WHO: Rare Disease Resolution Is Silent On Use Of TRIPS Flexibilities

WHO logo. Wikimedia Commons.

New Delhi, 28 January (Chetali Rao and K M Gopakumar) – The proposed World Health Assembly (WHA) resolution on rare disease maintains silence on the use of flexibilities in the Agreement on Trade-related Aspects of Intellectual Property Rights (TRIPS Agreement), administered by the World Trade Organization (WTO), and downplays the critical issues of access, availability and affordability of high-cost medicines and treatment for rare diseases.

The proposed resolution will come before the 156th session of the WHO Executive Board (EB)  that will take place from 3 to 11 February, at the WHO Headquarters in Geneva in a hybrid mode.

[It is a practice for WHA resolutions to be reviewed during the EB meeting before being presented for adoption at the WHA. There are nearly 20 resolutions currently under discussion among Member States and expected to be discussed during the upcoming EB meeting.]

The resolution on rare diseases being led by Egypt and Spain, is also co-sponsored by Chile, France, Kuwait, Malaysia, Panama, Palestine, and Qatar.

This is not the first time that rare diseases are addressed at a global platform. In 2019, countries adopted the UN Political Declaration on Universal Health Coverage that includes mention of rare diseases. The UN Resolution on Rare Diseases adopted in 2021 was the first text to provide visibility to persons living with rare diseases (PLWRD). Further, the 78th Session of the UN General Assembly adopted a follow-up resolution (UNGA A/Res/78/173) in December 2023. Paragraph 12 (a) of  this resolution states:

“To progressively cover persons living with a rare disease, and those with an undiagnosed rare disease, with quality essential health products, health services, including adequate preventive measures such as newborn screening, and quality, safe, effective, affordable and essential medicines and therapies, diagnostics, with the particular aim of shortening and easing the pathway to a diagnosis and treatment for persons living with a rare disease, health technologies, and strengthened primary health care, referral pathways, multidisciplinary care coordination plans, increased registration efforts, and access to specialized care, with a view to completely covering all persons living with a rare disease by 2030”.

Paragraph 12 (b) calls upon Member States  “To reverse the trend of catastrophic out-of-pocket health expenditure”.

Operational Paragraph (OP) 1.1 of  the draft WHA resolution proposes to commit Member States to take 14 actions, starting with providing appropriate support to WHO in developing a Global Action Plan on Rare Diseases.

From an access to medicines perspective, the most important sub-paragraphs are OP 1.1 (b) and (d). OP 1.1 (b) states:

“To integrate rare diseases into national health planning by developing and implementing national policies, effective programs and actions, including developing primary and secondary evidence-based preventive actions, and strategies aimed at preventing and improving health care services for PLWRD through an integrated approach, ensuring equitable access to timely, cost-effective and affordable, available, accurate diagnosis, particularly for newborns through universal screening program, and necessary cost-effective treatment, social and health care services;

To accelerate efforts toward achieving and extending UHC (Universal Health Coverage) by 2030, ensuring healthy lives and well-being for all individuals including PLWRD throughout their life course, in order to stop the rise and reverse the trend of catastrophic out-of-pocket health expenditure as appropriate, by re-emphasizing the commitment to progressively provide PLWRD with quality essential health products, healthcare services, and affordable medicines, diagnostics, and health technologies by 2030”.

Member States are believed to have reached a consensus on these two paragraphs. As detailed below, these paragraphs follow a narrow approach to accessing new and efficacious medicines for treating rare diseases, and fail to continue the momentum of the UNGA Resolution A/Res/ 78/173. The resolution fails to deliver any actionable outcomes that can help enhance affordable access to new and effective medicines to PLWRD.

Access Challenges In Rare Diseases

Rare diseases are a marginalized/neglected area in efforts to address global health challenges, both in terms of accessibility and in products that are being developed for rare diseases.

There are approximately 7,000–8,000 rare diseases which affect nearly 350 million to 475 million people globally. Rare diseases can be both life-limiting and threatening, with a disproportionate impact on children. 75% of rare diseases affect children and more than 30% of the children with a rare disease die before the age of 5 years. Unfortunately, treatments are available for only 5% of the known rare diseases.

The lack of access to medicines for rare diseases clearly shows the flaws of the current pharmaceutical system. For many rare diseases that affect small and/or marginalized populations, there was limited or no investments. This market-driven approach has severely impeded research and development in the area of rare diseases.

However, in recent times there has been a spurt in the number for drugs that are being developed for rare diseases. In 2024, of the 50 novel drugs approved by the USFDA  26 of 50, or 52% of these drug approvals were for rare diseases. While the benefits of these newer drugs and therapies are remarkable, their high prices render them inaccessible outside high-income countries. Despite the increase in approvals for drugs for rare diseases, innovation is not translating to accessibility, especially for developing countries as all such new drugs are being commercialized almost exclusively in high-income countries, with prices set as high as USD 4.25 million per patient (for a rare disease metachromatic leukodystrophy, or MLD). 

The high price of medicines for rare diseases is a major concern at a time when national governments face economic difficulties, and budget‐constrained healthcare systems find it difficult to meet the escalating demand for treatments.

WHO Member States with fragile health systems struggle to allocate budgets and resources specific for rare diseases, and where an amount is allocated, it is paltry to cover the high costs of drugs. The lack of concrete actions to address the access to high-cost medicines would lead to a rise in the catastrophic out-of-pocket health expenditure for PLWRD and their families, who continue the odyssey of navigating the challenges of obtaining these expensive treatments. For the vast majority of families in developing countries such treatment is simply not possible. The inability to procure costly medicines and ensure proper treatment ultimately erodes their trust in healthcare systems.

