BACKGROUND: Infantile spasms (IS) typically occur within the
first year of life. Children with IS frequently experience spasms,
hypsarrhythmia and psychomotor retardation. H.P. Acthar Gel® is an
adrenocorticotropic hormone (ACTH) analogue that is FDA approved
for the treatment of IS. Evidence-based guidelines recommend the use
of ACTH as first-line therapy for IS. Long-term prognosis of IS patients,
specifically neurodevelopmental outcomes, is relatively poor. Initiating
ACTH therapy soon after diagnosis may improve favorable outcomes
and may decrease the risk of mental retardation. Prompt diagnosis and
treatment may prevent developmental delays and may also reduce
healthcare utilization and costs.
OBJECTIVE: We compared the economic consequences of initiating
ACTH early (within 30 days of IS diagnosis) with that of late ACTH
treatment ( 30 days after diagnosis).
METHODS: All patients 30
days of the index date). Patients with continuous health plan enrollment
for 3-months prior and 12-months post index date were included in
this retrospective analysis. We estimated and compared the healthcare
resource use and costs (inpatient, outpatient, and pharmacy) separately
for patients in the two groups. Multivariate regression models were
constructed to adjust for gender and prior hospitalizations.
RESULTS: We identified a total of 259 IS patients who met our study
eligibility criteria. 197 (76%) patients used ACTH early and 62 (24%)
patients were late users of ACTH. Over the one-year follow-up from IS
diagnosis, early users had 16% fewer outpatient visits (95% CI: -13% to
-19%) and 15% less overall healthcare resource utilization (95% CI: -13%
to -18%) when compared to late ACTH users. Unadjusted 12-month
total outpatient costs (excluding the costs of administering ACTH)
for early users were 30% lower ($23,200 for early users as compared
to $33,500 for late users; 95% CI: -10% to -50%) as were the total
medication costs excluding cost of ACTH (50% reduction; 95% CI: -20%
to -70%). After adjusting for gender and prior hospitalizations, the
relative rate ratio of outpatient visits (0.89; 95% CI: 0.85 to 0.92), overall
healthcare resource utilization (0.91; 95% CI: 0.88 to 0.94) and total
medication costs (excluding ACTH costs) (0.5; 95% CI: 0.3 to 0.8) were
statistically significantly lower in early ACTH users than late users.
CONCLUSION: In this analysis, we found that the economic
consequences of treating patients with ACTH within 30 days of IS
diagnosis may be associated with decreases in overall healthcare
resource use, outpatient visits and total medication costs.
Anyone remember AFFY? I can't believe our mighty ACTH gel can also be used to treat dialysis outpatient.
Recent reports demonstrated Acthar gel is effective to induce remission of proteinuria in the Nephrotic Syndrome patients (Bomback 2011, 2012). There are limited published reports documenting the clinical response (creatinine, proteinuria, serum albumin and cholesterol) to ACTHAR therapy in a non-diabetic hemodialysis population. In addition to renal dysfunction and high risk for mortality, individuals who receive dialysis also have decreased strength, low exercise capacity, poor physical functioning, and a low quality of life (Edgell 1996, Johansen, 2001). Strength deficits are well documented in those with End Stage Renal Disease [ESRD] (Cheema 2010, Yoda 2012). There are several reports documenting an association between strength and gait deficits and other measures of physical performance in persons with ESRD (Bohannon 1994, Fitts 1997, Segura-Orti 2011). It is well known that measures of physical performance may predict risk for fall and hospitalization in older individuals (Guralnik JM, 2000).
Deficits to physical performance may be further compounded by poor nutritional status. Persons with chronic renal disease, particularly in the context of hemodialysis or chronic renal replacement therapy, are often malnourished and/or are affected by abnormal micronutrient status (McMahon 2012). Furthermore, previous studies have shown that more than a third of acute-care, nephrology-related admissions are characterized by malnutrition (Lim 2012), and that it affects from 23% - 76% of all patients receiving hemodialysis (including outpatients)(Blumenkrantz 1980, Ikizler 1996, Pecoits-Filho 2002).
Decreased functional status and concomitant malnutrition contribute to increased hospital readmissions, prolonged length of stay (and inevitably, increased medical costs), and increased morbidity and mortality patients (Isabel 2003, Lim 2012). Given the high prevalence of these characteristics in persons with renal disease, it is important to prioritize identification of novel and effective means by which to sustain and improve the functional capability of these patients, and to maintain their nutritional status and attenuate malnutrition.
