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Kan de EMEA nog om Rhucin heen?

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[verwijderd] 10 april 2010 12:12
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European Medicines Agency
7 Westferry Circus, Canary Wharf, London, E14 4HB, UK
Tel. (44-20) 74 18 84 00 Fax (44-20) 74 18 86 68
E-mail: mail@emea.europa.eu www.emea.europa.eu
©EMEA 2007 Reproduction and/or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged
London, 13 December 2007
Doc. Ref. EMEA/567761/2007
QUESTIONS AND ANSWERS ON RECOMMENDATION FOR THE REFUSAL OF THE MARKETING AUTHORISATION
for
RHUCIN
International non-proprietary name (INN): recombinant human C1 inhibitor
On 13 December 2007, the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for the medicinal product Rhucin 150 U/ml powder for solution for injection, intended for the treatment of acute attacks of angioedema in patients with congenital C1 inhibitor activity deficiency. The company that applied for authorisation is Pharming Group N.V. It may request a re-examination of the opinion within 15 days of receipt of notification of this negative opinion.
What is Rhucin?
Rhucin is a powder to be made up into a solution for injection into a vein. It contains the active substance recombinant human C1 inhibitor.
What was Rhucin expected to be used for?
Rhucin was expected to be used to treat sudden attacks of angioedema (swelling of the blood vessels). It was to be used in patients who have low levels of a protein called ‘C1 inhibitor’ due to a congenital (inborn) deficiency.
Rhucin was designated as an orphan medicinal product on 11 May 2001 for treatment of angioedema caused by C1 inhibitor deficiency.
How is Rhucin expected to work?
The active substance in Rhucin, recombinant C1 inhibitor, is very similar to the natural blood protein ‘C1 inhibitor’. C1 inhibitor is normally found in the blood, where it dampens down the activity of the ‘complement system’, which is part of the immune system (the body’s natural defences). Patients with low levels of the active form of C1 inhibitor suffer from attacks of inflammation and swelling that affect various sites in the body. Rhucin was expected to be used as a replacement for the missing C1 inhibitor, correcting the deficiency and reducing the inflammation and swelling that cause angioedema.
The active substance in Rhucin is produced using ‘recombinant DNA technology’: it is extracted from the milk of rabbits that have had a gene (DNA) inserted, which makes them able to produce the human protein in their milk.
What documentation did the company present to support its application to the CHMP?
The effects of Rhucin were first tested in experimental models before being studied in humans.
Rhucin was studied in two main studies involving a total of 48 adults with C1 inhibitor deficiency. In both studies, patients were given Rhucin if they had an attack of angioedema. In total, 17 attacks were treated in a total of 12 patients, but both studies were still ongoing at the time of the medicine’s initial assessment. The main measure of effectiveness was how long it took for the patient’s symptoms to improve. During the evaluation procedure, the company also supplied the initial results of a study comparing the effects of Rhucin with those of placebo (a dummy treatment). This included results from 25 patients.
The CHMP also took safety data from studies on healthy volunteers and patients without symptoms who were treated with Rhucin into account.

Het was in December 2007 en de daar op volgende her aanvraag op een paar belangrijke maar nu achterhaalde puntjes na bijna al gelukt!
Die goedkeuring daar zal men het ook bij de EMEA over eens zijn kan en moet men nu niet omheen willen.
Als er zoals Pharming melde No major concerns zijn dan zal het beoordelen van de gegeven antwoorden ook een fluitje van een cent zijn.
Dus houden we de April meeting goed in de gaten.:-)

Ruud..
Filasoft 11 april 2010 12:56
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Ik denk niet de EMEA zich onder druk laat zetten door dit forum e/o haar leden. En dat is maar goed ook.

MvG,
Philos.
Beursgoeroe 13 april 2010 10:07
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quote:

Filasoft schreef:

Ik denk niet de EMEA zich onder druk laat zetten door dit forum e/o haar leden. En dat is maar goed ook.

MvG,
Philos.
het is alleen maar te hopen, dat de EMEA niet plat is en omgekocht door andere Pharmaci- partijen, die niet willen dat Pharming op de martk komt, duimen maar .............
[verwijderd] 13 april 2010 10:35
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quote:

Fabelr schreef:

[quote=Filasoft]
Ik denk niet de EMEA zich onder druk laat zetten door dit forum e/o haar leden. En dat is maar goed ook.

MvG,
Philos.
[/quote]

het is alleen maar te hopen, dat de EMEA niet plat is en omgekocht door andere Pharmaci- partijen, die niet willen dat Pharming op de martk komt, duimen maar .............
ab
[verwijderd] 13 april 2010 21:28
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quote:

ingrid030 schreef:

[quote=Fabelr]
[quote=Filasoft]
Ik denk niet de EMEA zich onder druk laat zetten door dit forum e/o haar leden. En dat is maar goed ook.

