Latest Review Date: July 2024

Category: Medical    

POLICY:

The use of a drug-coated urethral balloon (e.g., Optilume® Urethral Drug-Coated Balloon Catheter System) is considered investigational for all indications including but not limited to the treatment of obstructive urinary symptoms associated with urethral stricture.

DESCRIPTION OF PROCEDURE OR SERVICE:

The Optilume drug-coated balloon offers a minimally invasive treatment option for men suffering from urinary tract conditions like urethral strictures. Urethral strictures are often caused by infections, trauma, and other medical procedures that injure the lining of the urethra. Close to 95-98% of urethral strictures in the United States are treated with endoscopic means, meaning with a dilation or urethrotomy. An open surgical procedure called urethroplasty, another treatment option for urethral stricture, is noted to have a better success rate however, it may require a longer recovery and possible side effects. The Optilume drug-coated balloon is a guidewire-compatible catheter with a tapered atraumatic tip. The distal end of the catheter has an inflatable balloon coated with a proprietary coating containing the drug paclitaxel that facilitates the drug's transfer to the urethral wall upon inflation. ECRI (Emergency Care Research Institute) noted in their Clinical Evidence Assessment on Optilume Drug-coated Balloon for Treating Urethral Stricture Disease (2022) that the evidence was inconclusive. The following evidence gaps were noted: Additional double-blind RCTs comparing Optilume with other urethral stricture treatments and reporting on patient-oriented outcomes at longer follow-up times (>2years) are needed to assess Optilume’s comparative safety and effectiveness. Three ongoing clinical studies may provide additional data and may partially address evidence gaps

For information regarding Optilume for the treatment of BPH, please refer to medical policy #725 Treatment of Benign Prostatic Hyperplasia.

KEY POINTS:

This evidence review was created and has been updated regularly with searches of the PubMed database. Most recently, the literature was reviewed through June 26, 2024.

Summary of Evidence

For individuals with obstructive urinary symptoms associated with anterior urethral stricture; the use of a device with a drug-coated urethral balloon was designed to be used in adult males for anterior urethral strictures of ≤3 cm in length. Data from one RCT and two single-arm studies reported significantly reduced stricture recurrence, increased urinary flow rate, and improved urinary symptom scores. The RCT reported significantly greater improvement with the drug-coated balloon versus endoscopic management. Drug-coated balloon treatment was more durable than endoscopic treatment at one-year follow-up. Limitations included most participants had bulbar urethral strictures, so it is unknown whether the treatment is generalizable to all types of urethral strictures; hematuria was more common with the drug-coated balloon; and this treatment has not been compared to urethroplasty which is most successful for treating recurrent strictures. In these studies, long-term follow-up beyond two years occurred only in small single-arm studies. Further, additional long-term data on paclitaxel in tissues is needed to assess device safety as is the effectiveness of treatment with an urethral drug-coated balloon compared with urethroplasty. The evidence is insufficient to determine that the technology results in an improvement in the net health outcomes.

Practice Guidelines and Position Statements

American Urological Association (AUA)

In 2023, the AUA published an amendment to the 2016 guidelines for the treatment of urethral stricture disease. These guidelines state, “Surgeons may offer urethral dilation or direct visual internal urethrotomy, combined with drug-coated balloons, for recurrent bulbar urethral strictures less than three cm in length (Conditional Recommendation; Evidence Level: Grade B). A Conditional Recommendation with Evidence Level B is defined as “Benefits equal to risk/burdens; the best action appears to depend on individual patient circumstances; better evidence could change confidence”

European Association of Urology (EAU)

In 2023 the EAU published a guideline on Urethral Strictures which stated:

• “Drug (paclitaxel)-coated balloon dilatation is associated with higher anatomic patency rates (at six months) and lower risk of retreatment (at one year) as compared to standard dilatation/DVIU in patients with short (< 3 cm), bulbar strictures that underwent at least two prior failed endoscopic treatments. (LE: 1b).”
• "Offer drug (paclitaxel)-coated balloon dilatation for a short (< 3 cm) bulbar stricture recurring after at least two prior endoscopic treatments, but only in patients for whom urethroplasty is not an option. (Strength rating: weak)".
• "Paclitaxel was detected in semen up to six months after treatment which urges for contraception if the partner has child-bearing potential".

