By: Greta Solinap, Patrick John Acuña, Abegail Aguil, Criscely Go and Nutan Sharma

Program context: Serving patients with rare diseases presents significant challenges to well-developed health systems. In remote and underserved settings, patients with rare diseases are often neglected altogether. This case study shows how a community health worker approach, using “community advocates,” improves patient care and outcomes in even the most remote and hard-to-reach settings.

X-linked dystonia parkinsonism (XDP) is an adult-onset, genetic movement disorder that occurs predominantly in men with ancestry to the island of Panay in the Philippines. It commonly presents as uncontrollable muscle contractions in one part of the body that spread to multiple body regions over time. Symptoms typically emerge at an average age of 39 years and consist of impairments in movement such as body rigidity, slowing of movements, and tremors. There is currently no cure for XDP and treatment is often symptomatic. Treatment regimens include muscle relaxants, pain medications, Parkinson’s disease medications, antidepressants, botulinum toxin injections to relieve painful muscle contractions and physical, occupational, and speech therapy. A small handful of patients with XDP worldwide have been treated with deep-brain stimulation (DBS) therapy, which has proven relatively efficacious.

The estimated prevalence of XDP is at least 5 cases per 100,000 people on Panay (population 4.5 million), where the majority of patients have been identified.¹ Due to this adult-age onset, XDP often causes patients to lose their ability to work and earn income, frequently devastating families. Additionally, Panay is primarily a rural community with extremely limited access to healthcare and social services. Without proper treatment, resources, and education, XDP will continue to affect generations of families to come.

To provide support for patients with XDP, anonymous donors established the Sunshine Care Foundation’s (SCF), a non-profit organization, in 2013. SCF instituted a monthly outpatient clinic named the Dystonia Clinic in a Roxas City community hospital located in the Capiz Province of Panay Island (Map²). Through private in-kind and monetary donations, free examinations and medications were provided during the monthly clinic. Grants given by institutions outside of the Philippines enabled a handful of Dystonia Clinic patients in Capiz to receive DBS treatment free-of-charge in the United States and Germany.

Despite the free services offered at Dystonia Clinic, providers observed low attendance rates and recognized multiple barriers to care, including financial insecurity, social isolation, and lack of understanding of the disease. The researchers in this project aimed to formally assess patients’ barriers to care and evaluate the efficacy and cost-efficiency of community-based interventions including the engagement of community health care workers to provide home care, and to address these barriers.

Program design: SCF employees identified XDP patients at the clinic and these patients provided verbal informed consent to participate in a community-organizing program. Between July and August 2014, SCF employees collected information regarding barriers to care through home interviews (n=84) and focus group discussions (FGD) (n=26). A Catholic sister, serving as a community organizer in the area, and a physician, who had been serving the community for several years, led the focus-group discussions. Topics included participants’ purpose in life, challenges and positive factors of daily life, and ideas for improvement of healthcare, including transportation, clinic schedules, and waiting times.

Based on the findings from this data collection, the SCF team designed a community-organizing program to improve patient care. The program hired local individuals in October 2014 to oversee a group of XDP patients in the region where they live. The first group of employees chose to call themselves “community advocates” (CAs). A key feature of the program was that all the community advocates hired were directly related to XDP patients (i.e. mothers, daughters, wives). The SCF paid them a monthly stipend comparable to an average local part-time salary. CAs began their role by assisting SCF members in conducting home visits and participating in team meetings about community needs. They were also trained by SCF employees on how to keep patient information confidential and to record and organize information.

After this initial training period, they were given independent tasks, which involved visiting a group of consenting patients in the vicinity that they were assigned to, usually between 5-10 patients. All participating patients were given the option to opt out at any time after the program started. Every month, the CAs conducted home visits to ask about urgent health concerns, facilitate patient attendance at the Dystonia clinic, and disseminate educational information. The SCF team held monthly meetings where CAs chose topics that were relevant to XDP patients, as patients frequently had other more common diseases in addition to XDP. SCF employees arranged for health professionals to train the CAs in chosen topics including nutrition, hypertension, diabetes, and government financial assistance programs.

