[Ip-health] EB 148: WHO board paper on expanding access to effective treatments of cancer and rare and orphan diseases including cell and gene-based therapies

Thiru Balasubramaniam thiru at keionline.org
Thu Dec 31 03:12:06 PST 2020


https://www.keionline.org/34868


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EB 148: WHO board paper on expanding access to effective treatments of
cancer and rare and orphan diseases including cell and gene-based therapies
Posted on December 21, 2020 <https://www.keionline.org/34868> by Thiru
<https://www.keionline.org/author/thiru>

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
<https://apps.who.int/gb/ebwha/pdf_files/EB148/B148_9-en.pdf>). 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 <https://www.keionline.org/32024>. 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)
<https://apps.who.int/gb/ebwha/pdf_files/WHA72/A72_R8-en.pdf>.


*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
<https://www.keionline.org/29578>, 2) WHO’s launch of a pilot program for
the prequalification of biosimilars to treat cancer
<https://www.who.int/news/item/04-05-2017-who-to-begin-pilot-prequalification-of-biosimilars-for-cancer-treatment>,
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
<https://www.politico.eu/article/belgian-secretary-of-state-accidentally-reveals-eu-vaccine-prices/>
paid
by the European Union
<https://www.politico.eu/article/belgian-secretary-of-state-accidentally-reveals-eu-vaccine-prices/>,
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.



-- 
Thiru Balasubramaniam
Geneva Representative
Knowledge Ecology International
41 22 791 6727
thiru at keionline.org


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