Annemans et al

Title: Real-Life Practice Study of the Clinical Outcome and Cost-Effectiveness of Photodynamic Therapy using Methyl Aminovulinate (MAL-PDT) in the Management of Actinic Keratosis and Basal Cell Carcinoma

Author: Annemans L, Caekelbergh K, Roelandts R, Boonen H, Leys C, Nikkels A, et al.

Journal Reference: Eur J Dermatol. 2008;18(5):539-46 (by clicking on this link you will be leaving this site - Galderma is not responsible for the content)

Key statements:

  • As shown by numerous randomized clinical trials (RCTs) before, this study under "real life conditions" demonstrates that methyl aminolevulinate photodynamic therapy (MAL-PDT) is a predictable and reliable treatment for actinic keratosis (AK) and basal cell carcinoma (BCC) lesions with high efficacy and very good cosmetic outcome.
  • This real-life study confirms the comparable efficacy and superior cosmetic outcome of MAL-PDT vs. the standard of care (cryotherapy for AK and superficial basal cell carcinoma [sBCC], excision surgery for nevoid basal cell carcinoma [nBCC]).
  • This real-life study demonstrates the cost-effectiveness of MAL-PDT in the management of AK and BCC. Results are in line with the cost prediction model, which adds to its predictive validity. More specifically:
    • Cost per lesion was almost identical for AKs (58.16 vs. 58.70 for the RCT) and within 20% for BCCs.
    • Cost of treatment (total and per lesion) for nBCC was higher than that for sBCC in both the real-life study and model due to a greater number of consultations and the use of a greater quantity of MAL-PDT.

Objective: To verify in a real-life practice study the results obtained in previous clinical trials with Metvix® in the treatment of AK, nBCC and sBCC, and to calculate the real-life cost of treatment and validate predictions from an economic evaluation model.

Design: this was a prospective, observational, one-arm, open study conducted by dermatologists in hospital institutions, in line with Belgian reimbursement criteria. There was a 6 month follow-up period from the date of the first application of Metvix®, during which the following outcomes were recorded:

  • Clinical response, assessed visually and by palpation, either as complete (disappearance of lesion) or incomplete. If incomplete, subsequent therapies were conducted (mainly cryotherapy for AK and Metvix® for BCC).
  • Cosmetic outcome classified using a 4-point scale: excellent, good, fair, poor.
  • Adverse events as a measure of patient tolerability.
  • Total cost of care and cost per lesion.

The above observational study outcomes were compared with the following reference Metvix® treatment models:

  • Clinical, cosmetic and tolerability data from RCTs: AK, Freeman et al. 2003; sBCC, Basset-Seguin et al. 2004; nBCC: Rhodes et al. 2004.
  • Health economics: updated model costs derived from the original, predictive model of Caekelbergh et al. 2006.

Key Results:

  • N=247; AK and BCC patients with average of 7.1 lesions and 1.7 at baseline, respectively.
    Complete clinical response at 6 months for the observational study and the RCTs are similar, as shown in Figure 1 (% of patients exhibiting good or excellent cosmetic outcome).
  • Patients with good or excellent cosmetic outcome after Metvix® treatment, observational study vs. RCTs results: AK 95% vs. 98%; sBCC 94% vs. 89%; nBCC 89% vs. 82%
  • Fewer AEs were reported with AK patients than in the RCT - 62% vs. 73% (mainly skin discomfort). AEs were reported by 51% of BCC patients.
  • Total cost of care in real-life study was higher compared with the model: AK 380.66 euros (average of 7.1 AKs) vs. 248.86 euros (average of four AKs); sBCC 297.92 vs. 227.49 euros; nBCC 318.16 vs. 278.01 euros.
  • Cost per lesion were similar for AKs (58.16 vs. 58.70 for the model), 18.1% lower than the model for sBCC and 17.5% higher than the model for nBCC.

Comments:

  • Costs elsewhere in the world are likely to be similar to, or lower than, those found in this study given the Belgium regulations (administration by a dermatologist within a hospital setting, and reimbursement criteria only allowing "difficult-to-treat" AK patients to receive Metvix®).
  • The same economic model could be used to evaluate the cost-effectiveness of Metvix® in other healthcare settings.

Conclusion: the clinical response, cosmetic outcome, and tolerability results of this real-life practice study confirm the efficacy and safety of Metvix® demonstrated in previous clinical trials. The costs calculated from this real-life practice study confirm the predicted cost-effectiveness shown in the original model for Metvix® in the management of AK and BCC.