Frankly, all of these criteria would be unlikely to be met in the area of haemophilia. Marketing exclusivity, while beneficial for the above-mentioned rare disease areas where no or little diagnosis and treatment is available, would be of great
detriment in haemophilia. It could potentially create a monopoly rather than market competition to ensure the widest possible access at the most affordable price. In addition, the potential for better products based on different A-769662 price mechanism’s of action may never be realized. Patients would be deprived of potentially better clinical options for their individual clinical needs. There would be no competition and therefore higher prices – thereby potentially hindering or severely limiting patient access to these products around Europe. Finally, there would be no cascading effect on lowering prices for current recombinant selleck screening library or plasma-derived treatment products or broadening market
access into European countries where patients have limited or severely limited access to treatment products. For the above-mentioned reasons, the EHC has been advocating on these issues for more than 2 years and is supported both by EAHAD as well as WFH. We are aware that the EMA and the Commission are currently considering the ‘similarity’ of these different longer acting products under the orphan drug designation that each of these products has received in Europe. The joint position of the EHC, EAHAD and WFH is that the new longer acting products are not similar and that each protein modification should be treated as distinct and therefore be granted marketing authorization. To
help guide the legal interpretation of ‘similarity’ and how to assess it, the European Commission published a Communication [6] in 2008, which interprets ‘similar active substance’ as one that has ‘the same principal molecular structural features and acting via the same mechanism of action’ and also interprets ‘same mechanism of action’ as meaning all that both products share ‘the same pharmacological target and the same pharmacodynamic effect. The bioengineering strategies used for the manufacturing of the longer acting FVIII and FIX products employ three main and dissimilar approaches. PEGylation, the covalent attachment of PEG polymers to a protein-, peptide- or small molecule drug, is one of the most promising techniques to improve pharmacokinetic and pharmacodynamic properties of therapeutic proteins by increasing their molecular size, making them less susceptible to proteolytic cleavage and degradation and changing their surface charge properties to interfere with receptor-mediated clearance processes [7]. Fc- and albumin fusion consist of the union of an immunoglobulin Fc domain or albumin to recombinant protein through a linker sequence.