National Institute for Health and Care Excellence (NICE)

The National Institute for Health and Care Excellence noted in their Medtech innovation briefing on Optilume for anterior urethral strictures (2021):

• The main points from the evidence summarised in this briefing are from 1 single-arm prospective study including a total of 53 men. It shows that Optilume is safe for treating urethral strictures, and early 2-year efficacy results are encouraging.
• Key uncertainties around the evidence or technology are that there are no comparative studies, the evidence has limited applicability to the NHS [National Health Service], and longer-term evidence is needed for Optilume, including the effect of paclitaxel on the tissues.

U.S. Preventive Services Task Force Recommendations

Not applicable.

KEY WORDS:

Optilume, Urethral Drug Coated Balloon (DCB)

APPROVED BY GOVERNING BODIES:

In December 2021, the FDA approved the Optilume Urethral Drug Coated Balloon (DCB) through the premarket approval application (PMA) process to be used to treat patients with obstructive urinary symptoms associated with anterior urethral stricture. The device is designed to be used in adult males for urethral stricture of ≤3 cm in length.

BENEFIT APPLICATION:

Coverage is subject to member’s specific benefits. Group-specific policy will supersede this policy when applicable.

ITS: Home Policy provisions apply.

FEP: Special benefit consideration may apply. Refer to member’s benefit plan.

CURRENT CODING: 

CPT Codes:

PREVIOUS CODING:

REFERENCES:

1. DeLong J.M., Ehlert M.J., Erickson B.A., et al. One-Year Outcomes of the ROBUST II Study Evaluating the Use of a Drug-Coated Balloon for Treatment of Urethral Stricture. Société Internationale d'Urologie Journal. 2022;3(1):2127.
2. EAU Guidelines. Edn. presented at the EAU Annual Congress Milan 2023. ISBN 978-94-92671-19-6. https://d56bochluxqnz.cloudfront.net/documents/full-guideline/EAUGuidelines-on-Urethral-Strictures-2023.pdf.
3. ECRI Institute. Optilume Drug-coated Balloon (Urotronic, Inc.) for Treating Urethral Stricture Disease. Plymouth Meeting (PA): ECRI Institute; 2022 March. (Clinical Evidence Assessment).
4. Elliott SP, Coutinho K, Robertson KJ, et al. One-Year Results for the ROBUST III Randomized Controlled Trial Evaluating the Optilume Drug-Coated Balloon for Anterior Urethral Strictures. J Urol. Apr 2022; 207(4):866-875.
5. Elterman DS, Coutinho K, Hagedorn JC. How I Do It: The Optilume drug-coated balloon for urethral strictures. Can J Urol. 2020 Aug;27(4):10322-10328.
6. Hayes, Inc. Evidence Analysis Research Brief. Optilume Urethral Drug-Coated Balloon (Laborie) for Treatment of Urethral Strictures. Hayes, Inc.; May 10, 2023
7. IOM (Institute of Medicine). 2011. Clinical Practice Guidelines We Can Trust. Washington, DC: The National Academies Press.
8. Kaplan SA, Moss J, Freedman S, et al. The PINNACLE Study: A double-blind, randomized, sham-controlled study evaluating the Optilume BPH Catheter System for the treatment of lower urinary tract symptoms secondary to benign prostatic hyperplasia. J Urol. September 2023; 210(3):500-509.
9. Kaplan SA, Pichardo M, Rijo E, et al. One-year outcomes after treatment with a drug-coated balloon catheter system for lower urinary tract symptoms related to benign prostatic hyperplasia. Prostate Cancer Prostatic Dis. December 2021; 24(4):1073-1079
10. Mann RA, Virasoro R, DeLong JM, et al. A drug-coated balloon treatment for urethral stricture disease: Two-year results from the ROBUST I study. Can Urol Assoc J. February 2021; 15(2):20-25
11. National Institute for Health and Care Excellence (NICE). Medical technologies guidance. Optilume for treating recurrent bulbar urethral strictures. November 29, 2022. Available at: www.nice.org.uk/guidance/mtg73.
12. National Institute for Health and Care Excellence (NICE). Optilume for anterior urethral strictures. National Institute for Health and Care Excellence. Medtech innovation briefing (January 2021) (MIB241). Available at:www.nice.org.uk
13. Optilume® Drug Coated Balloon. Manufacturer Information. Available at: www.urotronic.com
14. Pichardo M, Rijo E, Espino G, et al. Durable benefit after treatment of obstructive benign prostatic hyperplasia with a novel drug-device combination product: 2-year outcomes from the EVEREST-I study. World J Urol. 2023; 41(8):2209-2215.
15. SP Elliott, K Coutinho, KJ Robertson, et al. One-Year Results for the ROBUST III Randomized Controlled Trial Evaluating the Optilume® Drug-Coated Balloon for Anterior Urethral Strictures. J Urol. 2022;207(4):866-75.
16. U.S. Food and Drug Administration (FDA) Summary of Safety and Effectiveness Data. Optilume® Urethral Drug Coated Balloon. June 30, 2023. Available www.accessdata.fda.gov/cdrh_docs/pdf21/P210020B.pdf.
17. VanDyke ME, Morey AF, Coutinho K, et al. Optilume drug-coated balloon for anterior urethral stricture: 2-year results of the ROBUST III trial. BJUI Compass. 2024;5(3):366–73.
18. Virasoro R, DeLong JM, Estrella R, et al. A Drug-Coated Balloon Treatment for Urethral Stricture Disease: Three-Year Results from the ROBUST I Study. Res Rep Urol. May 6 2022; 14:177-183.
19. Wessells H, Morey A, Souter L, et al. Urethral Stricture Disease Guideline Amendment (2023). J Urol. 2023 Jul;210(1):64-71.

