Regulation and access to orphan drugs in Peru: challenges and opportunities

Maritza Reátegui Valdiviezo

Rodrigo, Elías & Medrano, Lima-Perú

mreategui@estudiorodrigo.com

Cecilia Alarcón Galarza

Rodrigo, Elías & Medrano, Lima-Perú

calarcon@estudiorodrigo.com

Access to orphan drugs represents one of the greatest challenges for health systems worldwide. These medications, designed to treat rare or uncommon diseases, are essential for thousands of patients whose therapeutic options are often limited or non-existent. However, their development, regulation, and availability are marked by significant barriers, ranging from high research and production costs to regulatory challenges.

Law No 29698, enacted in 2011, was a major milestone in Peru, declaring the care and treatment of individuals with rare or orphan diseases a matter of national and priority interest. Over the years, this legal framework has been strengthened through regulations, resolutions and, more recently, Law No 31738 (enacted in 2023).

Challenges persist despite these advances, but opportunities have also emerged to improve access to orphan drugs, which are essential to the quality of life of those who need them.

The Peruvian regulatory framework

Law No 29698 laid the foundation for a legal framework that acknowledges the priority attention required for rare or orphan diseases in Peru. This law defines these diseases as those that:

• are life-threatening or cause chronic disability;
• have low prevalence;
• present significant challenges for diagnosis and follow-up;
• are often of unknown origin; and
• entail multiple social problems with limited epidemiological data.

Since its enactment, the country has made progress in the associated regulations, developing administrative directives and guidelines aimed at effectively implementing the law.

One of the significant milestones was the approval of the law’s regulation through Supreme Decree No 004-2019, which aimed to establish technical and regulatory provisions for the implementation of Law No 29698. In parallel, sectoral commissions with specific objectives were created, including:

• the development of an official list of rare or orphan diseases;
• the drafting of a national plan for prevention, diagnosis and treatment; and
• the establishment of criteria to identify high-cost drugs associated with these diseases.

More recently, with the enactment of Law No 31738, a broader approach has been introduced, incorporating research, education and innovative mechanisms to guarantee treatments. Additionally, this law reinforces the right to comprehensive care, simplifies administrative processes, and ensures greater budgetary commitment.

Advances in implementation

Over the last decade, Peru has achieved significant progress in addressing rare or orphan diseases. The institutionalisation of the annual National Rare or Orphan Diseases Day, celebrated on 28 February, has raised the visibility of this issue, increasing awareness among the public and authorities.

The measures introduced by Law No 31738, such as the obligation to initiate treatment as soon as possible after diagnosis and the simplification of the sanitary registration process for drugs approved in countries with high regulatory oversight, are steps aimed at improving access to orphan drugs.

Despite these achievements, challenges remain in accessing orphan drugs in Peru. Limited diagnostic capacity hampers the timely identification and treatment of patients.[1] Moreover, the high costs associated with orphan drugs represent another critical obstacle.

Conclusion

Access to orphan drugs in Peru remains a challenge, but regulatory advances offer an encouraging outlook. Laws No 29698 and 31738 have laid the groundwork for more equitable and comprehensive care, and their effective implementation could significantly impact the lives of patients with rare or orphan diseases.

Prioritising technological innovation, fostering research and strengthening the health system will be essential to overcoming existing barriers. With determination and commitment, Peru has the opportunity to demonstrate that challenges can be transformed into opportunities to build a more inclusive health system.