The Case for Rare Disease Clinical Trials in the Middle East

Most rare disease companies get interested in the Middle East, particularly in the GCC region, at the commercialization stage, following regulatory approval by the FDA or EMA. This interest is amply justified since there is a significant potential for orphan and rare disease drugs. Nevertheless, only a handful of companies consider the area for inclusion in international multi-centric pivotal clinical trials.

Having had recently many discussions on this topic with rare disease companies in the US and Europe, it has become clear that this is only due to lack of familiarity with the region and because there is no requirement to conduct clinical trials to obtain local regulatory approval. Nevertheless, we believe there is a strong case to consider conducting clinical trials in the Middle East as illustrated by these eleven arguments:

  1. The region has a significantly high marriage consanguinity rate.
  2. Family size remains large with many siblings. Patients for rare disease trials are not always available or easy to find in the US and Europe. The region can facilitate and accelerate recruitment for these trials
  3. Most GCC countries have newborn screening (NBS) programs allowing early diagnosis, including mostly for now, metabolic and hematological conditions.
  4. Patient registries are still limited and mostly hospital based. This is evolving positively since many diseases are now treatable, generating a higher degree of interest in patient identification.
  5. The number of experts, although still relatively small is increasing. Many experts are educated in prominent western universities and hospitals before returning to practice.
  6. Physicians in the Middle East have developed a significant experience, surpassing sometimes that of their counterparts in other countries since they see large numbers of rare disease patients due to higher prevalence and incidence of these diseases.
  7. Although most countries in the GCC lack a long tradition of medical universities and basic research labs, there are few tertiary centers that have full staffed and experienced clinical trial and research departments and are certified by local and international regulators.
  8. The number of centers able to conduct clinical trials in the region is limited, hence it is easier to coordinate and centralize the trial centers. Most other hospitals would be treated as referral centers.
  9. There are a number of experienced regional and local CROs that can handle the process of setting up a center and conducting the feasibility study.
  10. Due to the budget constraints, the market access environment is rapidly maturing and evolving towards an outcome-based, risk-sharing pricing and funding. The different MOHs in the region are encouraging companies to initiate local trials to bridge the gap with access and build local experience for the physicians in the most innovative treatment options.
  11. Finally, the cost of conducting clinical trials in the Middle East remains relatively lower than other regions.

We at Genpharm strongly believe that an earlier interest and inclusion of few centers from the region in pivotal clinical trials would support a better commercial outcome in the longer-term. The trials would reduce the length of the current patient pathway by encouraging earlier diagnosis. The experience and confidence acquired by the treating physicians will create a faster treatment pick-up following regulatory approval.

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