For the nutrition project, the team hired a nutritionist to train the CAs and provide specific nutrition and diet information tailored for XDP patients. For an overview of government programs, the project engaged a federal social worker to give a presentation on the financial benefits for which XDP patients qualify. Once this information was taught to the CAs, the team gave handouts and specific activities to discuss with the patients and caregivers during their next home visits. Other key interventions implemented included reimbursing transportation costs for monthly clinic visits, creating transportation plans, and triaging patients to reduce wait times.

CHW program impact: The Community Advocate Program was a crucial mechanism for acquiring key information in addressing access to health services. The program conducted a survey (n=113) on the barriers to care and specific socioeconomic challenges patients faced (Figure 1). The major barriers were no money for transportation (44%), rain and weather issues (24%), patient not feeling well (16%), no companion to accompany patient (8.0%), patient embarrassment (5.0%), and other reasons (3.0%). Once these barriers were identified, the CAs discussed feasible ways to address them and formulated multiple plans, including transportation plans, arranging companions for patients with no family members available, and addressing the other comorbidities that XDP patients had.

Transportation to and from the clinic was a major barrier for patients. Very few patients owned vehicles, so they relied on public transportation to get to clinic. However, available transport – buses, motorcycles, and pedicabs (i.e. bicycles with seats for passengers) – often do not take patients directly to the clinic and typically require multiple transfers, making the journey grueling for immobile or movement-restricted patients. Modes of public transportation also have a limited schedule on Sundays so patients would sometimes travel four to five hours to get to the clinic. Additionally, the cost of bringing someone to accompany them was often unaffordable so patients would have to travel alone, making the journey even more strenuous.

Patients also reported discomfort being seen in public because XDP can cause repetitive, involuntary abnormal movements as well as pronounced muscle contractions, causing people to stare, move away, or otherwise make patients feel uncomfortable and embarrassed. To address these barriers, CAs found many innovative ways to get patients to clinic: hiring a public bus (“jeepney”) for the patients and their family members and creating routes for the bus to limit the amount patients had to walk, arranging for local city ambulances or vehicles to pick up severely incapacitated patients, and triaging patients so that patients who lived farthest from the clinic were seen first since they had long distances to travel.

Figure 1: Barriers to healthcare reported by patients (n = 113)

The data from the program suggests that clinic attendance can be improved by taking a community-based approach to address the major barriers in accessing healthcare. In the first and second month of program implementation, the number of patients attending the Dystonia clinic increased by 62%  and an additional 11% respectively. The clinic had to limit the patients it saw after these increases due to space and time restrictions of the providers. The average number of patients enrolled in the entire program from October 2014 to April 2015 was 111 patients per month (range 93-117) with an average of 91 home visits (range 81-112) conducted per month. The cost of transportation reduced 35% per patient after the program was implemented (5.81 USD compared to 3.78 USD per patient), demonstrating that the transportation plans implemented were more cost efficient than patients’ sourcing transport individually.

The average cost per month of the community advocate program during the first five months (excluding patient transportation) was 628 USD or 5.66 USD per patient involved, which includes the salaries of the CAs, costs associated with training CAs, transportation costs for home visits conducted by CAs, and material costs.  

Since 2014, the CA program has expanded from 10 advocates to 21 advocates, four of whom are based in surrounding provinces. The number of patients enrolled has more than doubled from 111 to 246. The program facilitates frequent communication between the CAs, SCF staff, medical providers, and patients. With this improved and long-term communication between providers and patients, the clinic has been able to use clinical resources more efficiently. For example, the SCF now provides a weekly Friday clinic led by a general primary care provider based on the island for patients with no active medical problems, while new patients or patients with new active medical problems related to their XDP can attend the Sunday clinics, which are staffed by movement disorder specialists who travel in from outside the island.