MvG,
Philos.
[/quote]

het is alleen maar te hopen, dat de EMEA niet plat is en omgekocht door andere Pharmaci- partijen, die niet willen dat Pharming op de martk komt, duimen maar .............
[/quote]

ab
laat ons duimen en bidden tot de Heer, dat alles goed gaat komen met Pharming.... Amen ..
[verwijderd] 17 april 2010 00:57
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[verwijderd] 22 april 2010 21:39
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First report on the Agency's Benefit-Risk Methodology Project published
Published 22/04/2010

A summary report on the first work package of the European Medicines Agency's Benefit-Risk Methodology Project – a project aiming to increase the consistency and transparency of the regulatory process through standardised practices for assessing the benefit-risk balance of medicines – has been published.


"Introduction

During the March 2008 plenary meeting, the Committee for Human Medicinal Products (CHMP) adopted the Reflection paper on benefit-risk assessment methods in the context of the evaluation of marketing authorisation applications of medicinal products for human use (EMEA/CHMP/15404/2007). One of the main recommendations for the CHMP was to explore further methodologies for benefit-risk analysis, including a wide range of quantitative and semi-quantitative tools, and involving assessors and experts. A consequence of this was the initiation of the Benefit-Risk Methodology Project in which the five National Competent Authorities of France, The Netherlands, Spain, Sweden and UK volunteered to participate. A project team was formed involving experts in the field of decision theory. The main objective of this project is to further explore methodologies that can improve the current practice of benefit-risk assessment for medicinal products, with an aim to increase the consistency and transparency of the regulatory process."

................

"Results

Between June and September 2009, the project team was invited to visit the five participating
agencies (France, The Netherlands, Spain, Sweden and UK). At a later point, Paul-Ehrlich-Institut
volunteered to participate in the project and was included in the visits in February 2010. In total, 55 people, involved in the benefit-risk decision making, were interviewed within the six agencies.
At present, there are no common definitions on the concepts of benefits and risks and no formal
guidance on the process of their balancing. As a result, interviewees, between agencies and within the same agency, expressed divergent views on the meaning of benefits and risks, and on their weighing. Especially with regard to risks, there was a great variance of responses. Most notably, the uncertainty of realising a benefit from the use of a pharmaceutical product was frequently mentioned as a risk.

In practice, formulating a benefit-risk balance for a medicinal product is an intuitive and implicit process based on expert judgement. In all the six agencies there was no mention of the use of a validated and structured process for assessing benefits and risks. In contrast, most of the interviewees acknowledged the need for a more systematic approach and the value it can add. A few assessors mentioned the use of a self-constructed and simplistic method for balancing benefits and risks. The benefit-risk balance is generally considered as the most difficult part of the assessment process, even for experienced assessors. It was mentioned that the most challenging situations are those in which there is substantial uncertainty about the benefits and the risks of a product, the products belong to a new class of drugs, or the products belong to the therapeutic area of oncology."

www.ema.europa.eu/pdfs/human/brmethod...
[verwijderd] 22 april 2010 22:34
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Ahh Declan, wat moet je hier nu op zeggen. Ik zou je graag gelijk willen geven, maar de uitslag is in handen van de hogere geleerden die niet alleen de werking en de effictiviteit van ruchin moeten bekijken aan de hand van de clinicaltrails en hun uitkomsten. Anderzijds is de puurheid goed genoeg om inschatting te kunnen maken dat het echt safe is zonder nieuwe generaties ziekte te genereren.

Juiste balance tussen winst en risico, waarin het bijwerkingseffect heel veel zwaarte heeft.

Wanneer men ook maar twijfelt aan mogelijke bijwerkingen is het klaar. Dat was de 1e,2e keer van belang en nu dus weer.

Moet niet vergeten dat het gewonnen wordt uit een dierenproduct en dus zeker nu we erg veel last hebben van bijv QKoorts,Vogelgriep etc. Moet er niet aan denken dat we daar ook nog VHS en Myxomatose aan toe kunnen voegen

Arak
[verwijderd] 22 april 2010 23:02
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FDAnews Drug Daily Bulletin
April 22, 2010 | Vol. 7 No. 79

Experts: More Transparency Needed in EMA New Drug Approval Process

The European Medicines Agency (EMA) should increase the transparency of its drug approval process by releasing substantial parts of drug dossiers to the scientific community. Greater transparency would “help independent interested parties define the benefit-risk profile of new medicines before they are allowed onto the market,” according to Silvio Garattini, director of the Mario Negri Institute for Pharmacological Research in Milan, and Vittorio Bertele, head of the institute’s drug regulatory policies laboratory.

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