Download: Optimising Clinical Development & Speeding Patient Access to Orphan Drugs

In Market Access, Partnering & Investment by Simon Reid1 Comment

Extract the Potential

In preparation for the 2014 World Orphan Drug Congress in Geneva, we have collated a number of interviews from some of the leading orphan drug developers. The industry-at-large are investing more and more resources into rare disease research, and numerous companies are now deploying the scientific, regulatory and strategic expertise to progressed an orphan drug to market.

With over 7000 known rare diseases, the opportunities both in a healthcare and commercial context are tremendous. This eBook holds key insight and top tips about how to overcome development challenges, how to establish fruitful strategic partnerships, what needs to be displayed to payers to demonstrate value and what the foreseeable game changers and opportunities may be that will affect the future of the industry. The feedback was assembled following several interviews conducted with confirmed speakers for the World Orphan Drug Congress Europe 2012.

Download the Top Tips here in our latest eBook here

The speaker faculty for the 4th Annual World Orphan Drug Congress is being finalised this month and so far there has been unprecedented interest and endorsement from the global biopharma and rare disease industry. Take a look at the sponsoring organisations here and book today to get involved with the leading figures of the orphan drug industry, expand your rare disease network and share ideas to create new solutions to meet your business objectives in this space.

For more updates on Pharma, biotech and R&D, follow us on Twitter: @biopharmaevents or join our LinkedIn group: BioPharma- Networking for Pharmaceuticals, Biotech and R&D


  1. Agim Vatovci

    Muscular Dystrophy Association of Kosova

    Was formed in 1977, from physiatrist Dr.Mihajlo Samargjiski who took the initiative for this Association, and Mr.Shaban Xheladini the Secretary of the Association. In the beginning it was very hard to find fitting members for this Association. So far our Association has 530 recorded members, from which:

    1 – Albanian 417
    2 – Serbs 78
    3 – Montenegrins 10
    4 – Muslims 20
    5 – Roma 5

    Total: 530

    If we were to compare the current data about the condition of the persons with Muscular Dystrophy in Kosova with those of the last 20 years, we would notice that their economical, social, and health condition is very poor – all this resulting as a consequence of the post-war state.
    In the future we will continue to advice, suggest and give help to persons with Muscular Dystrophy in Kosovo without hesitation. While in Kosova there are many places for employment of people with Muscular Dystrophy, in our society there exists a prejudice that they cannot do these kinds of jobs.
    The Association of Persons with Muscular Dystrophy in Kosova residents of Prishtina, “Pal Lucaj” street, No. 40, needs financial help to begin a large campaign in order to change the current opinion of Kosovars about these people and together surpass this kind of discrimination. This by means of:
    a) Informing the public by media about those kinds of workplaces where people with Muscular Dystrophy can be employed without the risk of showing inefficiency in their job.
    b) Contacting with written and electronic media, arranging conferences for press, seminars and tribunes, with the purpose of persuading journalists to write more on the need of eliminating prejudices in the Kosovar opinion resulting with a greater need for integration of persons with a limited abilities. This awareness of opinion can be achieved only through a massive long campaign.
    c) Making Medial pressure to local administrative institutions and international mechanisms so they seriously consider the problem and foresee obligated quote for employment of persons with Muscular Dystrophy into their legislation and mechanisms in Kosova.
    In its daily activities the Association is facing a great hindrance in the process of registering the new members. In Kosova’s society there exists an immense prejudice about people with Muscular Dystrophy, remarkably in rural communities, regarding their abilities, incorporation, devotion and diseases in society.
    Their families refuse to accept that they have a person with Muscular Dystrophy or with limited abilities inside their families, so they hide and separate them. Therefore, our Association thinks that the best way to fight this negative state is to contact directly with families where persons with Muscular Dystrophy live, and publish their true living life. In addition, we think to achieve this by an automobile which will tour through rural communities (faraway countries).
    Inside of our Association board there are 4 people employed: 1 Albanian, which is also the president of Association, 1 Serbian local president of Graçanica, 1 Muslim, the manager of the office and 1 Ashkali is office assistant.
    Volunteers are also included: 1 Neurologist, 1 Internist, 2 Pediatrician and 1 Cardiologist. All of them are contributing a lot in our activities in the Muscular Dystrophy Association of Kosova.

    President Association:
    Agim Vatovci,Str”Ardian Krasnici”N:6/22 – Prishtina- R.Kosovo
    Phon:038-247-721,Mob:044- 377- 377.

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