From AELMHU we publish the quarterly and annual Access to Information Report on the Orphan Drugs in Spain.

Through this project, which has already become a benchmark in the pharmaceutical sector and rare diseases, we seek to analyse the number and timing of approval and funding of these therapies in our country.

The data used for its elaboration are obtained from public sources of the European Union and the Ministry of Health of the Spanish Government, and the methodology used is detailed in each of the reports.

Access

REPORT

2024

PRIMER INFORME CUATRIMESTRAL DE ACCESO A LOS MEDICAMENTOS HUÉRFANOS EN ESPAÑA 2024

La Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos (AELMHU) presenta su primer Informe Cuatrimestral de Acceso de los Medicamentos Huérfanos en España 2024, en el que analiza la situación de los productos con nombre comercial que tienen vigente la designación huérfana europea, a 30 de abril de 2024.

Según los principales resultados del estudio realizado, durante los ocho primeros meses de este año se alcanzó la cifra de 202 productos con nombre comercial y designación huérfana por la Agencia Europea del Medicamento (EMA, por sus siglas en inglés), de los que 149 cuentan con autorización para la comercialización en la Unión Europea.

En términos nacionales, entre enero y abril de 2024, se asignaron 9 nuevos Códigos Nacionales y 9 nuevos medicamentos obtuvieron la designación huérfana, hasta alcanzar los 128 productos huérfanos con Código Nacional, de los cuales 85 ya están financiados (el 66%)

En cómputo global, en lo que llevamos de año han llegado a España un 86% de los medicamentos huérfanos autorizados en la UE, pero solo se encuentra financiado un 57% de los mismos, es decir, 85 productos en total.

Por otro lado, el tiempo de espera entre la obtención del código nacional y su financiación se mantiene en 23 meses en el primer cuatrimestre de 2024.

Actualmente, son 43 los medicamentos huérfanos con Código Nacional sin financiar en nuestro país.

Las fuentes utilizadas para la elaboración de este informe son la Agencia Europea de Medicamentos (EMA), el Registro Comunitario de Medicamentos Huérfanos, la Agencia Española de Medicamentos y Productos Sanitarios (AEMPS), y el Ministerio de Sanidad.

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ANNUAL REPORT 2023 ON ACCESS TO ORPHAN DRUGS IN SPAIN

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) presents the preliminary results of its Annual Report on Access to Orphan Drugs in Spain 2023, in which it analyzes the access situation of trade-name products that have an orphan designation in force as of December 31, 2023.

The first data from this study reveal that during the 2023 financial year there were 199 products with a positive trade name and orphan designation by the European Medicines Agency (EMA), of which 147 have authorization for Community marketing.

At the national level, between January and December 2023, 10 new national codes were assigned, making a total of 123 products with CN and 21 new orphan drugs were approved for financing, bringing the total number of products financed by the National Health System to 78.

Overall, 84% of the orphan drugs authorized in the EU reached Spain last year and 53% of the orphan drugs authorized in Europe were financed. However, 49% of the products were financed with restrictions, i.e. with limitations on the indications or with unfunded indications.

It also improves the waiting time for a financing decision in our country from 34 months in 2022 to 23 months in 2023, i.e. less than two years.

Currently, 45 orphan drugs are still pending funding in Spain, 49% of which have been waiting for three years for a favorable funding resolution, with the oncology and metabolomics therapeutic areas being the main ones without funding.

As for advanced therapies, as in 2022, none have obtained funding from the National Health System (NHS). The sources used to prepare this report are the European Medicines Agency (EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS), and the Ministry of Health.

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Executive summary

2023

SECOND QUARTERLY REPORT ON ACCESS TO HMMH IN SPAIN 2023

The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) presents its second Quarterly Report on Access to Orphan Drugs in Spain 2023, in which it analyzes the access situation of products with orphan designation in force on August 31, 2023 and trade name

According to the main results of the study, during the first eight months of this year, 202 products with a trade name and orphan designation were registered by the European Medicines Agency (EMA), of which 148 are authorized for marketing in the European Union.

In national terms, between May and August 2023, 2 new National Codes were assigned and 9 new orphan drugs were approved for funding, reaching 126 orphan products with a National Code and 77 funded products.

