Featured Research

from universities, journals, and other organizations

Vismodegib in basal cell carcinoma: Added benefit not proven

Date:
November 18, 2013
Source:
Institute for Quality and Efficiency in Health Care
Summary:
No added benefit was found with the use of the drug Vismodegib versus the appropriate comparator therapy in a study focused on basal cell carcinoma.

The drug vismodegib (trade name: Erivedge) is approved for the treatment of patients with two forms of basal cell carcinoma (BCC): symptomatic metastatic BCC and locally advanced BCC inappropriate for surgery or radiotherapy.

In an early benefit assessment pursuant to the Act on the Reform of the Market for Medicinal Products (AMNOG), the German Institute for Quality and Efficiency in Health Care (IQWiG) examined whether this new drug offers an added benefit over the current standard therapy. However, no added benefit can be derived from the data presented in the company's dossier.

Currently hardly any chances of cure in advanced basal cell carcinoma

Basal cell carcinoma (also called "basalioma") is a malignant form of skin cancer, which mainly occurs on the head or neck. In most cases, timely surgery can prevent it from spreading. In an aggressive ("locally advanced") form and the occurrence of metastases, however, treatment options are limited. Often only symptoms can be relieved, but cure is no longer possible.

The manufacturer has now presented data from an approval study. According to this study, tumours shrank under vismodegib in some patients with symptomatic metastatic or locally advanced BCC.

G-BA distinguished different comparator therapies

The Federal Joint Committee (G-BA) specified several appropriate comparator therapies: In patients with symptomatic metastatic BCC for whom surgery was not an option, the drug was to be compared with radiotherapy. In patients with symptomatic metastatic BCC for whom radiotherapy was inappropriate, surgery was to be used as comparator therapy. In patients with symptomatic metastatic or locally advanced BCC for whom neither radiotherapy nor surgery was an option, the benefit of vismodegib was to be compared with the benefit of best supportive care. "Best supportive care" means palliative treatment tailored to the individual patient, i.e. a treatment that does not aim to cure, but to alleviate symptoms as good as possible (e.g. with adequate pain therapy) and improve quality of life.

Pharmaceutical company deviated from the G-BA's specification

In contrast to the G-BA's specifications, the pharmaceutical company exclusively compared its drug with best supportive care -- claiming that surgery and radiotherapy in symptomatic metastatic BCC also had only a palliative effect. IQWiG did not accept this deviation from the specified appropriate comparator therapy: If it is assumed that different treatments are optimal for different patient groups, this differentiation also has to have an effect on the control groups.

Studies unsuitable for assessing the added benefit

However, the main problem was that the dossier was based solely on studies without control groups. An added benefit can only be derived from this kind of studies if "dramatic" patient-relevant effects occur in these studies. To estimate the effect size, other studies would have to be used for comparison -- in this case, studies known as "historical controls." If, for example, most patients died early in the historical controls, but many patients survived longer in the study with the new drug, this would be a "dramatic effect."

Based on the study data submitted, it was not possible to assess whether there were dramatic effects with regards to patient-relevant outcomes because tumour regression mainly measured with imaging techniques (the so-called "objective response rate") is a surrogate outcome and not necessarily patient-relevant. Moreover, the pharmaceutical company did not present any data on the appropriate comparator therapy so that no comparison could be made. Hence an added benefit versus the appropriate comparator therapy is not proven.

G-BA decides on the extent of added benefit

The dossier assessment is part of the overall procedure for early benefit assessments supervised by the G-BA. After publication of the manufacturer's dossier and IQWiG's assessment, the G-BA conducts a commenting procedure, which may provide further information and result in a change to the benefit assessment. The G- BA then decides on the extent of the added benefit, thus completing the early benefit assessment.


Story Source:

The above story is based on materials provided by Institute for Quality and Efficiency in Health Care. Note: Materials may be edited for content and length.


Cite This Page:

Institute for Quality and Efficiency in Health Care. "Vismodegib in basal cell carcinoma: Added benefit not proven." ScienceDaily. ScienceDaily, 18 November 2013. <www.sciencedaily.com/releases/2013/11/131118102632.htm>.
Institute for Quality and Efficiency in Health Care. (2013, November 18). Vismodegib in basal cell carcinoma: Added benefit not proven. ScienceDaily. Retrieved September 16, 2014 from www.sciencedaily.com/releases/2013/11/131118102632.htm
Institute for Quality and Efficiency in Health Care. "Vismodegib in basal cell carcinoma: Added benefit not proven." ScienceDaily. www.sciencedaily.com/releases/2013/11/131118102632.htm (accessed September 16, 2014).

Share This



More Health & Medicine News

Tuesday, September 16, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

EU Ministers and Experts Meet to Discuss Ebola Reponse

EU Ministers and Experts Meet to Discuss Ebola Reponse

AFP (Sep. 15, 2014) The European Commission met on Monday to coordinate aid that the EU can offer to African countries affected by the Ebola outbreak. Duration: 00:58 Video provided by AFP
Powered by NewsLook.com
Despite The Risks, Antibiotics Still Overprescribed For Kids

Despite The Risks, Antibiotics Still Overprescribed For Kids

Newsy (Sep. 15, 2014) A new study finds children are prescribed antibiotics twice as often as is necessary. Video provided by Newsy
Powered by NewsLook.com
FDA Eyes Skin Shocks Used at Mass. School

FDA Eyes Skin Shocks Used at Mass. School

AP (Sep. 15, 2014) The FDA is considering whether to ban devices used by the Judge Rotenberg Educational Center in Canton, Massachusetts, the only place in the country known to use electrical skin shocks as aversive conditioning for aggressive patients. (Sept. 15) Video provided by AP
Powered by NewsLook.com
Respiratory Virus Spreads To Northeast, Now In 21 States

Respiratory Virus Spreads To Northeast, Now In 21 States

Newsy (Sep. 14, 2014) The respiratory virus Enterovirus D68, which targets children, has spread from the Midwest to 21 states. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins