Interview: Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked

In Market Access by Freya Smale

imageAs part of a series of interviews conducted with leading industry experts in the run up to the World Orphan Drug Congress in Geneva, Total Orphan Drugs was delighted to sit down with Dr Tony Zbeidy, General Manager of Orphan-Europe Middle East FZ LLC. Total Orphan Drugs would like to take this opportunity to thank Dr Tony Zbeidy for speaking to us.

The Middle East is full of unmet needs for orphan disease drugs; companies should be hands-on, according to Tony Zbeidy, MD, General Manager of the MENA region at Orphan-Europe.

At drug development company Orphan-Europe, Tony Zbeidy, M.D. focuses on developing markets in the Middle East: Saudi Arabia, UAE, Qatar, Kuwait, Turkey, Iran, Iraq and Libya. According to Dr Zbeidy, the global economic downturn affecting the US and Europe has had little impact on the health sector in North Africa and the Middle East; countries in this region maintained a high growth rate. In fact, most Gulf countries in the Middle East have seen a double-digit growth, some reaching up to 40 percent for the past 2 to 3 years. This unusual growth is driven by the high rate of consanguinity—up to 60 percent in some countries, compared to 1 percent in the U.S. and 3 percent in Europe. It is also driven by the neonatal screening programs recently put in the place and the government policies in favor of taking in charge life-long diseases. “In the Middle East there is money and also the capacity for multi-centric studies.” Zbeidy said. There is full governmental reimbursement for orphan drugs in many countries. For the most part, there is some degree of healthcare infrastructure; health professionals with western training in their specialties as well as clinics and referral centers.

However, MENA countries are large, but have small populations. Specific cultural beliefs may be obstacles to patient care and accessibility of care. Each country has its own specific attributes. For example, fast growing populations such as Saudi Arabia, Turkey, Iran, Iraq and Libya have a high degree of consanguinity, which correlates with a high incidence of genetic disease. In Turkey, medical care tends to match European health standards. However, national scientific events are in Turkish and Westerners would encounter language barriers with physicians. In Iran, the government subsidizes orphan drug care.

In the Gulf Region, the government runs hospitals and referral centers.

In contrast, Algeria, one of the rare countries in the world which has not had a budget deficit for years, does not have an infrastructure for rare disorder treatment; so it is necessary for companies to focus on raising health awareness and physician training. “In Algeria there is not one single children’s hospital.” said Zbeidy. “There is not good diagnostic capacity. Physicians are not diagnosing and treating rare disorders.”

In Iran, non-medical challenges can include the political environment, language barriers, sanctions, lack of adherence to patent law, and use of products not registered by the FDA or EMA. Iraq has a high healthcare budget and medical care is Western-oriented. However, there are significant ongoing administrative and security issues.

International companies reluctant to invest or conduct clinical trials in MENA countries are missing on opportunities, as rare disease research should target regional populations.

Zbeidy recommends establishing specialised, dedicated platforms to run clinical trials. Patient organizations do not exist, especially because organization of groups is forbidden in the Gulf region; one tactic is to run patient days at medical centers that work with rare disease patients and invite patients and families.

Currently, international companies rely on local distributors to develop their markets. This is unwise, according to Zbeidy, as the distributors can take excess profits, which can damage market development and corporate image. It is far better to establish a regional office to direct medical marketing, and assign local distributors to deal with logistics only. Reliable market information is harder to source as the international standards and published economic data in the western world doesn’t apply in MENA.

Markets estimated to be limited have ended up as big businesses in MENA because some rare diseases are not that rare—the incidence rare diseases in MENA can be 10 and 11-fold that of the Western world, Zbeidy said.

To make multi-stakeholder partnerships work, Zbeidy advises companies to be hands-on in developing networks of expertise, especially as the target group of health professionals is small. “All you need is to find the right person who fully understands both Western and regional cultures and who is capable of establishing a highly scientific group of local representatives.” said Zbeidy.

According to Zbeidy, key game changers altering the dynamic of the orphan drug industry for developers include big pharma companies acquiring orphan drugs/orphan drug companies (Sanofi, Pfizer, Astra Zeneca, Recordati). A large number of rare disorders still remain without treatment, meaning competition for new entrants is less likely. The global economic situation implies tougher price controls of rare disorder drugs –however, taking into account the unmet medical need, defending a price is always possible. There is a correlation between consanguinity, demand and market potential for companies in the right place and making the right investment. Regional cultures have evolving human care standards –priorities are changing in the interest of health sectors and more and more patients are now being diagnosed and treated; for example, Qatar established a newborn screening program covering 36 disorders.

Zbeidy has the following recommendation for pharma companies considering entering North Africa and Middle Eastern countries:

1. Reconsider drug development strategies, instead of investing in clinical trials for US and EU only.

2. If you have a promising molecule, establish at least a small representative office in the target country.

3. Be aware that there are no organized patient groups in the Middle East and Gulf region but there are other ethical ways to work with patients.

imageSpecial thanks go to Wendy Wolfson who conducted the interview on behalf of Total Orphan Drugs.

Wendy Wolfson covers innovations at the intersection of medicine, science and healthcare as a columnist for Chemistry & Biology, a publication of Cell Press. She has contributed to magazines including Science, Nature Biotechnology, the Lancet, and Red Herring. Her work can be found at and she can be contacted at

Read more interviews in this orphan drug series
Partnering from the pharma perspective
How to deliver value to patients
Perspective from patient advocates in Sweden
Interview: Surmounting barriers to patient access
Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked
Where do the current and future challenges lie in bringing new orphan drugs to market?
Bringing Biotech Spirit to a Non-Profit Environment
Paving the way for a whole new medical paradigm
Tailoring your reimbursement strategy, step by step, country by country
An Interview With Pfizer on the Orphan Drug Industry: Part I
An Interview With Pfizer on the Orphan Drug Industry: Part II
An Interview with the Chair of the IRDiRC
An Interview with an Orphan Drug Payer