Updating Treatments for Fecal Incontinence III

1. What is the decision or change you are facing or struggling with where a summary of the evidence would be helpful?

The Evidence-Based Practice report on Fecal Incontinence dated March 2016 has been archived and labeled "not to be considered current." We are asking that  AHRQ update its March 21, 2016 systematic review entitled "Treatments for Fecal Incontinence" (FI) to reflect the latest evidence about treatment modalities, specifically to include the latest evidence about perianal bulking with non-animal stabilized hyaluronic acid/dextranomer [NASHA/Dx FI] ). NASHA/Dx FI Solesta^® is a biocompatible injectable bulking agent and is the only bulking agent approved by the U.S. Food and Drug Administration (FDA). Solesta was FDA approved in 2011 for the treatment of FI. At the time of the EBP review, there was no long-term data available covering the efficacy and durability of NASHA/Dx FI. However, such data are now available and should be factored into the FI treatment algorithm and associated recommendations. Moreover, other bulking agents other than NASHA/Dx FI are included in the EBP review and grouped into the same guidance, but have not been studied with scientific rigor to the degree as NASHA/Dx FI, nor have any achieved approval by FDA for the treatment of FI, so we struggle with AHRQ’s prior guidance generalized to all bulking agents, as they are not comparable.

2. Why are you struggling with this issue?

FI causes significant human suffering and socioeconomic burden. Patients suffering from FI have higher rates of unemployment, increased absenteeism, loss of productivity and reduced quality of life. NASHA/Dx FI fills an important gap as an effective FDA approved minimally invasive treatment for FI, with the potential to delay or entirely obviate the need for invasive surgery for FI management. A lack of updated guidelines limits the opportunity for patients to have access to NASHA/Dx FI as many Healthcare providers and payors look to this report for guidance on treatment and setting reimbursement policies. In the years since the publication of these guidelines, several important studies, including a 283-patient, FDA accepted Post Approval Study on NASHA/Dx FI, have demonstrated not only a significant reduction in symptom burden, but also a significant improvement in QoL through 36 months, including:

• NASHA/Dx FI is a safe, effective and durable treatment option for patients
• When compared to a sham arm (pivotal trial), a significantly greater percentage of patients receiving NASHA/Dx FI experienced at least a 50% reduction in incontinence free episodes, as well as a significant increase in the number of incontinence-free days from baseline through 36 months.
• NASHA/Dx was found to be efficacious in treating FI; >80% of patients did not require re-intervention through 36-months post-treatment.
• Treatment with NASHA/Dx significantly improves a patient's overall QoL and symptom burden over the long-term with improvement in each sub-scale of the FIQL and a reduction in symptom burden based on the CCFIS from baseline through 36-months post-treatment
• Treatment-related adverse events associated with NASHA/Dx treatment are low and NASHA/Dx demonstrates an excellent safety profile over the long-term.

This new evidence warrants an update, as the 2016 document ends with the words: "future studies with higher quality could change the conclusions of this review."

3. What do you want to see changed? How will you know that your issue is improving or has been addressed?

We would like to see updated guidelines with recognition that NASHA/Dx FI is the only FDA approved bulking agent and with all evidence of NASHA/Dx FI’s safety and effectiveness considered, including but not limited to:

• Mellgren et al., 2014, Publication: Reports long-term efficacy of NASHA/Dx at 12 and 36 months, not included among the sources cited for AHRQ's 2016 guideline. Concluded:
  • Efficacy of NASHA/Dx FI for reducing fecal incontinence episodes is stable for up to 3 years and that the incidence of adverse events remains low
  • Reduction of fecal incontinence episodes at 6 months was sustained at continued follow-up for 36 months
  • Cleveland Clinic Florida Fecal Incontinence Score improved (score reduction) significantly and Fecal Incontinence Quality of Life Scale scores improved significantly between baseline and 36 months
• Franklin et al., 2016, Publication: Analyzed & highlighted the benefit of NASHA/Dx FI vs Sham in specific key patient subgroups. Concluded:
  • Response to treatment was significantly greater with NASHA/Dx versus sham injection
  • Characteristics indicative of mild-to-moderate FI may exhibit the greatest benefit
• Al-Bayati I et al.,2017, Publication: Effectiveness of Bulking Agent Therapy in Fecal Incontinence in Patients Refractory to Conventional Therapies. Concluded:
  • Intrarectal injection of NASHA/Dx FI is effective for treating FI in patients who have failed standard medical treatments
  • The NASHA/Dx FI procedure is technically easy and safely performed as an outpatient procedure
• Danielson J, et al., 2020, Publication: Injectable bulking treatment of persistent fecal incontinence in adult patients after anorectal malformations Concluded:
  • NASHA/Dx FI is a promising treatment option for selected adult patients with persistent FI after anorectal malformations.
• NASHA/Dx FI Post Approval Study (PAS), 2020: 283-patient, 36-month, multi-center PAS with robust long-term outcomes data (recently accepted by FDA , abstract accepted, publication pending) Confirmed the benefits of NASHA/Dx FI treatment : Concluded:
  • Significant improvements in objective clinical measures, re-intervention rate (>80% of patients treated do not require re-intervention through 36-months post treatment)
  • Sustained statistically & clinically significant improvement in the CCFIS and FIQL scales
  • Durable and stable treatment outcome through 36 months
  • Real-world safety, efficacy, and medical appropriateness of NASHA/Dx FI
  • Study results reviewed by FDA and accepted on March 17,2020.  Initial publication abstract accepted for Pelvic Disease Week on June 23, 2020. Additional publications forthcoming and study data can be made available to AHRQ by the Palette Life Sciences.

In addition to the consideration of the above, we believe that this contemporary evidence warrants a recommendation for NASHA/Dx FI that is separate and distinct from other bulking agents, as none of the other agents are approved by FDA for this indication and none have produced evidence of sustained safety and effectiveness in real-world use.

4. When do you need the evidence report?

Monday, March 15, 2021

5. What will you do with the evidence report?

We will use the updated guideline primarily to educate physicians, patients, and third-party payers with respect to the current state of evidence in the treatment of fecal incontinence, which affects up to 8.3% of non-institutionalized adults, drives over $11B in annual healthcare spending and increases the likelihood of referral to a nursing home by 10–15%.

Supporting Document

Title or short description: Value Dossier, review of  FI disease state, therapeutic options and new data (PDF, 3.13 MB)

Comments or notes about this file: This dossier has a review of current data available including an extensive bibliography at the back of the document. Information in this dossier is intended to be used to inform medical policy coverage review. Please feel free to reach out to me for any additional questions or information.

(Optional) About You

What is your role or perspective? Represent the US Distributor for Solesta after many years in clinical arena

If you are you making a suggestion on behalf of an organization, please state the name of the organization: Palette Life Sciences

May we contact you if we have questions about your nomination? Yes