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Challenge: Inappropriate Selection

The appropriate selection of essential medicines for MNS disorders is critical to providing effective and accessible treatment to patients noted Hans Hogerzeil. The basic concept of essential medicines is that a limited range of carefully selected essential medicines leads to better health care, better medicine management, and lower costs. According to WHO, essential medicines are selected with regard to disease prevalence, evidence of efficacy and safety, and comparative cost-effectiveness (WHO, 2002b, 2013). It is the responsibility of each country to determine those needs and the list of medicines that are essential for their population; globally, most countries have an essential medicines list (WHO, 2002b, 2011c, 2014). Hogerzeil noted the absence of evidence-based treatment guidelines and said these, too, should be considered as important as the medicines included in a country’s essential medicines list. The development of these guidelines potentially could be financed and supplied in the public sector (e.g., governments).

Given the many challenges around access to existing medicines and the fact that 98 percent of the medicines on the WHO Model List of Essential Medicines are off-patent, another approach is to consider access to existing medicines that are off-patent and available from multiple sources as an option, rather than access to new, patented medicines that are generally more expensive and from a single source (Abegunde, 2011; Cameron et al., 2011a). In discussing financing and universal access to care, Hogerzeil noted that WHO has defined three dimensions of access to consider: proportion of population covered; proportion of services covered; and proportion of costs reimbursed. Increasing access may involve extending coverage, including additional services, and reducing cost sharing and fees (WHO, 2010b).

To help identify barriers to access, Hogerzeil detailed the nine diagnostic indicators or measurable points of assessment of country-level access to essential medicines drafted by WHO (see Box 3-1) (WHO, 2008a).

The selection of essential medicines at the individual country level is a two-step process, Hogerzeil explained. The first step selects from all the medicines in the world to those that are registered for use and allowed in the specific country. This step is usually the responsibility of the national regulatory agency and decisions are based on efficacy, safety, and quality. Hogerzeil noted that the number of registered products is likely to be extensive, so the second step is selecting which medicines will be stocked, used, and/or reimbursed. Decisions can be based on which products are the most clinically effective, safe, or cost-effective within therapeutic classes, said Hogerzeil. For example, in an SSA country, a national centralized medicines list may include between 500 and 700 products, and most would be expected to be available at teaching hospitals. A list for a district hospital may include 250 of those medicines; for a health clinic, perhaps 100; and for a center staffed by community health workers, maybe 30 products. Hogerzeil noted that although many medicines are allowed on the market, most are not actively

supplied or reimbursed by health insurance. High-quality medicines needed in the majority of health facilities and available at low cost are considered essential, said Hogerzeil.

APPLYING THE ESSENTIAL MEDICINES CONCEPT TO MNS DISORDERS

Hogerzeil highlighted several unique challenges for improving access to appropriate medicines for MNS disorders. Internationally, he noted there has been minimal to no political support for addressing issues related to MNS disorders. For example, MNS disorders are not listed in the MDGs¹ or the Alma-Ata Declaration.² However, a participant noted that WHO has recently developed a Comprehensive Mental Health Action Plan³ for 2013-2020 endorsed by 194 member states and adopted by the World Health Assembly.⁴

Focusing on selection, Hogerzeil stated that evidence on the effectiveness of medicines to treat MNS disorders is often unavailable, or is perceived to be unavailable. As a result, patient health outcomes are not yet well defined and are difficult to measure. Hogerzeil added that specialists such as psychiatrists oftentimes prefer to have a broad selection of medicines to prescribe for a specific MNS disorder rather than be confined to the limits of the country’s essential medicines list. There is a strong preference for “a range of personal choices” as opposed to the concept of essential medicines. Lastly, Hogerzeil stated that MNS disorders often require chronic treatment that may lead to catastrophic health expenditures, defined as more than 20 percent of income, and poverty due to unaffordable health care costs.

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¹The United Nations Millennium Development Goals are eight goals that all 191 UN Member States have agreed to try to achieve by the year 2015. The United Nations Millennium Declaration, signed in September 2000, commits world leaders to combat poverty, hunger, disease, illiteracy, environmental degradation, and discrimination against women. See http://www.who.int/topics/millennium_development_goals/en.

²International declaration that expresses the need for urgent action by all governments, all health and development workers, and the world community to protect and promote the health of all the people of the world. See http://www.who.int/publications/almaata_declaration_en.pdf.

³See http://www.who.int/mental_health/action_plan_2013/en.

⁴See http://apps.who.int/gb/ebwha/pdf_files/WHA66/A66_R8-en.pdf.

Barriers to Appropriate Selection

The WHO Model List of Essential Medicines includes products for MNS disorders in the areas of psychiatric disorders; mood disorders; anxiety, obsessive-compulsive disorders, and substance abuse; and epilepsy (WHO, 2013). Hogerzeil reiterated the point that most products on the list are generics because most are off-patent. Availability of generic supply in the public sector can be the most cost-effective way of supplying essential medicines, Hogerzeil said. However, low and/or variable availability of generic products in the public sector can drive patients to purchase products from the private sector, with a tendency toward branded products or branded generics that are three to six times the price. One participant noted the importance of further research on the impact of the availability of generic medicines in both the public and private sector on pricing and purchasing patterns. Several participants discussed the “psychology” of generic medicines as a barrier because of an inaccurate perception that more expensive medicines are more effective. A few participants noted that in some SSA countries, more than 30 percent of medicines are found to be counterfeit, fostering a negative attitude toward medications that do not carry a brand label (IOM, 2013b; SPS Program, 2011; WHO, 2008b). Hogerzeil added that poor-quality domestic medicines and counterfeits may be linked to “inadequate regulation and insufficient penalization” (Hogerzeil et al., 2013; WHO, 2010a). Given the small market value and low prices of medicines in SSA, Hogerzeil suggested there might be a lack of commercial interest in supplying medicines.