Overall, so far this year 85% of the orphan drugs authorized in the EU have arrived in Spain , but only 52% of them have been financed . In addition, 49% of the products have been financed with restrictions, i.e. with limited indications or with unfunded indications.

On the other hand, the waiting time to reach a price and reimbursement decision in our country remains unchanged at 26 months in both the first four months and the second four months of 2023.

Currently, there are 49 orphan drugs with a National Code that are not financed in our country.

The sources used to prepare this report are the European Medicines Agency (EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS), and the Ministry of Health.

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FIRST QUARTERLY REPORT ON ACCESS TO HMMH IN SPAIN 2023

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) presents a new edition of its Quarterly Report on Orphan Drug Access in Spain 2023, in which it analyzes the access situation of trade-name products with orphan designation in force as of April 30, 2023.

This study reveals that most of the data analyzed have improved compared to the previous year. During the first four months of 2023, 202 products with a trade name and orphan designation were registered, of which 146 have EU marketing authorization, 12 more than during the same period in 2022.

At the national level, between January and April 2023, 5 new national codes were assigned and 7 new orphan drugs were approved for funding, reaching 127 orphan products with a National Code and 70 funded products, 12 more products than during the same period in 2022.

Overall, these data show that during the first four months of this year 87% of the orphan drugs authorized in the EU have reached Spain and 48% of them have been financed, which represents four percentage points more products financed than in the same period of 2022.

It also improves the waiting time to reach a pricing and reimbursement decision in our country, from 32 months at the beginning of 2022 to 26 months in the same period of 2023, which represents a 19% decrease in waiting times over this year.

Currently, 57 orphan drugs are still pending funding in Spain, 3 less than at the end of 2022, and 42% of them have been waiting for three years or more for a favorable funding decision, seven percentage points less than in the same period of 2022. Likewise, 19 drugs with EU marketing authorization have not yet reached Spain, which represents 13% of the innovation authorized at European level.

As for advanced therapies, during the first four months of 2023, one has been authorized in the EU while 3 have received National Code, but none has been funded by the National Health System.

This report has been prepared using public sources: European Medicines Agency, Spanish Agency for Medicines and Health Products (AEMPS) and Ministry of Health.

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ANNUAL REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2023

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) presents the preliminary results of its Annual Report on Access to Orphan Drugs in Spain 2022, in which it analyzes the access situation of trade-name products that have an orphan designation in force as of December 31, 2022.

The first data from this study reveal that during the 2022 financial year, 195 products with a positive trade name and orphan designation by the European Medicines Agency (EMA) were reached, of which 146 have been authorized for marketing in the EU.

At the national level, between January and December 2022, 17 new National Codes were assigned and 9 new orphan drugs were approved for funding, reaching 123 orphan products with a National Code and 63 funded products.

Overall, 84% of the orphan drugs authorized in the EU reached Spain last year, but only 43% of them are financed. In addition, 49% of the products have been financed with restrictions, i.e., with limited indications or with unfunded indications.

On the other hand, the waiting time to reach a price and reimbursement decision in our country has clearly worsened from 24 months in 2021 to 34 months in 2022.

Currently, there are 60 orphan drugs with a National Code that are not financed in our country.

The sources used to prepare this report are the European Medicines Agency (EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS), and the Ministry of Health.

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Executive summary report

2022

SECOND QUARTERLY REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2022

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) presents its Second Quarterly Report on Orphan Drug Access in Spain 2022, in which it analyzes the access situation of trade-name products with orphan designation in force as of August 31, 2022.

This study reveals that during the second quarter of 2022, the number of products with a positive trade name and orphan designation by the European Medicines Agency (EMA) reached 186, of which 137 are authorized for marketing in the EU.

At the national level, between May and August 2022, 6 new National Codes were assigned and 4 new orphan drugs were approved for funding , reaching 116 orphan products with a National Code and 59 funded products.

Overall, so far this year 85% of the orphan drugs authorized in the EU have arrived in Spain, but only 43% of them have been financed . In addition, 47% of the products have been financed with restrictions, i.e. with limited indications or with unfunded indications.

