Increasing political pressure over the high prices of many new medicine has always been a challenge at a time when pharmaceutical and biotech , medtech app companies are developing more drugs and digital healthcare products targeted for niche patient populations.

On one hand the effect of Pandemy generated a significant sense of urgency resulted in a faster pandemic response and faster drug approval process; there are trade offs in regards to Public safety and risk. The question is whether FDA’s regulatory response strategy to public health emergency is appropriate as we transition to a postpandemic era…

There are many benefits of providing a greater flexibility in regulatory & market access to pharmaceutical companies driving innovation thanks to FDA, despite suspending routine on-site inspections for facilitating the process of relying on company documents and past inspection documents to ensure safety , loosening regulatory restrictions for many companies that have to change suppliers or switch certain medical device components because of disruption of the Global Supply Chain, manufacturing delays. During the covid19 crisis FDA has facilitated importation of personal protective equipment,medical devices and clearance of digital healthcare apps. One of the most important steps was reducing the data requirements for clinical trials and guiding the Clinical Research Community by issuing guidelines for safely conducting Clinical Trials while complying with social distancing because without relaxing in-person safety protocols & requirements Healthcare Providers would not been even able to administer certain drugs… Kudos to FDA for their vision in implementing some of these important steps that continued to improve patients and caregivers lives .

Back on June 15 2021, FDA revoked the EUA for the unapproved uses of Hydroxychloroquine & Chloroquine phosphate in hospitalized covid-19 patients as new studies show greater risk and less benefit. Unfortunately this contributed to the sense of public confusion on if these medications are legitimate treatments for COVID-19.

Innovation drives progress for humanity. When it comes to innovation in the development of new drugs and therapeutic biological products, the role of FDA, EMA, SFDA ( Chinese FDA) , TİTCK ( Turkish Medicine & Medical Device Agency) , PMDA ( Japanese Pharmaceutical & Medical Device Agency ) and MHRA ( Medicines & Healthcare Products Regulatory Agency) and other national agencies are crucial.

Innovative drugs often mean new treatment options for patients and advances in health care for the humanity. When it comes the development of new drugs and therapeutic biological products, FDA’s Center for Drug Evaluation and Research (CDER) provides clarity to drug developers on the necessary study design elements and other data needed in the drug application to support a full and comprehensive assessment. To do so, CDER relies on its understanding of the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat. (2)

I was impressed with the FDA’s initiative of educating the public by providing information through” know your treatment options for covid19″ and most importantly ” Coronavirus Treatment Acceleration Program” by taking a public- private approach to bring coronavirus treatments to market as fast as possible which has brought new approvals of new and effective drugs and potentially avoided further loss of lives as US has been significantly impacted with about 900000 lives lost out of 6 M lives to covid19 (4)

Lets take a look at the New Drugs at FDA : Approvals in 2022 & 2021 as of February 1 2022

