Diagram of the device, indicating External Trial Stimulator (ETS), Clinical Programmer (CP), Remote Control, Charger, Implantable Pulse Generator (IPG), Percutaneous Leads, and Surgical Paddle Leads.

Boston Scientific Spinal Cord Stimulation System

Boston Scientific 

INDICATION FOR USE:
Aid in the management of chronic intractable pain of the trunk and/or limbs including unilateral or bilateral pain associated with the following: failed back surgery syndrome, Complex Regional Pain Syndrome (CRPS) Types I and II, intractable low back pain and leg pain. Associated conditions and etiologies may be

  • radicular pain syndrome
  • radiculopathies resulting in pain secondary to failed back syndrome, herniated disc
  • epidural fibrosis
  • degenerative disc disease
  • arachnoiditis
  • multiple back surgeries

REGULATORY PATHWAY: PMA Supplement

DEVICE DESCRIPTION:

  • Implanted spinal cord stimulation system that was previously indicated as an aid in the management of chronic intractable pain of the trunk and/or limbs
  • Two main components include:
    • stimulator device (signal generator) implanted under the skin that sends electrical signals to the spinal cord through an insulated lead wire
    • hand-held remote control that allows the patient to control the implanted stimulator device in order to achieve the best pain control

EFFECTIVENESS AND SAFETY:

  • Based on published clinical studies (22 publications, 633 implanted patients) relevant to Spinal Cord Stimulation System features and indications
  • Improvement in pain ranged from 29.2%-100% for CRPS patients, and 37-77% for those with back and leg pain due to surgery associated conditions and etiologies across the studies
  • Most common adverse event: Need for an additional intervention (surgical revisions to correct lead migration, IPG discomfort, battery depletion, infection, fractured leads)
  • Other reported adverse events: Pain, unpleasant sensation, infection, inadequate stimulation, discomfort

REIMBURSEMENT:

  • ICD-10-CM (diagnosis) Coding Guide for Spinal Cord Stimulation
  • Documentation for medical necessity
  • Payer Medical Policy for Spinal Cord Stimulation

LABEL


Capture

ADMELOG (insulin lispro injection), subcutaneous or intravenous use

Sanofi 

INDICATION: Improve glycemic control in adults and pediatric patients 3 years and older with type 1 diabetes mellitus and adults with type 2 diabetes mellitus

ADDRESSING UNMET NEED: 

  • > 30 million diabetics in US
  • Increases risk of serious health complications, including heart disease, blindness, and nerve and kidney damage
  • Improvement in blood sugar control through insulin treatment
  • First short-acting insulin approved as a “follow-on” product

REGULATORY PATHWAY: NDA, 505(b)(2)

  • Relied on FDA’s finding finding of safety and effectiveness for Humalog (insulin lispro injection, Eli Lilly) to support approval
  • Demonstration that relianmalog safety and effectiveness was scientifically justified
  • Provided Admelog-specific data to establish the drug’s safety and efficacy for its approved uses

EFFICACY:

  • Clinical trials, adult and pediatric patients , Type I and II diabetes
  • Mean reduction in HbA1c that was non-inferior to that achieved with Comparator

SAFETY:

  • Most common adverse reactions: Hypoglycemia, itching, and rash
  • May cause low blood sugar (hypoglycemia), which can be life-threatening
  • Severe, life-threatening, generalized allergic reactions, including anaphylaxis, may occur

PRICING AND REIMBURSEMENT:

  • Increase competition in the market for prescription drugs
  • Lower-cost alternative

LABEL


CaptureIXIFI (infliximab-qbtx) injection

Pfizer

INDICATIONS: Crohn’s Disease, Pediatric Crohn’s Disease, Ulcerative Colitis, Rheumatoid Arthritis in combination with methotrexate, Psoriatic Arthritis, Plaque Psoriasis,  Psoriatic Arthritis and Plaque Psoriasis

ADDRESSING UNMET NEED: Third FDA-approved biosimilar to U.S.-licensed Remicade (infliximab)

REGULATORY PATHWAY: BLA

  • Biosimilarity based on a showing that it is highly similar to Remicade
    • No clinically meaningful differences in safety and effectiveness
    • Only minor differences in clinically inactive components 
  • Required pediatric assessments and postmarketing commitments

MECHANISM OF ACTION: Tumor necrosis factor (TNF) blocker

LABEL


Image result for nucala logoNUCALA (mepolizumab) injection

GlaxoSmithKline

INDICATION:  Treatment of adult patients with eosinophilic granulomatosis with
polyangiitis (EGPA)

ADDRESSING UNMET NEED:

  • Approximately 0.11 to 2.66 new cases per 1 million people are diagnosed each year with EGPPA, with an overall prevalence of 10.7 to 14 per 1,000,000 adults
  • First approved treatment for challenging, rare disease that can provide significant improvement in symptoms

REGULATORY PATHWAY: sNDA

  • Priority Review and Orphan Drug designations
  • Previously approved in 2015 to treat patients age 12 years and older with specific subgroup of asthma

EFFICACY:

  • Randomized, placebo-controlled, multicenter, 52-week trial, n=136, NUCALA vs placebo  while continuing their stable daily oral corticosteroids (OCS) therapy
  • Co-primary Endpoints: Total accrued duration of remission defined as Birmingham Vasculitis Activity Score (BVAS) = 0 (no active vasculitis) and proportion of subjects in remission
  •  Significantly greater accrued time in remission with NUCALA; significantly higher proportion of patients achieved remission at both week 36 and week 48
  • Significantly more patients achieved remission within the first 24 weeks and remained in remission for the remainder of the 52-week study treatment period

SAFETY:

  • Most common adverse reaction: Headache, injection site reaction, back pain, and fatigue

REIMBURSEMENT: GSK Patient assistance program

LABEL


CaptureLUXTURNA (voretigene neparvovec-rzyl)  intraocular suspension for
subretinal injection 

Spark Therapeutics

INDICATION: Adeno-associated virus vector-based gene therapy indicated for treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Patients must have viable retinal cells as determined by the treating physician(s).

ADDRESSING UNMET NEED:

  • Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in U.S.
  • Patients now have a chance for improved vision
  • First directly administered gene therapy targeting a disease caused by mutations in a specific gene
  • Reinforces potential of breakthrough approach in treating a wide-range of challenging diseases

REGULATORY PATHWAY: BLA

  • Priority Review and Breakthrough Therapy designations, Orphan Drug designation
  • Granted Rare Pediatric Disease Priority Review Voucher
  • Post-marketing observational study to evaluate long-term safety

MECHANISM OF ACTION:

  • Designed to deliver normal copy of gene encoding RPE65 to cells of the retina
  • RPE65 produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol during visual cycle critical in phototransduction
  • Mutations in the RPE65 gene blocks visual cycle resulting in impairment of vision

EFFICACY:

  • Open-label, two-center, randomized trial in pediatric and adult patients with biallelic RPE65 mutation-associated retinal dystrophy, n= 31 enrolled
  • Endpoint:  Multi-luminance mobility testing (MLMT) score change from Baseline to Year 1,  MLMT score change of two or greater considered clinically meaningful benefit in functional vision
  • Median MLMT score of 2 or greater with LUXTURNA vs 0 in control

SAFETY:

  • Most common adverse reactions: Eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear

REIMBURSEMENT:  

  • Could cost $1 million or more per patient
  • Will require assessment of coverage and reimbursement models

LABEL


Image credits: Boston Scientific, Sanofi, GSK, Pfizer, Spark

 

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