Primer to the Annual Rare Disease Week Celebration

Frequently Asked Questions (FAQs) on Rare Diseases and the Annual Rare Disease Week Celebration*

Rare but strong together

WHAT IS A RARE DISEASE?

A rare disease, otherwise called an orphan disorder is any health condition resulting from genetic defects that afflicts no more than 1 of every 20,000 individuals in the country. Rare disorders afflict babies in all socioeconomic levels. Besides the little information available about their nature, what makes dealing with rare disorders even harder is that they are long-standing, progressive, disabling and life threatening.

Filipino patients born with rare disorders (diseases) are “orphaned” by society. They suffer from social abandonment because of lack of existing network of support to aid them. Medical help is elusive under the conditions of the country’s health priority. The nature of their illness is hardly known due to lack of information and only a few medical professionals, particularly in the country, are aware of these disorders and know how to diagnose and address these conditions.

Pompe Disease, Gaucher Disease, Methymalonic Acidemia (MMA), Maple Syrup Urine Disease (MSUD), Fabry Disease, Galactosemia, and Mucopolysaccharidosis are some of the sixteen RARE or ORPHAN metabolic disorders, which afflict Filipino children. There are about 165 documented cases of these RARE or ORPHAN disorders in the Philippines.

WHY A RARE DISEASE DAY?

We constantly need to raise awareness on rare diseases among decision makers, health professionals and the general public.

Information is key to improving living conditions for rare disease patients; raising awareness is therefore one of our primary goals.

The International Rare Disease Day was first celebrated on 29 February 2008 and its annual celebration aims to:

• Strengthen the voice of patients as one

• Give hope and information to patients

• Bring stakeholders closer together

• Get equity in access to care treatment

• Coordinate policy actions in different countries.

WHAT IS PRESIDENTIAL PROCLAMATION NO. 1989?

Presidential Proclamation 1989 mandated the Philippine adaptation of the Rare Disease Day celebration after it was issued last February 8, 2010. The proclamation declared the 4th week of February as the National Rare Disease Week.

This week-long celebration enhances national information campaign that will instill public awareness about the nature and medical management of rare diseases and to generate full support for the special needs of children affected by rare disease from both public and private sectors.

WHY SUPPORT THE ENEACTMENT OF A RARE DISEASE ACT OF THE PHILIPPINES?

The RARE DISEASES ACT OF THE PHILIPPINES is a proposed legislation which provides the creation of the Rare Disease Program at the Department of Health. It will ensure:

• Provision for early and sustainable care for patients suffering from rare disease;
• Coordination of current activities of the DOH to provide patients and their families with better access to medical care, health information, and health products to treat their conditions;
• Support for public education and information campaigns and health professional training;
• Establishment and supervision of a system of research and development initiatives; and
• Coordination of resource generation efforts among relevant government agencies and private sector.

WHAT CAN WE DO?

Advocating for Rare Disease can simply include:

•  Spreading the word. You can promote awareness on these diseases.
• Collecting signatures. You can collect signatures from your family, friends, school and community in support of the enactment of a rare disease act.
• Persuading community leaders and youth organizations. Informing and engaging community leaders on this advocacy may open new doors on public support for rare disorders.

REFERENCES

Philippine Society for Orphan Disorders, Inc. (PSOD)

Internation Rare Disease Day Information Pack and Website. www.rarediseaseday.org

*Compiled by the Volunteer Youth Leaders for Health - Philippines. This 2012, VYLH-Philippines joins PSOD in the celebration of the 5th International Rare Disease Day and 3rd National Rare Disease Week with the  "Juan for Rare" campaign.