- Is there a legal definition of orphan drugs under your law?
- Are orphan drugs reimbursed in your jurisdictions?
- What is the process and what are the conditions for obtaining reimbursement?
- Is reimbursement temporary or permanent?
- Can an orphan drug which is not however listed in EMA orphan drug (i.e., it does not have an orphan designation) receive reimbursement?
- How many orphan drugs have received reimbursement in your jurisdiction from 2021 to date?
- How long does it take for an orphan drug to receive reimbursement?
- Any future developments?
1. Is there a legal definition of orphan drugs under your law?
Yes, orphan drugs are defined in Annex No. 1 to Ministry of Health Order No. 861/2014 as drugs “used for the treatment, prevention or diagnosis of conditions that affect up to five in 10,000 people in the EU or that are life-threatening, chronically debilitating or represent serious and chronic conditions of the body. In addition, for these diseases there is no satisfactory method of diagnosis, prevention or treatment authorised in the EU or, if such a method exists, the medicinal product is of significant benefit to those suffering from it.”
Romania follows the guidelines for identifying orphan medicinal products outlined in Regulations 141/2000/EC and 847/2000/EC, as well as the designation made by the Committee for Orphan Medicinal Products of the European Agency for the Evaluation of Medicinal Products in Article 3.
2. Are orphan drugs reimbursed in your jurisdictions?
Yes, in Romania orphan drugs can be reimbursed using the same procedure as other medicines. There is no special procedure specifically for orphan drugs.
3. What is the process and what are the conditions for obtaining reimbursement?
The list of compensated and free medicines attached to Government Decision No. 720/2008, as amended, is approved for medicines that have been granted marketing authorisation in Romania for approved indications, based on the score obtained during the evaluation of the documentation submitted to the National Agency for Medicines and Medical Devices of Romania.
According to Health Reform Law No. 95/2006, to obtain a marketing authorisation for a medicinal product, an application must be submitted to the National Agency for Medicines and Medical Devices of Romania, along with certain required details and documents, in accordance with the approved analytical, pharmaco-toxicological, and clinical rules and protocols for testing medicinal products.
Government Decision No. 720/2008 regulates which drugs are eligible for reimbursement, and it is updated periodically.
Ministry of Health Order No. 1004/2022 amends and supplements the relevant secondary legislation that approves therapeutic protocols for prescribing medicinal products with international non-proprietary names and therapeutic protocols for prescribing medicinal products for specific conditions or groups of conditions listed in the international non-proprietary name list corresponding to the medicinal products available to insured individuals, with or without personal contribution, by prescription under the social health insurance system.
According to the above-cited legislation, “therapeutic protocols are the basis for prescribing and monitoring the treatment of insured individuals with medicines based on prescriptions issued by doctors who have a contract with health insurance companies.”
Further, according to Article 2 of Government Decision No. 720/2008, the percentage of reimbursement for medicines can be 100%, 90%, 50%, or 20% of the reference price.
The supplier of medicinal products is required to submit a request to the health insurance company with which they have a contract for the reimbursement of payments.
The application must comply with the relevant provisions outlining the method of settlement of prescriptions for medicines covered by cost-volume-outcome contracts issued and registered in the national electronic prescribing system that have benefited patients who, after assessment, have recorded a result.
The health insurance company must settle the invoice submitted by the medicinal product supplier within 60 calendar days of its validation and the granting of the “good to pay” visa, within the limits of the funds approved for this purpose.
4. Is reimbursement temporary or permanent?
Whether the reimbursement is temporary or permanent depends on the updated list of compensated drugs, as the list of compensated drugs in Romania is periodically updated by the competent authorities.
5. Can an orphan drug which is not however listed in EMA orphan drug (i.e., it does not have an orphan designation) receive reimbursement?
Yes, an orphan drug can be reimbursed in Romania if it is included on the nationally approved list.
Government Ordinance No. 720/2008 approves the list of international non-proprietary names of medicinal products that are available to insured individuals, with or without a personal contribution, by prescription under the social health insurance system, as well as the international non-proprietary names of medicinal products that will be provided under national health programmes. This list is updated periodically based on the applicable needs.
6. How many orphan drugs have received reimbursement in your jurisdiction from 2021 to date?
We could not find official information in this regard. However, for context, note that according to the latest data from the “W.A.I.T. Study” 2021, out of the 160 innovative medicines that were approved by the European Medicines Agency for the period 2017-2020, only 38 had been included in the list of compensated and free medicines in Romania by January 1, 2022.
7. How long does it take for an orphan drug to receive reimbursement?
According to the EFPIA (European Federation of Pharmaceutical Industries and Associations Patients) W.A.I.T. Indicator 2021 Survey, the time it takes for a new medicine to receive marketing approval from the European Medicines Agency and be made available on the Romanian market under the compensation system is about 30 months (899 days), an increase from the previous report (883 days).
The main reasons for the delays seem to be the waiting time for the submission of the reimbursement file and the lack of clear deadlines for the development and publication of the List of Reimbursed Medicinal Products and Therapeutic Protocols.
8. Any future developments?
According to a recent press release, Romania's representatives in Brussels emphasised the importance of being aware of the dossiers being discussed at the European level that will subsequently have an impact on national legislation.
The National Alliance for Rare Diseases Romania stated that it is actively involved at the European level and has reviewed the main patient objectives for the upcoming European legislation. These objectives include:
- maintaining the prevalence framework to ensure that no disease is left behind, while also including an incidence threshold;
- encouraging early dialogue between all stakeholders to respond to patients' needs, including the need for national rare disease registries;
- developing an orphan drug development plan to guide the development of new treatments;
- modulating incentives judiciously for diseases that have no other therapeutic solutions, without a restrictive definition of unmet medical need criteria;
- accelerating approval for transformative or potentially curative medicines in areas of unmet need.
Some Romanian associations have stated that creating a registry for rare diseases is a challenge.
While it seems that so far everything has been based on estimates, the Health Ministry reiterated its support for the next steps and hopes that steps will be taken to ensure that patients have access as close as possible to European standards.
The same press release stated that a series of proposals to improve access were launched as part of the Health Innovation Pact, a platform for collaboration with policymakers and all stakeholders in health.
