02 December 2016

7th World Orphan Drug Congress Europe, Brussels, 15th – 17th November 2016

Report by Maureen Mason for Retina International

The European Orphan Drug Congress was attended by Patient Advocacy groups, Government organisations and payers as well as Clinical Research organisations, Industry (Pharmaceutical & Biotechnology) and other interested parties over three days in Brussels. The congress covered crucial topics affecting the whole sector and of particular interest to RI including, finding ways to overcome road blocks in clinical developments, exploring the different regulatory procedures and understanding Health Technology Assessment (HTA) access developments which are changing how orphan drugs are researched, manufactured and distributed. A focus was placed on Rare Cancers, Gene Therapies as well as Science & Strategy.

On Tuesday 15th of November Pre-Congress Workshops were held on

Pricing, reimbursement and market access as they present major hurdles in the EU health systems by delaying patient access to Orphan Drugs (ODs) approved by the European Medicines Agency, EMA. The real-world experience was also presented and potential solutions discussed to address key pricing, reimbursement and market access challenges for Advanced Therapy Medicinal Products in the EU context.

Advanced Therapy Medicinal Products (ATMPs) are medicines for human use that are cell based therapies, gene therapies and hybrid cell-gene therapies. Expanded access and compassionate use programs were discussed along with the benefits of registries and clinical study types. Also discussed were the recent developments in HTA and Reimbursement Decision-Making Considerations for Orphan Drugs in Europe and the emerging global regulatory pathway for treatments.

The following two days commenced with keynote plenary sessions both opened by Dr Ségoléne Aymé of INSERM. Presentations focused on:

  • The benefits of the European Referenceetworks (ERN) to promote clinical excellence by strengthening and facilitating research.


  • Assessment of value for money of orphan medicines was a key topic with suggestions put forward including the need for European consistency by creating more effective and sustainable methods/models adapted specifically to rare diseases. The need to achieve affordability of orphan medicines and sustainability of the orphan innovative industry. Key to understanding this is transparency and consistency. This could potentially be shown by making public the cost of Research & Development.. It was agreed that collaboration between all stakeholders, including research-based industry, payers, clinicians and patients is vital to this end.


  • There was an update from the EMA on what the current debate in the EU tells us about the guidelines regarding ‘significant benefit’ (SB) on orphan medicines and an update on PRIME which is a scheme launched by the EMA to enhance support for the development of medicines that target an unmet medical need


  • What needs to change and what strategy is needed to shape EU rare disease policies was also discussed and it was suggested that any research into rare diseases needs to have international cooperation.


  • The idea that all new medicines become orphan drugs in the era of personalised healthcare was also presented and discussed.

As a patient representative in attendance, there were many opportunities to participate in round table discussions on Clinical Development, Patient Access, Gene Therapy and Science & Strategy and most contained a good mix of stakeholders.

There was an acceptance by those in attendance that while patient orientated discussions had increased, progress was slow and improvements were required to bring about meaningful engagement. Many agreed that the patient “voice” needs to be involved from the beginning of any research/innovation process and through the lifecycle of drug development, a point advocated strongly by RI.  There was also discussion about patient organisations being “more professional” and how the European Patient Academy, EUPATI, has been successful in helping patient advocates engage with stakeholder at a more “respectful” level. The funding challenges that prevent patient organisations in participating in important discussions was mentioned by many of the patient representatives present.

Another interesting discussion concerned the importance of educating ‘society” at large as well as patients on the actual costs involved in drug development and issues around market access/reimbursement.

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