RDI Statement on COVID-19 response and recovery

July 2020

To ‘Build Back Better’, do not leave behind people livingwith a rare disease in COVID-19 response and recovery

What this global pandemic has shown is that“the virus does not discriminate, but its impacts do”
Antonio Guterres, United Nations Secretary General

Rare Diseases International (RDI) wishes to bring to the urgent attention of policy
makers and authorities the increased vulnerability that the 300 million people living witha rare disease (PLWRD) worldwide are facing during the COVID-19 pandemic. The impact
and recommendations outlined in this statement can be extended to the undiagnosed
population, a group within the rare disease community that is experiencing paralleledvulnerabilities, in addition to facing a diagnostic odyssey.
RDI commends the commitment and actions of health and social care professionals, aswell as policy makers from all over the world during the crisis. In addition, RDI calls forcontinued and reinforced efforts to: 1. Eliminate the risk of exacerbating preexisting inequalities; and 2. Protect people living with a rare disease from being
discriminated and becoming even more vulnerable in COVID-19 response andrecovery strategies, as well as in long-term health system restructuring plans.
The onset of the COVID-19 crisis has unveiled pre-existing health, social and economic
inequalities that prevail between and also within countries of the world and has
demonstrated the need to urgently address them. PLWRD are a vulnerable populationthat is disproportionately impacted considering the chronic, highly complex,
progressive and severely disabling nature of their diseases, which generate specificcare needs. As such, PLWRD need to be considered a high-priority high-risk
population during COVID-19 response and recovery.

RDI welcomes the efforts led to protect people living with non-communicable diseases(NCDs)1 and people living with all kinds of disabilities2 during the COVID-19 pandemic,
and wishes to highlight that PLWRD are often marginalised, underserved, and forgotten
even within these populations. With this statement, RDI urges authorities, NCDs anddisability advocates to acknowledge that PLWRD are an identifiable vulnerable
population having specific needs and concerns linked to rarity and requiring atargeted approach during COVID-19 response and recovery.
Member States have acknowledged the need to protect populations with pre-existing
conditions from COVID-193. In order to ‘build back better’ it is essential to remindthem of their universal health coverage (UHC) commitments, which go beyond
COVID-19 itself and include specific efforts to address rare diseases. Indeed, thisreflects the UN Political Declaration on UHC4 and is in line with the guiding principle to‘leave no one behind’ of the Sustainable Development Goals (SDGs) and earlier UN
Resolutions, such as WHA63.17 on Birth Defects5.

1. The impact of the COVID-19 pandemic on the global rare diseasecommunity
RDI and its members have collected the experiences of persons living with a rare disease
through surveys at the national and global levels. Despite differences in the extent of
impact depending on the country of residence - related to differing regulations,infrastructures and organisation of care, level of access to information and services,
organisation of civil society, and stage in the pandemic - results have demonstrated asimilar experience for this vulnerable community across the world.
The common global experience of the rare disease community is one of overall
disruption in access to care. During this crisis, people living with a rare disease haveexperienced limitations, delays - and at times denial – in access to testing, healthservices, surgeries, rehabilitation therapy, supportive care, and medicines.
It is important to note that if the collected data reveals such a negative impact, incountries where there is practically no support and infrastructures for PLWRD and wheresuch data collection is not possible, the impact of the pandemic is surely even worse.

1

Efforts led by this community have ensued in the creation of a WHO Working Group on COVID-19 and
NCDs which releases guidance and collects data, among other actions. Info here.
2
The International Disability Alliance (IDA) leads strong advocacy on COVID-19 strategies that take into
account the needs of persons with disabilities. In addition, the UN and WHO have released guidance on
disability-inclusive response (here and here).
3
World Health Assembly Resolution 73.1 on COVID-19 response, available here.
4
Political Declaration on Universal Health Coverage, reference to rare diseases under par. 34
5Sixty-third World Health Assembly WHA63.17 on Birth Defects, May 2010, available here.

Overall:•

PLWRD have experienced interruption in the care they usually receive
either by not being able to meet with their healthcare professionals, or by theircare being put on hold (9 out of 10 people according to a global survey6, 85%
in Colombia7, 74% in the USA8, 50% of respondents in Canada9)
In addition, a testimony from a rare disease organisation in the Asia Pacificregion10 reads “the usual centres for rare diseases have been embargoed for
COVID-19 response and rare disease patients have had to turn toovercrowded public hospitals, which leads to greater danger of infection”.

•

People have not been able to access appropriate testing in relation to theirdisease, which could prevent them from getting accurately diagnosed (6 in 10
people according to the global survey).
Linked to the issue of diagnosis, a testimony from an international federation
for a rare disease shows the impact that the COVID-19 can have on awarenessof rare diseases at the clinical level: “Opportunities for patients and clinicians
to network has been greatly affected, leaving patients less informed andempowered and fewer clinicians educated about a group of conditions thatfew clinicians know about”11.

6•

People have seen their appointments for rehabilitation therapies, such as
speech and physical therapies, postponed or cancelled – even when these
therapies represent the only available treatments (8 in 10 according to theglobal survey, 50% in Canada).

•

Not having access to infrastructures, healthcare workers and support services
has increased the caregiving burden on families who have also lost their support
from extended family and friends due to confinement measures. As aconsequence, carers have had to stop working or significantly reduce theirworking hours since the pandemic started (3 in 10, according to the global
survey), leading to increased pauperisation of families.

Rare Barometer Survey (June 2020) based on 8551 respondents (people living with a rare disease or theircarers) from all EU countries and beyond
7
According to the breakdown results of the Rare Barometer survey the impact was also high in other Latin
American countries: 95% in Uruguay, 94% in Ecuador, 89% in Peru, 85% in Mexico, 76% in Chile, and 75% in
Argentina
8
https://rarediseases.org/infographic-illustrates-impact-the-covid-19-pandemic-is-having-on-the-raredisease-community/
9
http://www.raredisorders.ca/content/uploads/Applying-Lessons-from-COVID-19-3.pdf
10 11
Collected through a survey carried out by RDI with all its member organisations on COVID-19 impact
and response•

PLWRD have also faced difficulties in accessing their medicines andtreatments as a number of countries have encountered supply difficulties(40% in Canada). For example, in patients with haemoglobinopathies, “thepandemic has secondary consequences, such as blood shortages”12 that may
have long-term effects on health status.

Also, a key feature of the rare disease community is the lack of effective treatments,
and therefore greater need to continue fostering research and innovation for rarediseases. The COVID-19 pandemic poses a threat to the continuation of clinical
trials and research on these diseases.
In addition, both confinement and deconfinement measures can have a severe
psychological impact on PLWRD and their carers/families due to increased
isolation and uncertainty, and to disruptions in access to mental health services(almost 6 in 10 have seen their psychiatry follow-up interrupted according to the global
survey). Under normal circumstances, being affected by a rare disease has a strong
impact on mental health, and the COVID-19 crisis has exacerbated this.
Furthermore, PLWRD are particularly vulnerable to negative economic
consequences ensuing from the crisis’ impact on the labour market and the economy
overall, such as lay-offs and reduction in income, loss of benefits due to loss of
employment, etc. A rare disease organisation from South Africa declared that “Somepeople living with a rare disease have lost their medical insurance cover due tothe COVID-19 pandemic”13. This could result in further difficulties to access the careand services they need for their health and a life in dignity.
PLWRD are concerned that worsening finances in individual countries will lead to longterm consequences in terms of withdrawal or non-provision of health and social care
support for them.

2. The added value of organisations of PLWRD to the global response
to COVID-19
Civil society has proven to play an essential role in identifying and meeting the needs
arising from the COVID-19 pandemic. Organisations of PLWRD worldwide havebeen no less in these efforts and should be seen as key partners in the global
response to the crisis.
It is important to understand that organisations of PLWRD emerged as a result ofexperience gained by patients and their families from being excluded from health caresystems and having to manage their own disease themselves due to lack of knowledge
12

Farmakis, D., Giakoumis, A., Cannon, L., Angastiniotis, M. and Eleftheriou, A., 2020. COVID‐19 And
Thalassaemia: A Position Statement of The Thalassaemia International Federation.
13

Collected through a survey led by RDI with all its member organisations on COVID-19 impact andresponseand attention by the scientific community and competent authorities 14. They also stemfrom the need to feel connected to other individuals and families experiencing thesame issues. These organisations often gather, produce and disseminate limited
existing information on a disease, they provide support services to PLWRD and they
advocate on their behalf for better lives.
This supportive role has become essential during the COVID-19 crisis. RDI member
organisations around the world have taken different actions, including:
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Developing surveys to identify the impacts of COVID-19 and the needs of theirconstituents;
Releasing public statements to raise awareness on the situation of theirmembers and reaching out to national authorities about the specific needs of
PLWRD;Developing information resources catered and adapted to the needs of theirconstituents and offering forums for discussion where PLWRD can voice theirconcerns and exchange good practices;
Assisting patients to continue to access treatment and care, for example throughthe creation of Special Emergency Funds;
Offering psychosocial support to patients and families;
Ensuring PLWRD have access to protective measures (masks, hand sanitizer, etc);
Helping local, national or regional authorities develop a response to the crisis
that takes into account the specific needs of PLWRD.

