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
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
IXIFI (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
NUCALA (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
LUXTURNA (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
Image credits: Boston Scientific, Sanofi, GSK, Pfizer, Spark