Novel Drug Approvals for 2022

3. 
   Kimmtrak

tebentafusp-tebn 1/25/2022

2. Cibinqo abrocitinib 1/14/2022
3. Quviviq daridorexant 1/7/2022
   (3)

Novel Drug Approvals for 2021

No. Drug Name Active Ingredient Approval Date FDA-approved use on approval date*

50. Adbry tralokinumab-ldrm 12/27/2021 To treat moderate-to-severe atopic dermatitis
51. Leqvio inclisiran 12/22/2021 To treat heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease as an add-on therapy
52. Vyvgart efgartigimod alfa-fcab 12/17/2021 To treat generalized myasthenia gravis
    Press Release
53. Tezspire tezepelumab-ekko 12/17/2021 To treat severe asthma as an add-on maintenance therapy
54. Cytalux pafolacianine 11/29/2021 To help identify ovarian cancer lesions
    Press Release
55. Livtencity maribavir 11/23/2021 To treat post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral treatment for CMV
    Press Release
56. Voxzogo vosoritide 11/19/2021 To improve growth in children five years of age and older with achondroplasia and open epiphyses
    Press Release
    43 Besremi ropeginterferon alfa-2b-njft 11/12/2021 To treat polycythemia vera, a blood disease that causes the overproduction of red blood cells
    Press Release
57. Scemblix asciminib 10/29/2021 To treat Philadelphia chromosome-positive chronic myeloid leukemia with disease that meets certain criteria
58. Tavneos avacopan 10/7/2021 To treat severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis) in combination with standard therapy, including glucocorticoids
59. Livmarli maralixibat 9/29/2021 To treat cholestatic pruritus associated with Alagille syndrome
60. Qulipta atogepant 9/28/2021 To prevent episodic migraines
61. Tivdak tisotumab vedotin-tftv 9/20/2021 To treat recurrent or metastatic cervical cancer with disease progression on or after chemotherapy
62. Exkivity mobocertinib 9/15/2021 To treat locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations
63. Skytrofa lonapegsomatropin-tcgd 8/25/2021 To treat short stature due to inadequate secretion of endogenous growth hormone
64. Korsuva difelikefalin 8/23/2021 To treat moderate-to-severe pruritus associated with chronic kidney disease in certain populations
65. Welireg belzutifan 8/13/2021 To treat von Hippel-Lindau disease under certain conditions
66. Nexviazyme avalglucosidase alfa-ngpt 8/6/2021 To treat late-onset Pompe disease
    Press Release
67. Saphnelo anifrolumab-fnia 7/30/2021 To treat moderate-to severe systemic lupus erythematousus along with standard therapy
68. Bylvay odevixibat 7/20/2021 To treat pruritus
69. Rezurock belumosudil 7/16/2021 To treat chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy
70. fexinidazole fexinidazole 7/16/2021 To treat human African trypanosomiasis caused by the parasite Trypanosoma brucei gambiense
71. Kerendia finerenone 7/9/2021 To reduce the risk of kidney and heart complications in chronic kidney disease associated with type 2 diabetes
72. Rylaze asparaginase erwinia chrysanthemi (recombinant)-rywn 6/30/2021 To treat acute lymphoblastic leukemia and lymphoblastic lymphoma in patients who are allergic to E. coli-derived asparaginase products, as a component of a chemotherapy regimen
    Press Release
73. Aduhelm aducanumab-avwa 6/7/2021 To treat Alzheimer’s disease
    Press Release
74. Brexafemme ibrexafungerp 6/1/2021 To treat vulvovaginal candidiasis
75. Lybalvi olanzapine and samidorphan 5/28/2021 To treat schizophrenia and certain aspects of bipolar I disorder
76. Truseltiq infigratinib 5/28/2021 To treat cholangiocarcinoma whose disease meets certain criteria
77. Lumakras sotorasib 5/28/2021 To treat types of non-small cell lung cancer
    Press Release
78. Pylarify piflufolastat F 18 5/26/2021 To identify prostate-specific membrane antigen-positive lesions in prostate cancer
79. Rybrevant amivantamab-vmjw 5/21/2021 To treat a subset of non-small cell lung cancer
    Press Release
80. Empaveli pegcetacoplan 5/14/2021 To treat paroxysmal nocturnal hemoglobinuria
81. Zynlonta loncastuximab tesirine-lpyl 4/23/2021 To treat certain types of relapsed or refractory large B-cell lymphoma
82. Jemperli dostarlimab-gxly 4/22/2021 To treat endometrial cancer
    Press Release
83. Nextstellis drospirenone and estetrol 4/15/2021 To prevent pregnancy
84. Qelbree viloxazine 4/2/2021 To treat attention deficit hyperactivity disorder
85. Zegalogue dasiglucagon 3/22/2021 To treat severe hypoglycemia
86. Ponvory ponesimod 3/18/2021 To treat relapsing forms of multiple sclerosis
87. Fotivda tivozanib 3/10/2021 To treat renal cell carcinoma
    11 Azstarys serdexmethylphenidate and
    dexmethylphenidate 3/2/2021 To treat attention deficit hyperactivity disorder
88. Pepaxto melphalan flufenamide 2/26/2021 To treat relapsed or refractory multiple myeloma
89. Nulibry fosdenopterin 2/26/2021 To reduce the risk of mortality in molybdenum cofactor deficiency Type A
    Press Release
90. Amondys 45 casimersen 2/25/2021 To treat Duchenne muscular dystrophy
    Press Release
91. Cosela trilacicilib 2/12/2021 To mitigate chemotherapy-induced myelosuppression in small cell lung cancer
    Press Release
92. Evkeeza evinacumab-dgnb 2/11/2021 To treat homozygous familial hypercholesterolemia
93. Ukoniq umbralisib 2/5/2021 To treat marginal zone lymphoma and follicular lymphoma
94. Tepmetko tepotinib 2/3/2021 To treat non-small cell lung cancer
95. Lupkynis voclosporin 1/22/2021 To treat lupus nephritis
    Drug Trials Snapshot
96. Cabenuva cabotegravir and rilpivirine (co-packaged) 1/21/2021 To treat HIV
    Press Release
    Drug Trials Snapshot
97. Verquvo vericiguat 1/19/2021 To mitigate the risk of cardiovascular death and hospitalization for chronic heart failure
    Drug Trials Snapshot
    *The listed “FDA-approved use” that has taken from FDA website is for presentation purposes only. To see the FDA-approved conditions of use [e.g., indication(s), population(s), dosing regimen(s)] for each of these products, see the most recent FDA-approved Prescribing Information (Please go to FDA website and click on the Drug Name) . (2)

My take home message is as we transition to a new digital era of “making the pandemy history ” policymakers will have to decide whether to retain these changes and watch for the top 11 trends in healthcare for 2022 & beyond https://opinyu.com/drysilay/trends-in-healthcare/ (1) which has succesfully expedited access to new pharmaceuticals and innovative medical products , potentially improved health outcomes for patients & caregivers , less expensive physical and digital healthcare products ; which also means decreased cost to the overall healthcare system.

References:

1) https://opinyu.com/drysilay/trends-in-healthcare/

2) https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021

3) https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2022

4) https://ourworldindata.org/coronavirus/country/united-states