Added benefit of fampridine is not proven

August 29, 2012

Fampridine (trade name Fampyra®) has been approved in Germany since July 2011 for adult patients suffering from a higher grade walking disability (grades 4 to 7 on the EDSS disability status scale), as a result of multiple sclerosis (MS). The German Institute for Quality and Efficiency in Health Care (IQWiG) has assessed the added benefit of the drug pursuant to the Act on the Reform of the Market for Medicinal Products (AMNOG). According to the findings, there is no proof of added benefit, as the manufacturer's dossier contains no evaluable study data for the comparison between fampridine and the appropriate comparator therapy.

G-BA specifies physiotherapy as the appropriate comparator therapy

MS is a chronic incurable inflammatory disease, in which the patient's own immune system damages nerve tracts in the brain and spinal cord. In some patients, some muscles are in permanent spasm or are paralysed. If the disease is more advanced, patients may develop a walking disability.

The Federal Joint Committee (G-BA) has specified physiotherapy as the appropriate comparator therapy for the benefit assessment. This treatment must fulfil the requirements of the German Guideline on Remedies (Heilmittelrichtlinie). In addition, the patients must receive optimized standard therapy for MS.

Requirements for an indirect comparison not fulfilled

There are no studies that directly compare fampridine with physiotherapy. Instead, the pharmaceutical company presented data on an indirect comparison. These data originate from studies in which fampridine was compared with a placebo or in which physiotherapy was compared with "no treatment".

The legal ordinance on AMNOG explicitly specifies that it is possible to prove added benefit using indirect comparisons too. However, specific methodological conditions apply, which were not fulfilled by the manufacturer in the fampridine dossier.

Marked differences in the grade of disability

In addition, the studies on physiotherapy which the pharmaceutical company has evaluated cannot be used, as they also included patients with a markedly lower grade of disability (EDSS from 1.5) than in the studies on fampridine. Thus, the populations were not similar enough to allow a comparison between the results.

Finally, the manufacturer does not discuss in the dossier whether the physiotherapy tested in these studies was in accordance with the criteria of the Guideline on Remedies and, if this is not the case, why these studies would nevertheless allow conclusions about the situation in Germany. Moreover, the manufacturer does not mention whether the patients actually received optimized MS standard therapy. However, these two points are conditions specified by the G-BA for the appropriate comparator therapy.

Hence the manufacturer did not present evaluable studies on the appropriate comparator therapy for the assessment of the added benefit of fampridine, thereby also failing to present an evaluable indirect comparison. Thus there is no proof of added benefit of fampridine.

G-BA decides on the extent of added benefit.

The dossier assessment is part of the overall procedure for early benefit assessment conducted by the G-BA. After publication of the manufacturer's dossier and its assessment by IQWiG, the G-BA initiates a formal commenting procedure which provides further information and can 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.
-end-
An overview of the results of the benefit assessment by IQWiG is given by the following extract. You can also find easily understandable and brief German-language information about fampridine on the website gesundheitsinformation.de, published by IQWiG.

The G-BA website contains both general English-language information about the procedure of benefit assessment pursuant to §35a Social Code Book V and specific German-language information on the assessment of fampridine.

Institute for Quality and Efficiency in Health Care

Related Disability Articles from Brightsurf:

Raising the bar on disability care
Encouraging paid workers to employ the 'right kind' of respectful personal relationship with young people with disability will lift standards in the sector, experts say.

Keep moving to prevent major mobility disability
According to research, being physically inactive is the strongest risk factor for disability as we age.

How gene mutation causes autism and intellectual disability
Scientists have discovered why a specific genetic mutation causes intellectual disability and autism spectrum disorder in children.

Is disability a risk factor for miscarriage?
A new study compared the proportion of women with any cognitive, physical, or independent living disability who experienced a miscarriage during the previous 5-year period to women without disabilities.

'Climate change is a disability rights issue'
In a high-profile Letter in Science, University of Konstanz climate scientist and ecologist Dr Aleksandra Kosanic, an Associate Fellow of the University of Konstanz's Zukunftskolleg, draws attention to the fact that disabled populations have, until now, been absent from international conversations about climate change and its impact.

Predicting frailty, disability and death
In a study led by investigators from Brigham and Women's Hospital, researchers analyzed patterns of movement among elderly study participants and found that irregular, spontaneous fluctuations could predict a person's risk of frailty, disability and death years later.

Movement patterns predict frailty and disability in the elderly
Elderly people who show more random changes in daily movement tend to be at greater risk of frailty, disability and death, according to a large study involving 1,275 individuals over the course of 13 years.

IQSEC1 gene mutations cause new intellectual disability syndrome
Researchers identify gene causing intellectual disability syndrome that is common in countries where consanguineous marriages are prevalent.

Best medications to reduce drooling for those with developmental disability
A new study has revealed the most effective medications to reduce drooling in young people with a developmental disability, which can affect their socialisation, relationships and community life.

Obesity worsens disability in multiple sclerosis
Obesity is an aggravating factor in relapsing-remitting multiple sclerosis, the most common form of the disease.

Read More: Disability News and Disability Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.