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Adcetris is the first ultra-orphan medicine recommended by AWMSG via the new assessment process

Takeda UK Ltd announced that brentuximab vedotin (Adcetris®) is the first medicine to be recommended for use in NHS Wales via the AWMSG’s new policy relating to ultra-orphan medicines. (1) Brentuximab vedotin has been recommended for restricted use for the treatment of adult patients with CD30+ relapsed or refractory Hodgkin lymphoma (HL) following an autologous stem cell transplant (ASCT) or following at least two previous therapies, when ASCT or multi-agent chemotherapy is not a treatment option. (1,2)

The availability of this cancer treatment offers further hope to patients in Wales with this stage of disease, who have limited effective treatment options remaining.

Commenting on the AWMSG decision, Dr Eve Gallop-Evans, Consultant in Oncology at Velindre Cancer Centre, Cardiff said “This decision is great news for eligible patients in Wales, as it provides access to an innovative treatment option for patients with Hodgkin lymphoma which has not responded to standard treatments. Results from real-life clinical practice and clinical trials for brentuximab vedotin are impressive, with efficacy data that is rarely seen with a single-agent therapy together with a very acceptable side effect profile”.

It was agreed with NHS Wales that brentuximab vedotin would be the pilot medicine reviewed by the AWMSG’s new policy relating to ultra-orphan medicines. The policy applies to medicines with orphan designation in the EU that are licenced for the treatment of diseases with a prevalence of less than one in 50,000 in the EU. (3) The review process adopts a broader approach to reviewing medicines, including considering degree of severity of the disease as presently managed, the impact of the medicine, whether the medicine may bridge a gap to a “definitive” therapy, as well as the innovative nature of the medicine. (3) In October 2014, brentuximab vedotin was also the first to be evaluated through the Scottish Medicines Consortium’s (SMC) new ultra-orphan decision making framework, recommending access to eligible HL patients in Scotland. (4)

In the pivotal phase II trial for brentuximab vedotin, 75% of patients with relapsed or refractory HL, post ASCT, achieved the primary end point of objective response rate (ORR), with 33% achieving a complete remission (CR). (5)

After a median of 32.7 months since their first dose of brentuximab vedotin, 50% of patients (51 of 102 patients) were alive at the time of last follow-up. The median overall survival (OS) was 40.5 months (95% CI: 28.7, – [range, 1.8 to 48.3 months]) with an estimated three-year survival rate of 54%. (5)

Data for patients with relapsed or refractory HL without prior ASCT were collected from Phase I dose escalation and clinical pharmacology studies (n=15) and a Named Patient Programme (NPP; n=26). Patients were dosed at 1.8mg/kg every three weeks. (2) 54% of relapsed or refractory ASCT-naïve HL patients achieved partial and/or CR with brentuximab vedotin and 19% of patients went on to receive a stem cell transplant following treatment. (2,6)

Brentuximab vedotin is also licenced for the treatment of adult patients with relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). (2) The AWMSG has not recommended brentuximab vedotin for use in relapsed or refractory sALCL as Takeda UK Ltd. has, to date, been unable to produce a health economic model for this indication due to the rarity of the condition. (1) Healthcare Professionals can still apply for funding for patients with relapsed or refractory sALCL through Individual Funding Requests (IFRs).

Brentuximab vedotin has a manageable side effect profile. (2) Further information on brentuximab vedotin can be found in the Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL) which can be obtained at


  1. All Wales Medicines Strategy Group. Final Appraisal Recommendation – 1215: brentuximab vedotin (Adcetris®) 50mg powder for concentrate for solution for infusion. May 2015. Available at: Last accessed June 2015.
  2. Adcetris Summary of Product Characteristics (SmPC). Available at:
  3. All Wales Medicines Strategy Group. AWMSG Policy relating to ultra-orphan medicines. Mar 2013. Available at: Last accessed June 2015.
  4. Scottish Medicines Consortium (SMC). Brentuximab vedotin (Adcetris®). Detailed Advice Document No. 989/14. Published 13th October 2014. Available at:
  5. Gopal et al. Three-year Follow-up Data and Characterization of Long-Term Remissions from an Ongoing Phase 2 Study of Brentuximab Vedotin in Patients with Relapsed or Refractory Hodgkin Lymphoma. Abstract No. 4382. American Society of Hematology, December, 2013, New Orleans, LA, USA.
  6. European Public Assessment Report (EPAR) 19 July 2012. EMA/702390/2012. Available at: Last accessed: June 2015.

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