Response to "Canadians Need Improved Access to Drugs for Rare Diseases, Not More Denial".

The comments provided by Rawson and Adams (2023) miss the mark of our articles (Sirrs et al. 2023a, 2023b). We agree that the patient perspective is critical and that patients with "rare diseases have a right to healthcare and have huge unmet needs …" (p. 7). However, we challenge Rawson and Adams' (2023) thesis that keeping drug prices higher in Canada than in most other countries would solve the problem of access to therapies for rare diseases that have no available treatment.

Expensive Drugs for Rare Diseases in Canada: What Value and at What Cost?

There has been explosive growth in the market for expensive drugs for rare diseases (EDRDs). Traditional standards of evidence are not achievable for rare diseases, so lower standards are applied. The price of these drugs is extremely high. This combination of lower standards and higher prices make EDRDs attractive to manufacturers. Legislation designed to incentivize drug development for rare diseases contains loopholes that drive prices up worldwide. Canada compounds those problems with a complex network of agencies that impede communication between those providing market authorization and those purchasing drugs. Drug pricing is not related to metrics like investment or value, but rather willingness to pay. Without high-quality evidence to assess value, we inadvertently prioritize patients with rare diseases over those with common diseases, creating conflict among ethical principles such as social utility, justice and the rule of rescue. Lack of transparency over what is being funded and for whom makes it hard to mitigate challenges through effective policy development. We review the evidentiary, economic and ethical issues around EDRDs and ways to move forward, including enhanced transparency and the development of high-quality evidence to ensure that we do not pay for drugs that do not work.

Expensive Drugs for Rare Diseases in Canada: Time for Action Everywhere and by Everyone.

Expensive drugs for rare diseases pose unique economic, evidentiary and ethical challenges, and these will continue to escalate unless steps are taken urgently to address these challenges. We propose concrete actions that all stakeholders (federal and provincial/territorial governments, patients, healthcare providers, the public and drug manufacturers) could take now as a first step toward enhancing sustainability in the use of innovative (albeit expensive) therapies within our publicly funded healthcare system.

Pharmacists' perceptions of their working conditions and the factors influencing this: Results from 5 Canadian provinces.

INTRODUCTION: Our previous study in British Columbia (BC) indicated that pharmacists have a poor perception of their working conditions. The objective of this study is to assess pharmacists' perceptions of their working conditions in 4 other Canadian provinces. METHODS: This was a cross-sectional study across Alberta, New Brunswick, Prince Edward Island and Newfoundland and Labrador, using a survey adapted from the Oregon Board of Pharmacy. Data collected previously from BC were also included in the analyses. The survey was emailed to all pharmacist registrants. Respondents were provided with 6 statements and asked to rate their agreement with them, using a 5-point Likert scale. Statements were framed such that agreement with them indicated good perception of working conditions. Logistic regression analyses were used to study the relationship between workplace factors on perception of working conditions. RESULTS: Pharmacists perceived their working conditions to be poor. Pharmacists indicated that they do not have time for break/lunch (48.3% of respondents), work in environments that are not conducive to safe and effective primary care (26.5%), are not satisfied with the amount of time they have to do their job (44.0%) and face shortage of staff (shortage of pharmacists: 33.7%, technicians: 36.4%, clerk staff: 30.3%). Significant factors associated with poor perception were workplace-imposed quotas, high prescription volume, working in chain pharmacies and long prescription wait times. CONCLUSION: A high percentage of Canadian pharmacists perceived their working conditions to be poor. Considering the patient-related consequences of pharmacists' poor working conditions and the system-related reasons identified behind it, we call for collaborative efforts to tackle this issue.

Factors associated with pharmacists' perceptions of their working conditions and safety and effectiveness of patient care.

BACKGROUND: In recent years, the role of pharmacists has changed, as have various provincial legislations, which now allow pharmacists to provide additional health services to patients. With these changes comes growing concern about how well the current pharmacy working environment is adapting and whether it may also be creating work-related stress that may contribute to potentially unsafe practices of patient care. METHODS: To characterize the current working conditions of pharmacists in British Columbia, an online survey was developed and distributed to all College of Pharmacists of BC (CPBC) registrants by email. The survey consisted of questions on pharmacists' demographics, practice setting and perceptions of workplace conditions. Responses were collected from October 1 to November 10, 2013. All data were summarized using descriptive statistics, and regression models were constructed to assess the association between various factors and pharmacists' self-reported working conditions. RESULTS: Twenty-three percent (1241/5300) of pharmacists registered with the CPBC responded, with 78% working in the community pharmacy setting (58% chain, 19% independent). Pharmacists mostly disagreed with the statements that they had enough time for breaks or lunches or to do their jobs, as well as enough staffing support. Pharmacists' perceptions of their workplace environment were negatively associated with workplace-imposed advanced service quotas (for medication reviews, immunizations and prescription adaptations); being employed at chain store pharmacies, compared to independent pharmacies or hospitals/long-term care settings; and higher prescription volume. DISCUSSION: Pharmacists working in chain community pharmacies who are required to meet monthly quotas for expanded services reported a substantial negative impact on their working conditions and perceived safety of patient care. Can Pharm J (Ott) 2016;149:xx-xx.

More the same than different: worldwide drug policy issues.

Pharmaceutical policies in Europe were described in the previous issue of the journal. These policies were described in 5 areas: regulation and access; pricing, price control, and cost; publicly administered drug insurance; patient advocacy; and research ethics. While the underlying policy issues are strikingly similar, each country has developed its own set of solutions to address them. This article provides insight into the Canadian drug policy environment by comparing and contrasting it with the European experience.