This web site uses cookies. Do you accept the use of external cookies? Yes No Know more x
Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"
The WODC 2018 was held on the 6-8th November in Barcelona, bringing dozens of pharmaceutical industry professionals and Health Technology assessment agency representatives together to discuss orphan drug development and other subjects around orphan drugs and the patient's role in research, as well as industry-specific subjects, such as financing, and company structures where patients and academia can have bigger roles.

Beyond Achondroplasia was invited to present a poster, presenting achondroplasia, the new BA platform, its relevance within the interested community and the BA survey, made to map the environment around achondroplasia and become a hub for real-world data, aiming to improve access to healthcare and research since access to clinical trials can be crucial for orphan drug approval.


The first day was fully dedicated to workshops on drug pricing, market access, and approval, targeted at pharma companies. The second day opened with the European Reference Networks with the panel session "What are they expected to deliver?". It was explained what the ERNs do, which countries have reference centers and what are the current limitations of these networks, such as sharing high-quality images for radiologic and imaging evaluation by international experts.

Achondroplasia is represented by ERN BOND, the Rare Bone Disorders network, and Inês Alves, creator and director of Beyond Achondroplasia, is one of the current 3 patient representatives in this relevant European initiative.

Afterwards a panel of pharma industry discussed the sustainability of orphan drug development, as well as several other questions related to this development, such as how companies approach the development of drugs for untreated diseases that are poorly characterized.


The rest of the morning was divided into 18 round tables of which we could attend 2 and chose to participate in discussing innovative ways patient groups can contribute to the drug development process and how patient led technological solutions are helping diagnosis, treatment, care, and paving the way for patient-centric medicines development. As an example, in one of the roundtables, participants approached the importance of empowering patients with knowledge on the process of drug development and how patient groups can contribute to this process, by providing an independent, patient-oriented view on clinical trial documentation.


During the afternoon there were 5 rooms with talks and discussion panels around the clock dedicated to:

  • Clinical development;
  • Market Approval and Access;
  • Cell and Gene Therapy;
  • Science and Strategy;
  • Pitch and Partner (focused on industry private and public partnerships).


From these sessions, we highlight "Successfully managing and implementing drug development programs for domestic and international commercialization of orphan drugs". In this session, several solutions and suggestions for problems commonly found in drug development were presented, including the fact that sometimes it's important to not only listen to the principal investigator but also the rest of the staff who contact directly with the patient and can often provide important information, and the implementation of electronically informed consents, which may ease the patient's involvement in the clinical trial.


On the last day, it was time for the PARADIGM project, an ongoing Innovative Medicines Initiative project led by EURORDIS with the purpose of providing concrete evidence on the advantages of engaging patients in the drug development process, both for the patient and the companies developing drugs. During the afternoon, it was time to give the floor to two experimental biotech companies, working with ultra-rare disease treatments that may represent new ways to accelerate drug development. 

Again, WODC Europe paved an important road in opening opportunities for companies to showcase their best practices, share knowledge and work in higher dedicated standards for diverse rare disorders.

A Thank You note to Terrapinn for inviting our collaborator to attend the full event and for the poster space.

To view this site you need Internet Explorer 8 (or a higher version)
or another internet browser.

Thank You.