Question Period Note: HIGH COST DRUGS FOR RARE DISEASES

Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. There are generally few or no treatment options, which means any available treatment is in high demand and can command significant prices (e.g., $100K to upwards of $2M per patient per year). These high prices combined with the increasing prevalence and severity of rare diseases, pose significant challenges to patients, caregivers, and the health care system, including the sustainability of public and private drug plans that pay for these drugs

• What is the Government doing to improve access to drugs for rare diseases and address the high cost of these drugs for Canadians living with rare diseases?

• We recognize that for many Canadians who require prescription drugs to treat rare diseases, the cost of these medications can be astronomically high.

• To help Canadians get better access to effective treatments, we will work with provinces, territories and other partners to develop a national strategy for high cost drugs for rare diseases. This is an important step in working with provinces and territories on national pharmacare.

• Budget 2019 proposes to invest up to $1 billion over two years, starting in 2022-2023, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need.

• Our Government has also been working with key stakeholders to improve access by encouraging the development and availability
of drugs for rare diseases for Canadians. In 2018, 11 of the 40 new drugs Health Canada approved were drugs for rare diseases.

IF PRESSED ON THE SPECIAL ACCESS PROGRAMME…

• Our Government recognizes the importance of Canadians having access to the treatments they need.

• Health Canada's Special Access Programme considers requests for access to drugs that are unavailable for sale in Canada. There have been approximately 150 drugs accessed through the programme for the treatment of rare diseases.

IF PRESSED ON RESEARCH…

• Our Government recognizes that supporting research is another important aspect of addressing rare diseases. Through the Canadian Institutes of Health Research, Canada is playing an important role, both at the national and international levels, to tackle rare diseases.

• For example, Canada, through CIHR, is a founding member of the International Rare Diseases Research Consortium and is also engaged in E-Rare, the European Union’s main instrument for funding research in areas related to rare diseases.

CANADIANS LIVING WITH RARE DISEASES
• Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. They are often genetic conditions, with onset either at birth or early childhood.

• More than 7,000 rare diseases (cancer and non-cancer) have been identified to date. Despite the number of rare diseases, each disease affects a relatively small number of patients (e.g., less than 5 in 10,000; typically closer to 1 in 100,000).

• Patients generally have reduced quality of life and shortened life span, and are high-end users of the public health care system.

HIGH COST DRUGS FOR RARE DISEASES
• Patients with rare diseases generally have few treatment options, resulting in unmet clinical need. Accordingly, treatments are in high demand and can command high prices. The pharmaceutical treatments for rare diseases are often referred to as orphan drugs, or expensive drugs for rare diseases, or high cost drugs for rare diseases.

• High prices are often attributed to factors such as the high cost of research, limited number of patients, small market size, and lack of competitors.

• The Government of Canada is working with provinces and territories as an active member of the pan-Canadian Pharmaceutical Alliance (pCPA) to combine the governments’ collective buying power to negotiate lower prices on brand name drugs for all public plans, including high cost drugs for rare diseases.

• Additionally, in August 2019, the Government announced final amendments to the Patented Medicines Regulations. These amendments will give the Patented Medicine Prices Review Board (PMPRB), the tools it needs to protect Canadians from excessive prices and make patented medicines more affordable for all payers - public and private drug plans, as well as Canadians who pay out of pocket for their prescription drugs, including high cost drugs for rare diseases.

ACCESS TO HIGH COST DRUGS FOR RARE DISEASES
• Canadians have been able to gain access to drugs for rare diseases through participation in clinical trials, or as drugs are approved under Division 8 of the Food and Drug Regulations. In cases where criteria are met, Health Canada’s Special Access Programme (SAP) considers requests for access to drugs that are unavailable for sale in Canada. About 150 of the drugs accessed through the SAP are for the treatment of rare diseases.

INVESTMENT IN RESEARCH
• Through the Canadian Institutes of Health Research (CIHR), the Government is supporting research on rare diseases and has taken a leadership role by joining important international research initiatives, such as the International Rare Disease Research Consortium, which aims to accelerate medical breakthroughs for people affected by rare diseases and includes 58 organizations from 22 countries.

• As of 2017, this international Consortium has yielded 279 new medicinal products and therapies for rare diseases.

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