Is REvalMed facilitating or hindering orphan drug access in Spain? A report by the AELMHU


The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has released a report detailing the conclusions of an investigation into the Therapeutic Positioning Report (IPT) process in Spain. Analysis of all IPTs from 2013 until those published in May 2022 has shown that 3/10 therapies being investigated in Spain are in orphan indications, and growth of 137.5% from 2013 to 2018 shows this is a trend that is continuing to develop. Despite this, many orphan drug IPTs currently remain unpublished, limiting access to therapies in orphan indications.

The report describes areas where it believes REvalMed is currently underperforming and issues several calls to action for both the Spanish Agency of Medicines and Medical Devices (AEMPS) and REvalMED, with the aim of driving change in the future. Jake James investigates this further.

The number of therapies seeking orphan status is increasing

In recent years the number of drugs seeking approval in orphan indications has risen significantly. According to the report, 3/10 medicines being evaluated in Spain through the IPT process are orphan drugs. As such, the number of IPTs being published for orphan drugs has risen 137.5% from 2013 to 2018.

Over 50% of orphan therapies undergoing evaluation in Spain can be split into three therapy areas:

  • 30% focus on oncology indications
  • 21% focus on metabolic diseases
  • 10% are immunomodulatory drugs

Current access to orphan therapies in Spain

The report describes access to innovative drugs in Spain as slow and unpredictable. 40% of all IPTs written for orphan drugs between 2013 and May 2022 have not yet been published, and of those that have been, an average time to publication of 414 days has significantly slowed access to potentially disease-modifying treatments.

 There are around 6000 currently identified orphan diseases, and the thousands of patients seeking a therapy for their disease are currently being met with a lengthy barrier to access.

Current targets are not being met

When REvalMed was introduced in November 2020, it launched with the intention of sending IPTs to the common portfolio within 97 days of the identification of candidates by the co-ordinating group. In practice, this target is currently not being met.

Of the 7 IPTs that REvalMed has written for orphan drugs, an additional 266 days was needed on average to pass the report to the common portfolio. Additionally, the report details other areas that they consider REvalMed is underperforming in:

  • The prioritisation criteria, which aims to produce IPT reports for therapies with the most need, is not clear
  • A lack of transparency in follow-up and co-ordination, since the ‘control panel’ announced by the Ministry of Health has not been made public
  • They consider the work of the co-ordinating group is not transparent, and does not consider the opinions of all stakeholders involved


The report concludes that the extensive evaluation of orphan drugs in Spain is delaying access to patients. Despite the exhaustive analysis at the European level through the EMA, there is still a lengthy re-evaluation at the national level, with a reluctance to accept incomplete data packages, and is ultimately a barrier to the access of potentially life-changing treatments for those suffering with rare diseases, or conditions with no current treatment options.

Regarding REvalMed, the report’s analysis has revealed that, currently, the Spanish healthcare system is experiencing no greater access to rare disease therapies through REvalMed than it did prior to its inception. AELMHU has issued a call to action for the AEMPS to allocate both extra human and financial resources to REvalMed, to ensure that it can successfully meet the objectives that it set out to achieve.

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AEMPS: Spanish Agency of Drugs and Drug Devices; AELMHU: Spanish Association of Orphan and Ultra-Orphan Drug Laboratories; EMA: European Medicines Agency; IPT: Therapeutic Positioning Report


Article published 6 July 2022.