To date, there are no reports of the impact of Acthar gel therapy on renal function, strength, physical performance, nutritional status and quality of life in NDHD patients. This study will determine if Acthar gel therapy will maintain or improve overall kidney function as measured with 24 hour urine study at baseline and at the end of study period. In addition this study will determine if Acthar gel therapy will improve nutritional, physical and biochemical status in an outpatient non-diabetic hemodialysis population.
Todd, It is dated on May 8, 2015. PLoS One. 2015 May 8;10(5):e0125638. doi: 10.1371/journal.pone.0125638. eCollection 2015.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with a complex etiology and pathology that makes the development of new therapies difficult. ACTH has neurotrophic and myotrophic effects, but has not been tested in an ALS mouse model. The G93A-SOD1 mouse model of ALS was used to test the ability of this drug to delay ALS-like symptoms. We showed that within a specific dose range, ACTH significantly postponed the disease onset and paralysis in the mouse model. To our surprise and of greater significance is that ACTH significantly reduced the levels of soluble SOD1 in the spinal cord and CNS tissues of G93A-SOD1 treated mice as well as cultured fibroblasts.
Hazelwood-based drugmaker Mallinckrodt PLC is among bidders for Boehringer Ingelheim's Roxane Labs business in the United States, according to people with knowledge of the matter.
Other potential buyers for the generic pharmaceutical unit, which may fetch as much as 2 billion euros, or $2.2 billion, include Hikma Pharmaceuticals and Perrigo, the people said, asking not to be identified because the process is private. A decision on the winner could be made within weeks, though the German drugmaker may still choose to keep the asset, they said.
Closely held Boehringer Ingelheim said in January that it's exploring strategic options for Roxane Labs, after Bloomberg News reported that the company was working with Morgan Stanley on the process. The unit comprises Roxane Laboratories, and its manufacturing and supply-chain affiliate, Boehringer Ingelheim Roxane Inc.
Options include asset swaps or a sale, the people said. While the drugmaker would prefer an asset swap, such a transaction may be more difficult to carry out than a straight sale, they said.
A spokeswoman for Boehringer said that the company began approaching interested parties for Roxane in April, but was not in a position to comment further on the process. Representatives for Hikma, Perrigo and Mallinckrodt declined to comment.
Citigroup analysts said in a research note in March that Roxane could be an attractive target for Hikma given its broad portfolio and its previous experience with the seller. Hikma last year acquired Bedford Laboratories assets from Boehringer Ingelheim's Ben Venue Laboratories, as well as an option to potentially acquire all of assets of the Ben Venue manufacturing facility in Bedford, Ohio, according to a statement from the company.
Faced with declining sales and expiring patents on certain portfolios, some pharmaceutical companies have been dividing assets into units that can then be split off, or beginning divestment processes for the drugs.
While both GlaxoSmithKline and Sanofi announced plans to sell parts of their business last year, the potential divestments were canceled.
UCB is trying again to sell its U.S. generics business, Kremers Urban Pharmaceuticals Inc., after a previous $1.5 billion sale to buyout firms agreed to in November fell apart.
Mallinckrodt shares fell 1 percent to $115.66 at the close in New York. Perrigo slid 3.4 percent to $184.14.
Mr. Carlson, is an executive and lawyer with an extensive background in international business, acquisitions, and government affairs. Mr. Carlson has handled a broad range of complex transactions across the world and has served as head of legal, government affairs, business development and compliance of publicly-traded companies. His experience in the medical device field includes serving as Senior Vice President, General Counsel, & Secretary of Medtronic, the world's largest independent medical device company, and Vice President & General Counsel of Synthes, which was acquired by Johnson & Johnson for $20 Billion in June 2012. Mr. Carlson has been honored by the Directors' Roundtable as Distinguished Global General Counsel, one of the “Top 45 Corporate Lawyers in the USA under Age 45” and, "Dealmaker of the Month” by Daily Deal Magazine.
Mr. Carlson received his JD from the University of Michigan, his BSB in Finance & Accounting from the University of Minnesota, and attended the General Manager executive program at Harvard Business School.