On 16 December 2020 the World Health Organization (WHO) published a paper that delighted in the name, Expanding access to effective treatments for cancer and rare and orphan diseases, including medicines, vaccines, medical devices, diagnostics, assistive products, cell- and gene-based therapies and other health technologies; and improving the transparency of markets for medicines, vaccines, and other health products (EB148/9). The reason for the unwieldy title was documented by KEI in November 2019, WHO defers South Africa’s proposal on expanding access to cancer treatments including cell and gene therapies until 2021. This WHO board paper merges a submission by South Africa on cell and gene therapies (17 September 2019) and proposal by Peru on rare and orphan diseases (18 September 2019). In addition, the paper provides a report back by the WHO Secretariat on their implementation of the WHO transparency resolution (WHA72.8).
Background
On 17 September 2019, South Africa submitted a proposal for inclusion at the 146th session of the Executive Board entitled, “Expanding access to effective medical treatments for cancer, including drugs, vaccines, medical devices, diagnostics, assistive products, cell- and gene-based therapies, and other health technologies.”
On 18 September 2019, Peru submitted a proposal for inclusion at the 146th session of the Executive Board entitled, “Access to medicines and vaccines with emphasis on rare or orphan diseases”.
In October 2019, the Officers of the 146th Executive Board decided to merge the requests by Peru and South Africa and defer discussion till January 2021. The Officers of the Board decided to:
merge and defer to the 148th session of the Board the items proposed by the Governments of Peru and South Africa on, respectively, Access to medicines and vaccines with emphasis on rare or orphan diseases and Expanding access to effective medical treatments for cancer, including drugs, vaccines, medical devices, diagnostics, assistive products, cell-and gene-based therapies, and other health technologies.
EB148/9 – Report by the Director-General
The WHO board paper is comprised of two sections. Section A deals with “progress made in implementing resolution WHA70.12 (2017) on cancer prevention and control in the context of an integrated approach, on and access to health products for rare and orphan disease”. (Source: EB148/9). Section B deals with the the implementation of WHO transparency resolution.
Section A – ACCESS TO SAFE, EFFECTIVE, QUALITY-ASSURED AND AFFORDABLE HEALTH PRODUCTS FOR CANCER AND RARE AND ORPHAN DISEASES
In relation to rare and orphan diseases, the Secretariat paper lays out the following challenges facing many countries.
Because many patients pay for health products through out-of-pocket expenses, even low-cost products may be unaffordable. Chronic conditions require long-term care, placing a greater burden on patients and governments. Rare and orphan diseases affect a small number of patients and their management presents specific challenges, including the need for complex and specialized care. These diseases may not be considered a priority in universal health coverage packages and may thus be left out of public procurement and reimbursement.
On the role of cell and gene therapies, the Secretariat paper posits the following.
Cell therapies, gene therapies and cell-based gene therapies have the potential to meet the medical needs of individuals with certain cancers and rare and orphan diseases. Cell therapies, gene therapies and cell-based gene therapies vary in nature, and the relevant regulatory framework and evaluations are not harmonized or even in place. The high prices of these products and patent barriers limit access to a few high-income countries: chimeric antigen receptor T-cell therapy, for example, is priced in the hundreds of thousands of US dollars and gene therapy for spinal muscular atrophy is priced in the millions of US dollars.
Absent from the WHO analysis is South Africa’s request for WHO to “discuss the role of the public sector and charities in funding research for new cell and gene therapies, and measures to promote more transparency of the licensing of intellectual property rights from public sector research, and concrete measures in licenses to address the objective of universal access.” (Source: South Africa’s request to EB 146).
In reporting back on the implementation of the WHO cancer resolution, the Secretariat paper notes the following developments: 1) publication of WHO’s Technical report on Pricing of cancer medicines and its impacts, 2) WHO’s launch of a pilot program for the prequalification of biosimilars to treat cancer, 3) the publication of guidance for the WHO Model List of Essential In Vitro Diagnostics, which contains tests for cancer, and 4) WHO technical guidance and specifications for medical devices to screen and treat precancerous lesions in the prevention of cervical cancer.
