Other News
Orphan Medicines: almost 20 years on
The EU orphan medicine scheme has been in place since 2000, with the principle aim to encourage companies to develop medicines for rare diseases. According to EU statistics, there are over 6,000 rare diseases and approximately 30 million people sufferers. Due to the lack of readily available therapies, treating these patients has always presented a challenge, and thus a huge unmet need had arisen. To date, the EMA has given over 1900 products orphan designation and, according to their database, over 140 of these products are now available on the market - providing treatment options for patients who previously often had none.
According to the EMA, a rare disease is one where fewer than 5 in 10,000 people have the condition. The orphan medicine scheme offers incentives for the research and development of these medicines, to help off-set the huge costs involved in their development. These incentives are based on certain criteria being met, such as no previous existing treatment, or if the proposed new treatment is of significant additional benefit to the patient. The incentives for developers can be fee reductions, access to scientific advice and 10-year market exclusivity if the orphan designation is maintained.
The EMA has also recently announced that it will be publishing orphan maintenance assessment reports for all products that have been granted a marketing authorisation by the agency. The aim of this report is to provide a summary on whether a medicine still fulfills its designation at the time of authorisation. This new steps helps to address the concerns raised by some stakeholders that some drugs may not still meet the criteria at the time of authorisation.
What is clear is that the EMA is still striving to bring orphan medicines to patients, and is constantly looking at ways to streamline the process for designation and approval. Here at ELC Group we have a strong track record in helping clients with their development programs for orphan medicines, as well as scientific advice meetings and regulatory strategy. As always, we are on hand to help you with any of your regulatory needs.
Also see our
press coverage
News Index
05 Oct 2018
EU/US MRA - Brief overview and update
21 Aug 2018
Brexit - has the music stopped?
13 Jul 2018
15 Jun 2018
08 May 2018
MHRA sets its post-Brexit strategic direction
12 Apr 2018
20 Mar 2018
Clinical Trials: all change 2019
22 Jan 2018
Orphan Medicines: almost 20 years on
15 Dec 2017
05 Oct 2017
How recent moves by the FDA spell good news for generics in the US
11 Sep 2017
Medicines for the Elderly – is your drug development strategy up to the mark?
14 Aug 2017
The MNAT initiative – what’s new?
19 Jul 2017
ICH Q12: Light at the end of the tunnel…?
05 Jun 2017
Q3D: Are your established products in compliance?
02 Jun 2017
Key pharmacovigilance trends to watch
08 May 2017
10 Apr 2017
New EMA Consultation on the Quality of Inhaled Pharmaceutical Products
16 Mar 2017
European Parliament’s adoption of ‘Options for improving access to medicines’ procedure
16 Feb 2017
Article 58: a hidden potential
24 Jan 2017
Global lifesciences partner ELC Group teams with PSR Group for advanced drug development
19 Jan 2017
A positive step forward for Biosimilar registration
20 Dec 2016
Falsified Medicines Directive – Are you making the right preparations?
21 Nov 2016
New Public consultation launched by the European Commission (EC)
19 Oct 2016
Transatlantic collaboration to boost research and development of medicines for rare diseases
03 Jun 2016
ELC Group to showcase regulatory affairs solutions at CPhI events in Shanghai and Barcelona
16 Jun 2015
ELC Group in early-stage development of novel influenza vaccine
09 Jun 2015
ELC Group expands into pharmaceutical product development market
21 Apr 2015
ELC Group signs 1.5 million Euro regulatory affairs contract with Chinese pharma giant
24 Feb 2015
02 Feb 2015
ELC GROUP to present at “Regulatory & Legal Affairs in Russia 2015” Conference
15 Sep 2014
20 Aug 2014
ELC Group Appoints Dr. Siddharth Chachad as Head of Global Clinical Development and Medical Affairs
28 Jul 2014
ELC Group Appoints Head of Regulatory Affairs for Russia and CIS
14 Apr 2014
10 Oct 2013
17 Sep 2013
Global regulatory affairs specialist ELC Group appoints former MHRA Expert Medical Assessor
23 Apr 2013
27 Feb 2013
Global regulatory affairs specialist ELC Group to deliver pharmaceutical training event in Latvia
20 Feb 2013
Global pharmaceutical regulatory affairs specialist ELC Group launches Clinical Trials services
10 Jan 2013
31 Dec 2012
07 Nov 2012
Global pharmaceutical regulatory affairs specialist ELC Group establishes new office in Singapore
22 Aug 2012
12 Jun 2012
XEVMPD software provider Pharma eReport launches Excel Data Import Service
06 Jun 2012
28 Mar 2012
ELC Group joins DIA EuroMeeting in Copenhagen
21 Mar 2012
08 Mar 2012
ELC Group Hosts Successful International Regulatory Seminar
06 Mar 2012
Pharmaceutical regulatory affairs specialist ELC Group opens new office in Mumbai
23 Feb 2012
Regulatory challenges for 2012 and their impact on marketing authorisation
21 Feb 2012
08 Feb 2012
Pharmaceutical regulatory affairs specialist ELC Group establishes global HQ in Cambridge, UK
07 Dec 2011
27 Sep 2011
12 Jul 2011
ELC Group aim to foster strategic and economic ties within India and China
25 May 2011
ELC Group announces doubled revenues
11 May 2011