Only 52% of orphan drugs authorised by the EMA are marketed in Spain.

Only 52% of orphan drugs authorised by the EMA are marketed in Spain.

The AELMHU study reveals that, of the 94 orphan drugs approved by the EMA between 2002 and 2016 with current orphan drug designation, only 49 were marketed in Spain.

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has presented, within the framework of the XXXVII Conference of the Association of Health Economics (AES), updated data up to 2016 regarding the situation of access to orphan drugs (OAM) in Spain.

The AELMHU study Current situation on the accessibility of orphan medicinal products in Spain shows that between 2002 and 2016, the European Medicines Agency (EMA) authorised 94 medicines with orphan designation still in force, for which the Spanish Agency for Medicines and Health Products (AEMPS) granted 77 national codes (CN), with only 49 orphan medicines being marketed. The new data also show that the average time between EMA authorisation and marketing was 19.2 months, and 12.6 months from obtaining the CN-AEMPS to marketing.

A comparison of the number of orphan medicines authorised with P&R in Spain versus those authorised by the EMA in two specific periods, 2002-2011 and 2012-2016, shows that the number of orphan medicines authorised with P&R in Spain fell from 90.5% to 31%. In the analysis of the same periods, the number of orphan medicinal products that were still under price and reimbursement (P&R) negotiation increased fourfold, from 9.5% to 39.7%.

Authorised HMMH in Europe / Spain in the periods 2002-2011 and 2012-2016

 Period 2002-2011Period 2012-2016
EMA-authorised MMHH with current orphan designation4258
MMHH authorised with P&R in Spain38 (90,5%)18 (31%)
MMHH that have not applied for NC in Spain017(29,3%)
MMHH pending P&R4 (9,5%)23** (39,7%)

* Includes MMHH who have lost their orphan designation at present

**For 11 (48%) of these MMHH have been denied funding in the NHS according to the IPT17 available as of 31/7/2017.

Comparison with European countries
According to data presented by AELMHU, Spain markets 52% fewer orphan drugs than the European Union.

For the study, 16 non-oncology orphan medicines that were not marketed in Spain in 2016 were identified and analysed. On average, 14 months elapsed from authorisation to obtaining the CN. Of these 16 orphan drugs, 12 of them (75%) are treatments for long-term chronic diseases. In this context, considering 15 of the 16 orphan drugs analysed, which represent new molecules, it is estimated that in total there would be a maximum of 4,300 patients who are candidates for one of these treatments. Of the 16 orphan drugs analysed, the UK marketed 13, Italy 6 and France 8 orphan drugs.

Finally, the economic impact of the introduction of 13 of these 16 non-oncological non-commercialised non-oncological MMHHs in Spain was evaluated, and it was found that they would represent 0.07% of the Total Public Health Expenditure and 0.75% of the Hospital Pharmaceutical Expenditure in the first year after their commercialisation.

Budgetary impact
Currently, gaining market access for orphan drugs in Spain is no easy task. Despite having been approved by the European Medicines Agency (EMA), the subsequent negotiation of funding and price in Spain prevents patients affected by rare diseases from having equal access to these new drugs.
On the other hand, and as reflected in the economic impact analysis mentioned above, although the individual cost per patient of orphan drugs can be high, the overall cost associated with them is very low due to the low prevalence of rare diseases, resulting in a low impact on the health budget.

At the beginning of 2016, AELMHU conducted a study on access to orphan drugs in Spain, in comparison with the most relevant countries in Europe, which revealed a significant decrease in the number of orphan drugs that have obtained funding and price in the last four years, and which revealed a change in trend that distances Spain from the ratios identified for other European countries, with respect to effective access to these therapeutic innovations. Specifically, the data revealed that, of the 94 new orphan drugs approved by the EMA in the period 2002-2015, with orphan drug designation in force, only 51 were marketed in Spain, just over half.

The study also analysed the situation from drug approval by the EMA to the funding and pricing decision, to determine marketing and effective access to the new medicine. The analysis excluded cancer treatments and assessed, among other parameters, the impact of orphan drugs in terms of prevalence and budgetary impact, comparing the situation in Spain to that of the main European countries (UK, Italy and France). Germany is not included in the analysis as it has a different financing and marketing model. The references noted in this analysis have been reconfirmed in the updated data as of 31 December 2016 now presented by AELMHU.