The direct impact on patients and families includes improving the healthcare they receive from the SCF – as a result of improved communication of symptoms and medical information – reducing wait times at clinics and increasing the time practitioners spend with patients. Additionally, the CAs aid in forging relationships with patients and practitioners in other cities and islands. The SCF has organized Dystonia Clinics in five other cities which have yielded information for the national registry of XDP patients.

In addition to improving the use of clinical resources, the CA program has helped the SCF gauge interest in clinical research among patients and establish partnerships with key researchers in the Philippines and abroad in the hopes of initiating clinical trials and studies that can significantly improve understanding of XDP and improve patients’ quality of life. One of these clinical trials includes testing the use of “MINGO”, a food supplement made of locally available moringa, rice and mung beans, and investigating whether its use can reduce the drastic weight loss seen among XDP patients.³

Lessons learned: The CA model provides a way to provide culturally sensitive, effective, and practical medical care and social services to populations in extremely resource-limited settings. For example, new transportation options significantly improve clinic attendance and longitudinal care. The new transportation options also address other barriers to attending the clinic: lacking a companion, feeling embarrassed in public, and not knowing when the clinic is held. The SCF model could be replicated in other contexts by coordinating with local hospitals and government agencies to leverage funds available for disabled patients. As the number of patients and families affected by XDP continue to increase along with awareness of XDP in the general population, other mechanisms such as forming community savings funds may help further expand access to XDP services.  

Patient and family input are critical to the development of an effective program. Ideas for change and solutions in the XDP project are primarily generated and implemented by family members and patients. CAs often produce ideas and suggestions from talking to the patients and families. Patient healthcare needs are better identified and understood by the team of diverse providers due to the improved knowledge of the communities’ health. Furthermore, employment as a CA provides a source of income for XDP-affected families, including patients, as all the CAs are selected from XDP-patient households, therefore contributing to the net household income.

Sustainability: A pipeline of initiatives to improve the long-term sustainability of the program is being developed. The free medications that patients receive are sustained by financial donations from various international donors, including SCF donors, as well as through donations directly from pharmaceutical companies. Physicians and other healthcare staff who work for the clinic volunteer their time. To mitigate the risk of losing volunteer providers, some professional medical associations have made volunteering a core part of their societies, including the Movement Disorders Society of the Philippines and the Philippine Neurological Association. Additionally, with the clinic’s improved efficacy, fewer volunteers are needed to address the medical need.

All other expenses, including fees to maintain the clinic and the community advocate program, are supported by the SCF. The SCF is attempting to establish partnerships with local schools that will allow trainees from fields such as nursing, nursing assistants, and healthcare administration to work in the clinics and CA program. Another active initiative includes collaborating with PhilHealth, the national health insurance provider of universal healthcare, to increase reimbursement of health services to patients with XDP.

A major goal of the SCF is to provide income-generating opportunities to patients and their families. Multiple initiatives are in the pipeline, including providing job training in fields like organic farming, sewing, and craft-making through collaboration with various local organizations. The long-term goal of the CA program is to be financially independent.

Author Affiliations

Greta Solinap: Warren Alpert Medical School of Brown University, Providence, Rhode Island, U.S.A.

Patrick John Acuña and Nutan Sharma: MGH Collaborative Center for XDP, Massachusetts General Hospital, Boston, Massachusetts, U.S.A.

Abegail Aguilc and Criscely Go: Sunshine Care Foundation, Capiz, Roxas City, Philippines

[1] Lee LV, et al. (2002) The natural history of sex-linked recessive dystonia parkinsonism of Panay, Philippines (XDP). Parkinsonism Relat Disord 9(1):29-38.

[2] Eugene Alvin Villar. https://en.wikipedia.org/wiki/Capiz#/media/File:Ph_locator_map_capiz.png

[3] MINGO Supplemental Trial in X-linked Dystonia-Parkinsonism Patients (MINGO). Sponsor: Sunshine Care Foundation. https://clinicaltrials.gov/ct2/show/NCT03019458