In this context, RDI calls on policy makers to engage with and support
organisations of PLWRD. These organisations are connected to their grass-rootscommunities and can back the healthcare and social services by ensuring
information flow, good communication and peer support. ‘Build back better’ will
require solutions that are co-created with patient organisations.

3. Recommendations to protect the rare disease community during
COVID-19 response and recovery plans
COVID-19 has reinforced the call for equity embedded within recent international UHC
agreements and has emphasized the need for explicit strategies to reach and protect
vulnerable groups15. Ensuring equity in access to care directly contributes to thefulfilment of everyone’s right to health, including that of PLWRD, while it helps preventthe spread of the infection, ultimately resulting in better health outcomes for all and

14

Aymé, Ségolène & Kole, Anna & Groft, Stephen. (2008). Empowerment of patients: Lessons from the rarediseases community. Lancet. 371. 2048-51. 10.1016/S0140-6736(08)60875-2.
15
UHC 2030 Discussion paper on health emergencies and UHC (May 2020), available here

economic prosperity and security16. It is essential to integrate a human rights-based
approach in the response to COVID-1917.
For equity to work in practice and protect members of the rare disease community
during COVID-19 response and recovery plans, RDI calls on policy makers to implement
the following measures in accordance to the stage of the pandemic in each country:

16•

Recognise PLWRD as a high-priority high-risk population in need oftargeted policies in the monitoring of and response to the COVID-19 pandemic.

•

Safeguard the continuity of care and access to medical consultation for
PLWRD. A key action is to direct funds and efforts towards reinforcing medical
workforce and equipment. Having strong health care systems, with sufficient
capacity to address the needs of all, would prevent discrimination and the needto resort to triage in clinical practice.

•

Guarantee access to testing and personal protective equipment (PPE) for
PLWRD as part of the vulnerable high-risk population group.

•

Ensure the adoption of concrete measures/protocols warranted by thecomplex needs of rare disease patients in the provision of emergency
healthcare during the COVID-19 crisis. This includes contacting the patient’s
permanent medical practitioner and/or expert centre, involving patients andcarers in decision-making, and considering the creation of temporary special
hospital wards for particularly vulnerable rare disease patients.

•

Vulnerable populations, including PLWRD, should be granted priority
access to vaccine or other prophylactic treatment if their health condition
allows for such vaccination.

•

Access to medication to treat COVID-19 should also be ensured forvulnerable populations, including PLWRD.

•

Governments should actively take measures to protect the most vulnerable
groups in the population, and not only leave measures to the discretion ofindividual service providers and employers. Clear non-discriminatory
guidelines18 that prioritise access to healthcare, social care, opening of schools
and educational facilities and resource centres are needed both duringconfinement periods and as these measures are progressively lifted.

UHC2030 Statement on COVID-19 and UHC (26th March 2020), available here
WHO Publication 21 April 2020: Addressing Human Rights as Key to the COVID-19 Response, available
here
18
Refer to WHO Disability considerations during the COVID-19 outbreak, published 26 March 2020,
available here
17

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Schools, educational facilities and resource centres should be prioritised
when relaxing confinement measures, as they are essential for both
PLWRD and their family and carers. These service providers should be
supported to ensure that they have the necessary flexibility to assess individual
situations and adopt adequate measures for PLWRD. The guiding principle in allpolicies put in place should be “assisting those who need intensive support tolive independently”19.

•

Parents and caregivers of PLWRD (adult or child) should be waived fromrequirements to return to the office if/when their jobs allow for remote
working - as exposure to the virus puts them and their loved ones atgreater unnecessary risk. When the nature of a job does not allow for homeworking, adjustments to the workplace or flexible working arrangements should
be made to prevent people putting themselves and their families at risk. These
guidelines should be clearly communicated to the public.

•

Facilitate the use of online consultations when people are not able tophysically access healthcare facilities because of logistical/practical reasonsor fear. Access to an internet connection / other digital resources is not universal,
therefore alternatives or support to access them need to be ensured. Necessary
clinical assurance and data protection safeguards should also be guaranteed.

•

Ensure and facilitate the continuation of research on rare diseases by, forexample, disseminating and following-up on the guidance issued on clinical
trials during the COVID-19 pandemic.

RDI and its members call upon the whole society to uphold the principles ofsolidarity, equity and cooperation in efforts to ‘build back better’.
All citizens must play their role in protecting the general population and the mostvulnerable ones, in particular by adhering to the guidelines and minimising the risk ofinfection.
It is also the time to invest in people and prioritise strong, well-funded, public healthand welfare systems that also address the needs of the most vulnerable, and tocoordinate and collaborate in public health policy across borders.
The rare disease community stands ready to co-create this brighter future.

19

In line with article 19 (Living independently and being included in the community) of the Convention on
the Rights of Persons with Disabilities.