In terms of policy recommendations moving forward, the secretariat paper does not meet the brief set out by Peru and South Africa. In Peru’s September 2019 submission on rare and orphan diseases, Peru raised the following concerns.
The growing number of rare diseases awaiting treatment is an important public health issue because they require medicines which have also been termed “orphans”. This is because the pharmaceutical industry has little interest in developing and marketing products intended solely for a small number of patients with very rare medical conditions. A good medicine for a patient with a rare disease is an affordable medicine that is available in the country where they live.
Because these medicines are so costly, the situation around accessibility is getting worse. Even without a uniform definition, their characteristics are determined by a range of elements and procuring these medicines represents a significant charge on the public health financing system and/or out-of-pocket expenditure by families and individuals.
Therefore, with a view to treating patients with rare or orphan diseases in our countries, there is a need to establish mechanisms to register unavailable medicines and promote them, thereby giving visibility to the supply and marketing of medicines for the treatment of orphan diseases and guaranteeing their availability at health facilities.
While the WHO board paper did address regulatory systems strengthening, it does not respond to Peru’s specific request for guidance to “establish mechanisms to register unavailable medicines and promote them, thereby giving visibility to the supply and marketing of medicines for the treatment of orphan diseases and guaranteeing their availability at health facilities”. (Source: Peru’s request to EB 146).
In relation to cell and gene therapies, South Africa asked the WHO Secretariat to:
invite a discussion of the development of and access to new cell and gene therapy technologies, and the response of the global community to the apparent severe inequality of access to such technologies, and
discuss the role of the public sector and charities in funding research for new cell and gene therapies, and measures to promote more transparency of the licensing of intellectual property rights from public sector research, and concrete measures in licenses to address the objective of universal access.
The WHO secretariat board paper did not heed South Africa’s request.
Section B – IMPROVING THE TRANSPARENCY OF MARKETS FOR MEDICINES, VACCINES, AND OTHER HEALTH PRODUCTS
In reporting of countries’ implementation of the WHO transparency resolution, WHO observes:
Some countries have made political commitments to improve market transparency. The European Region plans to convene a meeting in 2021 for its Member States and relevant stakeholders to discuss approaches to improving transparency and affordability of high-cost innovative medicines. The Eastern Mediterranean Region is considering a proposal to establish mechanisms that improve collaboration and information exchange between countries on prices of medicines and vaccines. The Secretariat has raised awareness of this issue via the Pharmaceutical Pricing and Reimbursement Information network. It continues to explore the feasibility and potential value of an international data platform and forums for sharing of information on prices and pricing approaches.
The secretariat make a reference to work done in the WHO European Region and the Region of the Americas to “explore legislative barriers to transparency”.
Work is being done in the European Region and the Region of the Americas to explore the
legislative barriers to transparency, given the acknowledged impossibility of promoting price and
pricing transparency under the laws and (confidential) commercial agreements in many jurisdictions.
In light of the recent imbroglio involving the accidental leaking of COVID-19 prices paid by the European Union, the upcoming WHO Executive Board (18 January 2021 to 26 January 2021) discussions on price transparency may ignite some heated debate.
The WHO board paper asserted the that the evidence on the “potential impact of promoting the transparency of prices and pricing of pharmaceutical products from comparative studies remains limited.” (Source: EB148/9, paragraph 30).
In relation to the Fair Pricing Forum, WHO revealed that it is working with the Government of Argentina to prepare the agenda for the Fair Pricing Forum in 2021.
The 2021 Fair Pricing Forum will be an important milestone at which to evaluate lessons learned and to reaffirm the commitment of Member States and all relevant stakeholders to health product affordability and transparency of prices and costs. The Secretariat will continue to provide technical support and guidance to Member States in their efforts to achieve and monitor transparency of markets for health products.