The global Pompe disease market will experience a boost during the study period of 2019-28. Pompe disease is a rare inheritable disorder with a combined Pompe disease prevalence of 1 in 40,000, affecting males and females equally.
The Pompe disease therapeutic market revolves around pharmacological treatment (which comprises of only a single component, i.e., Enzyme Replacement Therapy as well as supportive therapies used for the management of Pompe Disease. Earlier, supportive care had been the backbone of Pompe disease treatment. But after the approval of ERT, most of the revenue generated in the Pompe disease market was because of it. ERT was the first disease-specific Pompe disease treatment for an inherited muscular disorder consisting of the intravenous administration of recombinant human GAA (Myozyme). However, the cost of Pompe disease therapies has been an issue, especially in countries where basic medical insurance is not very good, and the overall burden falls on the patient.
Pompe Disease Emerging Markets
Companies like Amicus Therapeutics and Actus Therapeutics are already in the race to develop drugs accelerating Pompe disease treatment market forward. The expected launch of emerging therapies such as AT-GAA (Amicus Therapeutics), is expected to create a favourable impact on the Pompe Disease market size in the upcoming years. The increase in Pompe disease awareness and support from various organisations have assured many other companies from various different geographies to carry forward their research in treating and developing treatments for Pompe disease. Companies from 10 Major Markets, regions are also known as ‘Pharmemerging’, Middle East (UAE AND Saudi Arabia), Asia (China and Taiwan), Eastern Europe (Turkey and Russia) and Latin America, or LATAM (Colombia, Argentina, Brazil and Mexico) are fueling the Pompe disease market.
Among all of these countries, China is contributing to the highest amount of revenue, followed by Brazil. Although the Pompe disease treatment scenario in emerging markets is more or less alike as in the United States and Europe.
For instance, Mexico, one of the 10 MM, has reported higher access to state-funded medicines, but the benefits at the ground level have not been handed over to the payers. Therefore, it appears that improved access has not been translated into the lower self-pay structure for Mexico. If we talk about, Russia, the situation is a bit tricky with almost an equal share of access to state-funded medicines as well as self-pay element within the reimbursement framework. The same factors come into play when it comes to state-listed medicines that are access and trust (quality) issues. There seems to be an ever-increasing reliance on branded drugs which requires out of the pocket payment from the buyers. The trend in Brazil and Turkey, however, appears to be reversing a bit since the past five years with a slight fall in reported levels of self-pay. However, when compared to the figures reported for patient access to state-funded reimbursements, these numbers seem to be too low. Coming to China, the state insurance and reimbursement scenario in China is a tad different. Although the Chinese government has rolled out state medical insurance for some drugs over the past decade, a majority of orphan drugs are not included. The situation has, however, been changing of late with the China Food and Drug Administration pulling up their socks to speed up the process for approving new treatments.
No doubt, that the promising Pompe disease market has attracted companies from all over to invest in developing and advancing Pompe disease therapeutic market. Despite these developments, in a nutshell, it can be concluded that getting insurance coverage in the Emerging markets for orphan drugs is very tough, whereas the system in the developed world (the US and Europe) is more mature and robust.