Rare diseases difficulties like isolation, absence of treatment, lack of access to treatment in hospitals during COVID-19 time and scarce information have generated an emotional discomfort to these patients and their families.

Rare diseases often encounter difficulties related to orientation and medical pathway to establish diagnosis and subsequently adequate follow-up, both medical and social. Although there isn't always an effective cure for rare diseases (active research is ongoing, encouraged by a policy which promoted orphan medicines), early and adequate care may improve the survival rates and the patients' quality of life.

At the beginning of COVID-19 pandemic we organized virtual meetings to assess the needs of the patients and the results of our questionnaires are published on: www.bolirareromania.ro.

Based on these results we have advocated for access to home care for eligible patients with rare diseases that have infusions in hospitals, several times per month and the inclusion of telemedicine among the reimbursed services for patients with rare diseases. National Health Insurance have issued the Order 715/2020 establishing the way these services might be available for our patients. The legislation for telemedicine was also approved: LAW for the provision of remote medical services in Romania.

Medical procedures that can be practiced through telemedicine are:
a. establishing a diagnosis;
b. monitoring patients at risk with the purpose of prevention or post-therapeutic monitoring;
c. interpretation of some results of medical investigations;
d. requesting another specialized opinion;
e. preparing a therapeutic decision;
f. issuing medical prescriptions;
g. monitoring the patients' health condition.

As regarding the feedback from National Health Insurance for organizing the home care for patients with rare disease we can mention:

If the attending physician considers that it is possible to administer specific infusion drugs at the patient’s residency (depending on the patient's clinical and biological condition, living conditions and level of education of his or her relatives, safety data on administration of the drug specified in the summary of product characteristics), with the support of the management of the health unit, the administration of specific treatment at the patient's home under the supervision of medical staff can be organized.

In Annexes 30A and 31A of the Order of the Minister of Health and of the President of the National Health Insurance Company no. 397/836/2018 regarding the approval of the Methodological Norms for the application in 2018 of the Government Decision no. 140/2018 for the approval of the service packages and the Standard Contract that regulates the conditions for providing medical assistance, medicines and medical devices within the social health insurance system for the years 2018-2019 with subsequent amendments and completions, are included the basic medical services for home health care, as well as the conditions for providing these services.

According to the normative act mentioned above, the administration of infusion treatments, apart from injections and infusions of human origin, can be ensured with the recommendation of the medical specialist from the hospital or outpatient clinic, within the home care services. As it follows:

Annex 31A
Article 1
(2) The recommendation for performing at home health care services is made by outpatient specialists, including general doctors and by hospital doctors at the discharge of the patient, doctors who are contracted by the health insurance companies. The recommendation for performing medical care services at home follows the model presented in annex no. 31 C.
(5) Specialist in outpatient clinics, including general physicians, recommend home health care only because of the medical consultation reported to the health insurance company.
(6) The recommendation regarding the need to perform medical care at home is recorded by the patient's attending physician in the discharge note, at his discharge, or in the outpatient consultation register amongst recommendations issued by general doctors / clinicians.

Article 2
(1) The list of health care services available at home is provided in annex no. 30A
(3) The duration for which a person with medical insurance may benefit from health care services at home is established by the doctor who made the recommendation, with the obligation to specify the rhythmicity / periodicity of services, recorded in the form "Recommendation for health care at home", but not more than 90 days of care / in the last 11 months in several stages (episodes of care). An episode of care is a maximum of 15 days of care, respectively a maximum of 30 days for medically justified situations. If, according to the recommendation for medical care at home, the care is not provided on consecutive days, when calculating the 90 days / in the last 11 months, only the days on which the care was provided are taken into account. Consequently, in the event that the attending physician considers that it is possible to administer specific infusion drugs at the patient's home, a home care recommendation may be issued to perform this service or, if there are no providers in an area, or the existing ones do not have the capacity to provide this service, with the support of the health unit the administration of the specific treatment under the supervision of the medical staff can be organized at home.
Given the above and taking into account the epidemiological context caused by the SARS-CoV-2 coronavirus infection, we specify that, in order to administer home treatment for lysosomal diseases, it is necessary to analyze the stages involving the administration of treatment at home by specialists in in the sense of establishing the responsibilities of those involved in the administration of the treatment, monitoring the administration at home, as well as the transport of the medication in perfect conditions of safety.
In this way we assure you of our full cooperation to solve your requests.