On the other hand, the waiting time to reach a price and reimbursement decision in our country has slightly improved from 32 months in the first quarter to 26 months in the second quarter of 2022.

Currently, there are 57 orphan drugs with a National Code that are not financed in our country.

The sources used to prepare this report are the European Medicines Agency (EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS), and the Ministry of Health.

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FIRST QUARTERLY REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2022

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has presented its First Quarterly Report on Orphan Drug Access in Spain 2022, in which it analyzes the access situation of trade-name products with orphan designation in force as of April 30, 2022.

This study reveals that during the first four months of 2022, the number of products with a positive trade name and orphan designation by the European Medicines Agency (EMA) reached 181, of which 132 have EU marketing authorisation.

At the national level, between January and April 2022, 4 new National Codes were assigned and 2 new orphan drugs were approved for funding, reaching 113 orphan products with a National Code and 58 funded products.

Overall, 86% of orphan medicines authorised in the EU reached Spain in the first four months of this year, but only 44% of them have been funded, which is one percentage point more than in 2021.

On the other hand, the waiting time for a price and reimbursement decision in our country is worsening, from 24 months in 2021 to 32 months at the beginning of 2022, which means a 33% increase in waiting times over this year.

Currently, 55 orphan drugs are still awaiting funding in Spain, 49% of which have been awaiting a favourable funding decision for three years or more . Furthermore, 19 medicines with Community marketing authorisation have not yet reached our country, which represents 14% of the innovation authorised at European level. This report has been prepared using public sources: the European Medicines Agency, the Spanish Agency for Medicines and Health Products (AEMPS) and the Ministry of Health.

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2022

ANNUAL REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2021

The Spanish Association of Orphan and Ultra Orphan Medicines Laboratories (AELMHU) publishes its Annual Report on Access to Orphan Medicines (OMH) in Spain for the year 2021.

This report analyses the status of trade-named products with orphan designation in force as of 31 December 2021.

This study reveals that there are currently 176 products with a trade name and orphan designation by the European Medicines Agency (EMA), of which 129 have marketing authorisation.

At the national level, 17 new National Codes (CN) were assigned during the year and 14 new orphan drugs were approved for funding. This allows us to have 111 orphan products with NCs in our country and 56 products financed by the NHS (43% of the innovation approved at European level).

On the other hand, although the waiting time for a price and reimbursement decision is reduced from 33 months in 2020 to 24 months in 2021, it is still far from the average of 14 months in 2019.

Currently, 55 orphan drugs are pending funding in Spain, 51% of which arrived in Spain more than 3 years ago.

The report has been prepared using public sources: European Medicines Agency (EMA), Spanish Agency for Medicines and Health Products (AEMPS) and Ministry of Health.

Executive summary

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Press release

2021

SECOND FOUR-MONTH REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2021

The Spanish Association of Orphan and Ultra Orphan Medicinal Products Laboratories (AELMHU) has presented its 2nd Quarterly Report on Access to Orphan Medicinal Products (OMPs) in Spain 2021, in which it analyses the access situation of products with trade names that have an orphan designation in force as of 31 August 2021.

This study reveals that during the second four-month period of 2021, 173 products with a positive trade name and orphan designation were registered by the European Medicines Agency (EMA), 126 of which have been granted EU marketing authorisation, thus continuing to boost European pharmaceutical research in the complex and sensitive field of rare diseases.

At the national level, 4 new National Codes (NCs) were allocated between May and August 2021, 43% less than in the first four months of the same year, and 6 new orphan drugs were approved for funding, three times more than between January and May 2021.

Overall, 107 orphan products have CNs in Spain, 48% of the orphan drugs coming from the EU, but only 51 are funded (40% of the innovation approved at the European level).

On the other hand, although the waiting time to reach a price and reimbursement decision has slightly improved from 27.8 months in the first four months to 27.3 months in the first months of 2021, 5 of the 6 MWH financed in this period have taken 2 years or more to obtain price and reimbursement.

Currently, 56 orphan drugs are still pending funding in Spain, some of them since 2011.

The report has been prepared using public sources: European Medicines Agency (EMA), Spanish Agency for Medicines and Health Products (AEMPS) and Ministry of Health.