A central challenge influencing appropriate selection is lack of demand, said Hogerzeil. As discussed by Gureje, lack of public procurement may be due to the lack of diagnosis and treatment capacity in the public sector, patient demand, and political interest. Hogerzeil noted that patients will not seek care if there is no trust in the system to provide affordable and effective health service. Additionally, if health care providers are not trained to request certain medicines to treat MNS disorders, then the medicines will not be available. A participant noted that regulatory bodies in SSA do not always consult health care providers and experts in the field regarding which medicines are essential. Another participant added that staff changes within health facilities may impact the medicines procured for a public-sector pharmacy.

Prescription practices also impact selection, said Hogerzeil, and international evidence-based treatment guidelines are important for MNS

disorders. In the private sector, many health care providers prescribe and dispense. This practice may create a conflict of interest due to economic incentives to sell more expensive medicines. In the public sector, patient demand for medicines is weak due to low expectations of this sector (Basu et al., 2012). According to Hogerzeil, the field might benefit from focusing on building up the public sector by training and supporting health care providers to better treat MNS disorders. A participant pointed out that up-to-date national treatment guidelines are needed to reflect current practice and pricing. Revisions to national essential medicines lists and treatment guidelines can be slow and may include medicines that are not relevant, a participant noted. For example, a participant noted that amitriptyline—an antidepressant—has a higher degree of toxicity, but widely used throughout SSA because it is relatively inexpensive compared to the alternative safe medication, fluoxetine. A participant suggested that if WHO revised the essential medicine list to put fluoxetine ahead of amitriptyline, countries might modify which medication is selected.

Several participants suggested considering improving selection of essential medicines for a range of neurological disorders. However, one participant noted that for many neurodegenerative diseases, there are few effective or cost-effective medicines. WHO now has a special essential medicines list with appropriate dosages for children⁵; however, there remains a lack of safety data for pediatric use of these medicines. Hogerzeil noted that only one-third of national essential medicines lists include pediatric formulations.

The concept of essential medicines is a global concept, Hogerzeil stressed. The selection of essential medicines is closely linked to evidence-based clinical practice guidelines. In summary, Hogerzeil said that universal access to essential care for MNS disorders might be:

• Based on the expansion of outpatient care;
• Performed by trained paramedical personnel;
• Supported by evidence-based treatment guidelines and a national list of essential medicines;
• Supplied as generic medicines; and
• Reimbursed within social health insurance schemes.

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⁵See http://apps.who.int/iris/bitstream/10665/93143/1/EMLc_4_eng.pdf?ua=1.

LESSONS LEARNED FOR ADDRESSING INAPPROPRIATE SELECTION

As previously mentioned, five example programs addressing access to medicines were presented during the workshop to facilitate exploration of best practices and lessons learned from other programs. The examples were selected by planning committee members and included two country-level programs, an infectious disease project, and two noncommunicable disease programs. Highlights from the presentations of the lessons learned for addressing inappropriate selection are provided in Box 3-2. A full description of the examples as presented can be found in Appendix A.

CHALLENGES AND OPPORTUNITITES FOR ADDRESSING INAPPROPRIATE SELECTION

In preparation for focused discussions on selection, Atul Pande, senior vice president at the Neurosciences Medicines Development Center at GlaxoSmithKline, summarized key challenges that were discussed in the presentations and example programs. In discussing the criteria for selecting appropriate medicines several participants noted that for many of the medicines used to treat MNS disorders, the evidence on effectiveness is poor. In some cases, the designation of essential medicines are based on health care provider preferences as opposed to health outcome data. Likewise, deficiencies in training and supervision allow inappropriate selection to persist Pande noted. The principles of chronic disease management apply equally to MNS disorders as they do to conditions such as diabetes and hypertension.

Following the focused discussion, Pande summarized that three barriers were identified by individual participants relative to selection of MNS medicines: (1) lack of mechanisms to clearly define which MNS disorders to treat; (2) lack of evidence-based approaches for developing essential medicine lists and treatment guidelines; and (3) gaps related to training in developing point-of-care prescription lists and continued awareness of evidence to update individual country lists. All constraints and/or barriers and potential opportunities noted by individual participants are included in Table 3-1.

The need for a national strategy for the treatment and care of MNS disorders was emphasized by several participants, Pande said, but such a strategy that can drive the appropriate selection of MNS medicines is still lacking or inadequate in some countries. A few participants noted that adequate resources, including specific budget allocations, might be needed to promote such a national strategy. Several participants also discussed the value of the WHO essential medicines list and mhGAP treatment guidelines as starting points for developing country-specific lists and guidelines. Pande noted that adoption of the WHO list and mhGAP guidelines might result in greater use of evidence-based approaches to treating MNS disorders. A few participants noted that the selection of medicines at the point of care is variable based on prescriber preference, and the consistent application of treatment guidelines could help to achieve some degree of uniformity. Lastly, many participants discussed the role of continuing education and training to ensure provider awareness and adherence to evidence-based treatments for MNS disorders.

TABLE 3-1 Opportunities to Address Inappropriate Selection of Essential Medicines as Identified by Individual Workshop Participants¹

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¹This table presents challenges and opportunities discussed by one or more workshop participants. During the workshop, individual participants engaged in active discussions. In some cases, participants expressed unique ideas and/or differing opinions. However, because this is a summary of workshop comments and does not provide consensus recommendations, workshop rapporteurs endeavored to include all workshop participant comments. This table and its content should be attributed to the rapporteurs of this summary as informed by the workshop.

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^aAlbert Akpalu

^bYonas Baheretibeb

^cHans Hogerzeil

^dDavid Michelson

^eAtul Pande

^fSolomon Teferra