Resolution’s Weak Stance on Addressing TRIPS Flexibilities

In OP.1.1 the text states as follows:

“… (b) To integrate rare diseases into national health planning by developing and implementing national policies, effective programs and actions, including developing primary and secondary evidence-based preventive actions, and strategies aimed at preventing and improving health care services for PLWRD through an integrated approach, ensuring equitable access to timely, cost-effective and affordable, available, accurate diagnosis, particularly for newborns through a universal screening program, and necessary cost-effective treatment, social and health care services;…

(d) To accelerate efforts toward achieving and extending UHC by 2030, ensuring healthy lives and well-being for all individuals including PLWRD throughout their life course, in order to stop the rise and reverse the trend of catastrophic out-of-pocket health expenditure as appropriate, by re-emphasizing the commitment to progressively provide PLWRD with quality essential health products, health care services, and affordable medicines, diagnostics, and health technologies by 2030;…”

These two paragraphs  not only legitimise the high cost of drugs but also do very little to address the existing intellectual property barriers that have made many critical lifesaving medicines out of reach of PLWRD. The TRIPS Agreement includes various provisions that allow member countries to prioritize public health over intellectual property rights. The 2001 Doha Declaration emphasized that the TRIPS Agreement should not prevent WTO Members from taking measures to protect public health and promote access to medicines. However, governments have not effectively utilised the flexibilities provided by the TRIPS Agreement to protect public health and allow equitable access to drugs for rare diseases, often due to the political pressure from developed countries.

Pharmaceutical companies largely overlook the needs of the people suffering from rare diseases in developing countries. For instance, the drug Trikafta which is critical for patients suffering from Cystic Fibrosis is unavailable in India – despite epidemiological data suggesting that the expected Cystic Fibrosis population in the Indian subcontinent is greater than that of the United States. The drug is priced at a cost of $326,000 per patient per year (PPY). Vertex has filed multiple patents in India to block generics from entering the market in India, but has not taken any steps to register the drug in India.

Similarly, Risdiplam a drug for treating Spinal Muscular Atrophy (SMA) is steeply priced at $80,000 PPY, leading to significant access issues in developing countries including countries like India, where generic manufacturers have the capacity to produce the drugs at a price which is 95% lower than the originator price. Abuse of patent monopolies by pharmaceutical companies has undermined the potential of countries like India to tap the benefits of a robust generic industry. Such patent monopolies have resulted in the denial of access to thousands of PLWRD in developing countries.

WHO has a history of adopting resolutions that include clear language on use of TRIPS flexibilities, that has enabled countries to take steps to enhance access to life saving drugs. In previous resolutions such as WHA 57.14, Member States were urged to adapt national legislation to make full use of TRIPS flexibilities to access essential drugs for HIV/AIDS.

One notable instance of the use of these flexibilities was a case of Hepatitis C treatment, where several countries utilized TRIPS flexibilities to improve access to life-saving drugs. Though an absence of language on TRIPS flexibilities does not take away member states’ rights to use these flexibilities, yet an emphasis on TRIPS flexibilities in the resolutions reflects WHO’s commitment to prioritizing right to access medicines over strict patent protection. The use of TRIPS flexibilities such as issuance of a compulsory license is often regarded as a discretion or last resort by the government rather than an obligation.

Missing Transparency on R&D Costs for Rare Disease Drugs

Another point where the resolution falters is the lack of specific language on the R&D costs for drugs for rare diseases. Pharmaceutical companies prioritise profitability over public health needs in the case of rare diseases. What worsens the situation is that there is no transparent way of assessing the cost of production to justify the high prices of these drugs.

Pharmaceutical companies claim the average price of bringing a new drug to the market as $2.6 billion, a figure that also includes the costs of their failed developments. However, they overlook the fact that drugs for rare diseases receive a slew of tax incentives, extended exclusivity periods and lower production costs due to a smaller set of trials, among others. Charging inflated prices for these drugs seems completely disproportionate to the cost of production of these drugs. Research findings have suggested that the production costs of rare disease drugs is 90% lower as compared to the originator prices.

According to Dr. Melissa Barber, the drug Risdiplam (for SMA) provides a glaring example of how the pharmaceutical ecosystem is prioritising shareholder profits over the needs of people. While the market price of Risdiplam is much higher, research shows that the drug can be produced at a fraction of the price by a generic manufacturer. Similarly, an analysis by researchers on Trikafta suggests that a year’s supply of drugs can be manufactured for less than $6000, while the drug is being sold at $326,000 PPY.

With no clear verified information on the cost of production, countries struggle to negotiate fair prices with pharmaceutical companies. The absence of language in the proposed WHA resolution on transparency of production costs raises concerns on the effectiveness of the resolution in ensuring equitable access to rare disease medicines, leaving a void in the accountability of the pharmaceutical companies.

Silence on Addressing Technology Transfer a Step Backward

Paragraph 7 (b)  of  the UNGA Resolution 78/173 encourages Member States “To structure and coordinate, at the national and international levels, knowledge on the subject of rare diseases, and information-sharing to optimize the use of existing resources and facilitate access for all persons with an undiagnosed rare disease, while acknowledging the need to support developing countries in building expertise and in developing local and regional manufacturing capacities for health products and technologies”.

The WHA resolution’s conspicuous silence on technology transfer, represents a regression from the UNGA resolution.

By maintaining a stoic silence on pressing issues such as the use of TRIPS flexibilities, transparency in R&D costs and technology transfer, the resolution will perpetuate inequity rather than pave the way for equitable access to rare disease medicines and improving the health outcomes for PLWRD. – Third World Network (TWN)