Table 3.

Challenges encountered during the implementation and ongoing management of the Humanitarian Program

	Challenges	Solutions
Infrastructure	Lack of infrastructure for diagnosis, treatment, and management of individuals with LSDs Availability of specialists in treatment of LSDs and treatment centers	Involve all stakeholders, including external partners (individuals with rare diseases, physicians, government, patient association groups, and NGOs) and internal team members at global, regional, and local level Facilitate diagnostic services by utilizing global and regional networks in countries where local testing is not available Engage with a respected NGO with a strong local presence to help facilitate development of in-country capacities Create expert medical committees to increase local knowledge and understanding of disease awareness, diagnosis, treatment benefit, and follow-up
Medical expertise	Requirement for ongoing training and education for healthcare providers to raise awareness of LSDs, support accurate diagnosis, assist with treatment access, knowledge of treatment administration, and disease management	Create a sustainable ecosystem for individual care within a given country Create centers of excellence and referral hospitals to facilitate proper diagnosis and initiation of treatment Provide local expertise and enhance relationships that help support individuals and navigate the challenges that may be involved in reaching them
Logistical	Order processing and shipment logistical delays Extensive delays incurred due to the need for advanced provision of extensive documentation and bureaucratic challenges, for example, obtaining import permits Navigating local financial bureaucracy around donations, including import taxes, duty regulations, and cost exemptions Additional lead time needed for testing, release, shipping, and packaging products in view of cold-chain considerations During the COVID-19 pandemic, many countries closed their borders, thereby disrupting the medication supply for many individuals	Partnerships with other humanitarian and patient organizations globally Coordinate with NGOs in order to obtain import permits, tax waivers, and to better understand bureaucratic obstacles Integrate Humanitarian Program activity into all business processes, including demand forecasting, supply planning, shipping, and trade compliance Find creative solutions to ensure uninterrupted treatment for individuals (eg, delivery via road rather than air, cargo reallocation of product between hospitals, higher shipment quantity to reduce number of shipments); critical during COVID-19 pandemic and times of country crisis
Regulatory	The need to work with local government, healthcare providers, and key stakeholders to establish sustainable healthcare services for individuals with LSDs that are compliant to local regulatory and/or government requirements from diagnosis through treatment administration and follow-up	Involve all stakeholders, including external (patients, physicians, government, patient association groups) and internal team members at a global, regional, and local level
Program sustainability and unmet needs	Ensuring program is sustainable Addressing unmet needs to specific populations due to program limitations (eg, lack of treatment access for newly diagnosed adults with Gaucher disease)	Active commitment and engagement from company leadership Engage with all local stakeholders and clearly communicate the program benefits and requirements Listen to the rare-disease community, recognize that there are unmet needs, and continue to evaluate program product offerings and criteria
Cultural and personal	Ensuring respect of cultural beliefs Recognizing spectrum of disease stigma	Rely on ethicists, religious leaders, and local expertise to respectfully navigate various cultural beliefs (eg, Islamic countries such as Egypt, where parents want their sons to be treated over their daughters) Partner with international patient associations when patient voice is needed and locally is not possible due to disease stigma

LSD, lysosomal storage disease; NGO, non-governmental organization