CPhI Online is the largest global marketplace in the pharma ingredients industry
NICE Recommendation Set to Leave Patients with Newly Diagnosed Advanced Melanoma Without Access to Yervoy (Ipilimumab)
Bristol-Myers Squibb has expressed its disappointment that the National Institute for Health and Care Excellence (NICE) has issued draft guidance which effectively denies access to Yervoy (ipilimumab) for NHS patients with previously-untreated advanced (unresectable or metastatic) melanoma, not involved in clinical trials.
Ipilimumab is currently recommended by NICE for the treatment of adults with previously treated advanced melanoma and, in November 2013, European regulators extended its licence to include use in previously untreated patients, recognising both its potential to significantly increase overall survival and the unmet need in this patient population. Unless reversed, this decision will restrict access to all NHS patients that have not had prior therapy. Ipilimumab is a fully human monoclonal antibody that works by stimulating the patient’s own immune system to fight the cancer and is one of the most significant treatment advances for this disease in many years.
The National Cancer Drug Fund (NCDF) panel has reviewed ipilimumab in previously untreated patients and decided to refer it for baseline commissioning by NHS England. This is a 6-month process, during which time patients will not be able to access ipilimumab through the National Cancer Drugs Fund.
“Clinically, there is little difference between pre-treated and untreated advanced melanoma patients and ipilimumab is licensed to treat both types,” said, Professor John Wagstaff, Consultant Oncologist at the South West Wales Cancer Institute & Swansea School of Medicine. “Today’s decision will be disappointing news for many patients with advanced melanoma who have not received prior treatment, leaving them without immediate access to this innovative therapy. Ipilimumab has demonstrated its ability to increase overall survival in this cancer, in some cases having a long-term effect on the survival of previously-treated patients. Making it available as a first treatment option should therefore be seen as an important step in helping to maximise survival in this disease.”
In previously treated advanced melanoma, ipilimumab has been shown to provide durable long-term survival in some patients. Upon its approval in 2011, it became the first treatment to demonstrate an overall survival benefit in a Phase III clinical trial in this patient population. In the pivotal Phase III clinical trial assessing ipilimumab in previously treated patients, published in the New England Journal of Medicine in 2010, 46% (63 people out of 137) of patients were still alive at one year in the ipilimumab arm and 25% (34 people out of 136) in the comparator, a vaccine called gp100.
The median overall survival was 10.1 months among patients receiving ipilimumab alone, compared to 6.4 months among patients receiving gp100 alone.5 In 2013, Bristol-Myers Squibb announced results from a pooled analysis of survival data taken from previously-treated and previously-untreated patients who received ipilimumab at different doses and regimens, including combinations with other agents (n=1,861) [1,6]. When looking at survival over time, a plateau in the survival curve began at approximately 3 years, with a few patients followed for up to 10 years. Approximately 22% of patients (95% CI: (20–24%) were alive at 3 years (number of patients available for analysis at this time point was 254) [1,6]. The analysis was retrospective and did not include a control arm.
Ipilimumab’s use in previously untreated patients with advanced melanoma is supported by data pooled from Phase II and III studies, as well as from two retrospective observational studies in previously-untreated advanced melanoma patients who were treated with ipilimumab 3 mg/kg monotherapy (studies CA184- 332 and CA184-338) [7,8]. Available data have demonstrated that, as monotherapy at this 3 mg/kg dose, treatment with ipilimumab has the potential to improve the overall survival of patients, irrespective of whether they have received prior therapy or not for their metastatic melanoma [1,5,7,8]. The safety profile of ipilimumab in previously-untreated patients is also comparable to that seen in those who have been previously-treated [5,7,8].
Gill Nuttall, Melanoma UK commented: “Advanced melanoma is a devastating diagnosis, yet treatment options remain severely limited for many patients facing this disease. Unless reversed, this decision by NICE will sadly continue this trend and will leave some patients without a medicine that could potentially help extend their lives.”
Commenting on the decision, Amadou Diarra, European Vice-President and General Manager, Bristol-Myers Squibb UK & Ireland, said: “Bristol-Myers Squibb is disappointed in the draft recommendation that NICE has issued and we are committed to doing all we can to ensure that patients ultimately get access to this important treatment as early as possible. Ipilimumab is a pioneering immuno-oncology therapy that has been recognised by European regulators as an important option for advanced melanoma patients who have not received prior therapy. We therefore hope that the significant unmet need and clinical evidence backing this treatment will result in a review of this decision.”
1. Schadendorf D et al. Pooled analysis of long-term survival data from phase II and phase III trials of ipilimumab in metastatic or locally advanced, unresectable melanoma. Abstract # 24LBA, presented at the European Cancer Congress 2013
2. http://guidance.nice.org.uk/TA268 (accessed February 2014)
3. http://info.cancerresearchuk.org/cancerstats/keyfacts/skin-cancer (accessed January 2014)
4- www.medicines.org.uk/EMC/searchresults.aspx?term=yervoy&searchtype=QuickSearch (accessed: January 2014)
5. Hodi FS, O’Day SJ, McDermott DF, et al. Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med. 2010; 10:1-13
7. Patt D, Juday T, Penrod JR et al. A community-based, real-world, study of treatment-naive advanced melanoma (AM) patients treated with 3mg/kg ipilimumab (IPI) in the United States. European Cancer Congress; Amsterdam, Netherlands 27 September–1 October 2013, Poster 3.751
8. Margolin K, et al. Effectiveness and safety of first-line ipilimumab 3 mg/kg therapy for advanced melanoma: Evidence from a U.S. multisite retrospective chart review. ECCO/ESMO 2013 Abstract #3742
9. Larkin J, Gore M. Malignant Melanoma (metastatic). Clinical Evidence 2008; 08; 1718
This second facility gives the company a significant power multiplier for export capacity and geographical reach
News CPhI Discover: Remote excipient audits are on the increase, but we must do more to develop novel excipientsAhead of the session, Value Added Excipients to Unlock the Potential of APIs, at CPhI Discover on Thursday 18th May – global pharma’s largest ever virtual gathering – we spoke with one of the speakers, Dr Iain Moor...
Need clarity on determining your QA from your QbD and your cGMPs? You’re not alone! In our new Pharma Explained series from CPhI, we bring you clear cut definitions from industry experts on a myriad of Pharma Terms, delivered in bitesize video presenta...
News Former FDA Commissioner lays out roadmap for building out excess capacity to deal with pandemic situationsDr Scott Gottlieb shares insights into COVID-19 vaccine development, the upcoming US election and pharma pricing at the CPhI Festival of Pharma
Inspection conducted remotely using video-conferencing and an online private document sharing platform.
Decision clears legal pathway to expand access for patients living with multiple sclerosis in markets across Europe.
The pandemic that has swept over the world in 2020 has caused huge disruption to the European pharmaceutical industry's regulatory procedures and supply chain management. But the crisis has also provided a platform for a learning process for the sy...
The European Medicines Agency's human medicines committee (CHMP) has recommended the suspension of European marketing authorizations of generic medicines tested by Panexcell Clinical Laboratories at its site in Mumbai, India, citing irregularities in h...
Position your company at the heart of the global Pharma industry with a CPhI Online membership
Your products and solutions visible to thousands of visitors within the largest Pharma marketplace
Generate high-quality, engaged leads for your business, all year round
Promote your business as the industry’s thought-leader by hosting your reports, brochures and videos within your profile
Your company’s profile boosted at all participating CPhI events
An easy-to-use platform with a detailed dashboard showing your leads and performance