Orphan Drugs & Rare Diseases
Want to know more about orphan drugs and rare diseases in Chile? Read on! Prepared in association with Carey, a leading global law firm, this is an extract from The Pharma Legal Handbook: Chile, available to purchase here for USD 99.
1. What is the definition of Rare Diseases in your country?
Currently, we don’t have a legal or regulatory definition for rare diseases in Chile. Indeed, neither our Sanitary Code nor Supreme Decree No. 03/2010 (S.D. No. 3/10), which sets forth our pharmaceutical products regulations contemplate a definition for rare diseases. Additionally, although mentioned in Law No. 20.850 which sets forth a High Cost Treatment Financial Protection System (commonly known as Ricarte Soto Law) rare diseases are not defined.
Nevertheless, Exempt Resolution No. 411 of February 2015 (Res. No. 411/2015) issued by the Public Health Institute (ISP) – which approves recommendations for the sanitary registration (Marketing Authorization – MA) of orphan drugs– does include a definition of rare disease as follows:
“Uncommon, minority, rare or orphan disease: one with prevalence less than 5 cases per 10,000 inhabitants”.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
We have no specific legal or regulatory definition for orphan drug and S.D. No. 3/10 does not contemplate a definition or concept for the same.
Nevertheless, Res. No. 411/2015 does include a definition for orphan drug which is related or linked to the concept of rare disease set forth in that same resolution, as follows:
“Orphan pharmaceutical product or orphan drug: that medicine intended for the diagnosis, prevention or treatment of a rare disease or a condition whose aetiology has an equivalent frequency”.
In this regard, the ISP does not provide for a specific orphan drug designation process, but a pharmaceutical product could be considered as an orphan drug by meeting this concept.
3. What is the regulatory framework for the authorization of an Orphan Drug? (Is this regulatory framework based on Rare Disease status or can it alternatively be based on Orphan Drug foreign status?)
S.D. No. 3/10 does not contemplate a specific procedure for the authorization of an Orphan Drug.
Considering the above mentioned, the ISP on 2015 issued Res. No. 411/2015, including recommendations for obtaining the approval of an Orphan Drug. However, these recommendations indicate that the request must be made, among other requirements, through the abbreviated registration procedure – which, in case of an Orphan Drug should not last more than 4 months to obtain the registration–, providing evidence that the product is an orphan drug as certified by sanitary agencies such as the FDA or EMA and, additionally, requiring a prior resolution from the Ministry of Health (MoH) to that effect.
Unfortunately, to date there is no clarity on how to carry out this process before the Ministry of Health or how long this process will take in order for the MoH to issue such resolution. Therefore, Res. No. 411/2015 has not been as useful as intended, in the sense that it does not effectively guarantee a shorter term of approval of the registration under an abbreviated procedure.
Consequently, in case that an Orphan Drug registration is required, a good approach is to discuss it directly with the ISP, on a case by case basis.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Our regulations do not contemplate provisions for relaxed clinical trial/scientific evidence in respect of Orphan Drugs. Therefore, in principle, full clinical trial/scientific evidence must be submitted before ISP in order to obtain the sanitary registration of an Orphan Drug.
Nonetheless, Res. No. 411/2015, within its recommendations, indicates that as Orphan Drugs are unlikely to have phase III clinical trials, with a large number of patients, to evidence safety and efficacy as they are destined for the prevention, diagnosis or treatment of low prevalence or frequency diseases (both in their expression and agents). In this regard, the applicant may submit safety and efficacy clinical studies with a reduced number of patients, notwithstanding that the design and the risk/benefit profile will be evaluated and supported by the preclinical and clinical information available.
5. Is there an expedited pathway for Orphan Drugs?
Please see question 3 above.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
No, any Orphan Drug intended to be commercialized in the Chilean territory must apply for a sanitary registration and must comply with the local requirements established in our regulations, regardless the fact that said product may have a registration in other jurisdiction.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
In Chile, there is no general regulatory reimbursement system or process.
In this regard, orphan drugs will have coverage within the functioning of the Chilean Healthcare system.
The Chilean healthcare system is primarily structured by a mandatory medical coverage which is required by law. This coverage is financed by health insurance contributions paid to the providers of healthcare insurance (FONASA or the ISAPREs) on a monthly basis by certain persons such as employees (in which case the relevant employers are legally bound to withhold the relevant amounts from employees’ monthly wages), independent workers, pensioners, etc. The law provides for a minimum medical coverage and the additional features depend on the health institution and the health plan chosen by each individual.
Additionally, there are two universal coverage programs, which are Explicit Guarantees in Health (GES plan) and the High Cost Treatment Financial Protection System (provided by Law No. 2.8580, Ley Ricarte Soto), which cover specific treatments and medicines. Certain orphan drugs are included within the specific coverage provided by GES but main coverage for certain specific orphan drugs have been provided under Law No. 20.850.
Isolated coverage has also been provided under the Extraordinary Auxiliary Fund administered of the MoH.
For further information, please see answers to queries No. 10 and 11 of Exhibit A.
8. How are the prices of Orphan Drugs regulated?
Up-to-date, there is no price regulation for medicines in Chile. The owner of the product can determine the sale price of a medicine, such as Orphan Drugs.
9. In case of reference price based on a basket of countries, what countries are included?
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
In Chile judicial activity for coverage of pharmaceutical products has not been historically high, but there have been increasing numbers of judicial cases brought for high cost medicines in the last years, including orphan drugs, mainly throughout constitutional protection actions.
11. Are there proposals for reform or significant change to the regulation of Orphan Drugs? If yes, when are they likely to come into force?
There have been a few bills of law which were subject to discussion in Congress for rare diseases and orphan drugs in Chile but with no active development. Bill of law No. 7826-11 was submitted for discussion in August of 2011 and includes provisions on designation of orphan drugs, market exclusivity for orphan drugs and registration of patients and patient associations and protection of data privacy, among other matters. The bill has not had any movement since its submission.
Bill of Law No. 7643-11 was submitted for discussion in May of 2011 and it has similar provisions as Bill of Law No. 7826-11. Proceedings for this bill of law were recently reopened in July of 2019, but with no further discussion.
Finally, the bill of law for Medicines II (bill of law No 9914-11) which is in its final discussion phase in Congress, includes a provision allowing pharmacies, wholesalers and pharmaceutical laboratories to import raw material necessary for the treatment of rare or low prevalence diseases considered as orphan drugs by the local or international authorities, duly recognized as stringent regulatory authorities.
Nevertheless, this provision has been recommended for its exclusion in the final discussion in Congress.