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.

Diversifying stem cell debates: Including Muslim contexts and perspectives.

Greater transcultural and transdisciplinary engagement within Muslim contexts and deliberate inclusion of diverse Muslim voices in the development of international guidelines is required to improve understanding of the state of stem cell science, strengthen thinking about attendant ethical complexities, enhance compliance, deepen public deliberation, increase trust, and strengthen practice standards.

Can multi-criteria decision analysis (MCDA) be implemented into real-world drug decision-making processes? A Canadian provincial experience.

OBJECTIVE: To describe the implementation of multi-criteria decision analysis (MCDA) into a Canadian public drug reimbursement decision-making process, identifying the aspects of the MCDA approach, and the context that promoted uptake. METHODS: Narrative summary of case study describing the how, when, and why of implementing MCDA. RESULTS: Faced with a fixed budget, a pipeline of expensive but potentially valuable drugs, and potential delays to drug decision making, the Ministry of Health (i.e., decision makers) and its independent expert advisory committee (IAB) sought alternative values-based decision processes. MCDA was considered highly compatible with current processes, but the ability as a stand-alone intervention to address issues of opportunity cost was unclear. The IAB nevertheless collaboratively voted to implement an externally developed MCDA with support from decision makers. After several months of engagement and piloting, implementation was rapid and leveraged strong pre-existing formal and informal communication networks. The IAB as a whole rates new submissions which serves as an input into the deliberative process. CONCLUSIONS: MCDA can be a highly adaptable approach that can be implemented into a functioning drug reimbursement setting when facilitated by (i) a truly limited budget; (ii) a shared vision for change by end-users and decision makers; (iii) using pre-existing deliberative processes; and (iv) viewing the approach as a decision framework rather than the decision (when appropriate). Given the current limitations of MCDA, implementing an academically imperfect tool first and evaluating later reflects a practical solution to real-time fiscal constraints and impending delays to drug approvals that may be faced by decision makers.

Ethics and the Law.

Health care providers' interpretation of law can have intended and unintended effects on health care delivery in Canada. At times, health care providers encounter situations where they perceive the law to conflict with their sense of what is most ethically justified. In many cases, these health care providers feel especially torn because they assume that the legal requirements must dictate the decision, and cannot be explored or questioned. We challenge this assumption: the law is not as cut-and-dried as some assume; therefore, its significance to health care decisions should be carefully considered. Within a systematic ethics process, legal considerations can be a source of values and information and can create opportunities for further dialogue. This approach is justified because it appropriately reflects the relationship of the law to ethics. This way of thinking about the law and ethics also avoids potentially harmful consequences of legalistic approaches to decision-making, such as breakdowns in communication, adversarial relationships, and a reduction of ethically complex decisions to simple rule following.

Ethically justified decisions.

Good Decisions is a framework that assists healthcare leaders to make ethically justified system-level decisions. This article describes some of the features that make a decision ethically justified and discusses the experience of its use in one Canadian health authority. The framework sees the membership and relationships of the decision team, the quality of analysis, the breadth of consultation, and the implementation of and follow up on decisions, as all impacting a decision's ethical justification.

Des décisions justifiées sur le plan éthique.

Good Decisions est un cadre qui aide les leaders en santé à prendre des décisions systémiques justifiées sur le plan éthique. Le présent article décrit quelques-unes des caractéristiques qui rendent une décision justifiée sur le plan éthique et expose l'expérience de son utilisation dans une régie régionale de la santé canadienne. Le cadre tient compte des membres et des relations de l'équipe de décision, de la qualité de l'analyse, de l'étendue de la consultation ainsi que de l'adoption et du suivi des décisions, qui ont tous des effets sur la justification éthique d'une décision.

Exploring disparities between global HIV/AIDS funding and recent tsunami relief efforts: an ethical analysis.

OBJECTIVE: To contrast relief efforts for the 26 December 2004 tsunami with current global HIV/AIDS relief efforts and analyse possible reasons for the disparity. METHODS: Literature review and ethical analysis. RESULTS: Just over 273,000 people died in the tsunami, resulting in relief efforts of more than US$10 bn, which is sufficient to achieve the United Nation's long-term recovery plan for South East Asia. In contrast, 14 times more people died from HIV/AIDS in 2004, with UNAIDS predicting a US$8 bn funding gap for HIV/AIDS in developing nations between now and 2007. This disparity raises two important ethical questions. First, what is it that motivates a more empathic response to the victims of the tsunami than to those affected by HIV/AIDS? Second, is there a morally relevant difference between the two tragedies that justifies the difference in the international response? The principle of justice requires that two cases similarly situated be treated similarly. For the difference in the international response to the tsunami and HIV/AIDS to be justified, the tragedies have to be shown to be dissimilar in some relevant respect. Are the tragedies of the tsunami disaster and the HIV/AIDS pandemic sufficiently different, in relevant respects, to justify the difference in scope of the response by the international community? CONCLUSION: We detected no morally relevant distinction between the tsunami and the HIV/AIDS pandemic that justifies the disparity. Therefore, we must conclude that the international response to HIV/AIDS violates the fundamental principles of justice and fairness.

Ethical and scientific issues surrounding solid organ transplantation in HIV-positive patients: Absence of evidence is not evidence of absence.

End-stage liver disease is emerging as a leading cause of death among HIV-positive patients. Historically, an HIV diagnosis was a contraindication for a liver transplant; however, because of the efficacy of highly active antiretroviral therapy (HAART), HIV-positive patients have one-year, two-year, and three-year post-transplantation survival rates similar to that of HIV-negative patients. Based on this evidence, HIV-positive patients are now considered eligible for transplantation. However, newly emerging guidelines include the stipulation that HIV-positive patients must be on HAART to be placed on a waiting list for transplantation. The purpose of the present paper is to evaluate the scientific and ethical probity of requiring HIV-positive patients to be on HAART as a condition for being on a liver transplant waiting list. It is argued that the emphasis should be placed on the probability of post-transplantation HAART tolerance, and that concerns about pretransplantation HAART tolerance are of secondary importance.