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FIRST FOUR-MONTHLY REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2021

The Spanish Association of Orphan and Ultra-Orphan Medicinal Products Laboratories (AELMHU) has presented the first four-monthly report on access to orphan medicinal products (OMPs) in Spain, in which it analyses the access situation of products with trade names that have an orphan designation in force as of 30 April 2021.

This study reveals that during the first four months of 2021, the number of products with a positive trade name and orphan designation by the European Medicines Agency (EMA) reached 168, of which 121 have authorisation for EU marketing, thus maintaining the commitment to pharmaceutical research in such a complex and sensitive area as the European Union.

At the national level, between January and April 2021, 7 new National Codes (NCs) were assigned and 2 new GMHs were approved for funding, bringing the total number of orphan products with NCs to 105 and the number of funded GMHs to 47.

In global terms, Spain authorises 87% of the HMM coming from the EU but only finances 39% of the HMM. This is one percentage point less of products financed compared to the global data for 2020.

On the other hand, the waiting time to obtain a price and reimbursement decision in our country has slightly improved from 33 months in 2020 to 28 months today, although the 2 products financed in this four-month period had to wait two or more years to obtain a positive price decision.

At present, 58 HRM are still pending funding in Spain, of which 55% have been waiting for more than three years for a positive funding resolution.

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ANNUAL REPORT ON ACCESS TO ORPHAN DRUGS IN SPAIN 2020

According to the Annual Report on Access to Orphan Medicinal Products in Spain prepared by AELMHU, during 2020 a record 165 products with a trade name and positive orphan designation by the European Medicines Agency (EMA) was reached, which is evidence of the great moment that pharmaceutical research is experiencing in its commitment to a field as complex and sensitive as the EERRs.

But while the European Union (EU) reached its best figures in 2020, with 22 new products with positive orphan designation and 18 new EU marketing authorisations, in Spain the different parameters of the marketing and financing process of MMHH show the worst data of the last five years.

In fact, in the last financial year, 10 National Codes (NCs) were assigned in our country and 5 new medicines were approved for funding, which represents a 6% decrease in NCs and 45% fewer medicines funded compared to 2019. Beyond the percentage, these figures reveal that, in 2020, only 4 out of every 10 NHMs in Europe will finally reach Spanish patients.

Report

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Press release

2020

Second FOUR-MONTH report 2020:
data on access to orphan drugs in Spain

A four-monthly study prepared by the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) that analyses the approval and marketing processes for orphan drugs in Spain until 31 August 2020.

Spain continues to be at a clear disadvantage with respect to the availability of orphan drugs compared to the European Union. While the EU had 108 medicines available at the end of August, in Spain the figure is drastically reduced to 52 (48%).

The data presented in the Bulletin also show that there are another 44 orphan drugs authorised in Spain that are not yet available to patients because they are awaiting the National Health System to indicate the reimbursement price. A bureaucratic process that, as a result of the COVID-19 pandemic, is taking even longer.

In fact, since January 2020, only two new orphan drugs are available, the price of which was approved at the meeting of the Interministerial Commission on the Pricing of Medicines and Medical Devices on 4 March 2020. These medicines had obtained the National Code in 2018 and 2019, respectively.

Currently, 100% of the orphan drugs that have obtained their National Code in 2020 are still awaiting pricing for marketing.

Report

First FOUR-MONTH report 2020: access data for orphan drugs in Spain

A four-monthly analysis prepared by AELMHU to determine the number of orphan drugs marketed in Spain and the approval timelines until 30 April 2020.

Report

2019

Second FOUR-MONTH report 2019:
data on access to orphan drugs in Spain

First FOUR-MONTH report 2019:
data on access to orphan drugs in Spain

2018

Analysis of the evolution of access to orphan drugs in Spain
2018

Jorge Mestre-Ferrándiz, Margarita Iniesta, Marta Trapero-Bertran, Jaime Espín, Max Brosa.

Article

2017

AELMHU Report 2017:
Current situation on accessibility to orphan drugs in Spain

Margarita Iniesta, Naciba Zetchi, Noemí López, Maribel Cuesta, Max Brosa.

Poster presented at the XXXVII Jornadas de Economía de la Salud (XXXVII Health Economics Conference)

Poster