POLICY HISTORY:

Medical Policy Group, July 2024 (11): Created new medical policy for Urethral Drug Coated Balloons for the treatment of Urethral Strictures. Optilume previously investigational per MP 495 Investigational Criteria. On draft from 8/1/24-9/15/24.

Medical Policy Administration Committee, July 2024

This medical policy is not an authorization, certification, explanation of benefits, or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All medical policies are based on (i) research of current medical literature and (ii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.

This policy is intended to be used for adjudication of claims (including pre-admission certification, pre-determinations, and pre-procedure review) in Blue Cross and Blue Shield’s administration of plan contracts.

The plan does not approve or deny procedures, services, testing, or equipment for our members. Our decisions concern coverage only. The decision of whether or not to have a certain test, treatment or procedure is one made between the physician and his/her patient. The plan administers benefits based on the member’s contract and corporate medical policies. Physicians should always exercise their best medical judgment in providing the care they feel is most appropriate for their patients. Needed care should not be delayed or refused because of a coverage determination.

As a general rule, benefits are payable under health plans only in cases of medical necessity and only if services or supplies are not investigational, provided the customer group contracts have such coverage.

The following Association Technology Evaluation Criteria must be met for a service/supply to be considered for coverage:

1. The technology must have final approval from the appropriate government regulatory bodies;

2. The scientific evidence must permit conclusions concerning the effect of the technology on health outcomes;

3. The technology must improve the net health outcome;

4. The technology must be as beneficial as any established alternatives;

5. The improvement must be attainable outside the investigational setting.

Medical Necessity means that health care services (e.g., procedures, treatments, supplies, devices, equipment, facilities or drugs) that a physician, exercising prudent clinical judgment, would provide to a patient for the purpose of preventing, evaluating, diagnosing or treating an illness, injury or disease or its symptoms, and that are:

1. In accordance with generally accepted standards of medical practice; and

2. Clinically appropriate in terms of type, frequency, extent, site and duration and considered effective for the patient’s illness, injury or disease; and

3. Not primarily for the convenience of the patient, physician or other health care provider; and

4. Not more costly than an alternative service or sequence of services at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of that patient’s illness, injury or disease.