Rapid Patient Access to Innovative Medicines – An Awaited Policy Change: Rawson and Adams for Inside Policy

By Nigel Rawson and John Adams, March 24, 2022

Much has been written in recent years about the risk of patient access to new drugs, prompted by federal government plans to introduce new regulations for the Patented Medicine Prices Review Board (PMPRB ).

The PMPRB’s intended role is to prevent the abuse of time-limited drug patents. The government wants to replace countries with higher drug prices with those with lower prices in the PMPRB’s international price comparison, use health technology assessments to control prices (although they are not not intended for this purpose), imposing a price reduction if annual sales of a drug in Canada exceed a certain amount and requiring pharmaceutical companies to disclose confidential information about discounts negotiated with their customers.

These changes would make the PMPRB a profit regulator, forcing drug developers to cut prices to unsustainable levels. Legal challenges to the revised settlement and abuse of power have so far resulted in three cases in which eight of the nine judges (including six appeal court judges) ruled that something was wrong. at all in the regulations and how the PMPRB uses its powers; another decision from the Court of Appeal is awaited.

These rulings are likely why the government has delayed implementation of the regulations for a fourth time until July 2022. Instead of appealing to the Supreme Court of Canada, the federal government should limit its proposed amendments to modification of the international comparison of prices, which could provide an acceptable compromise for the biopharmaceutical industry.

Drug developers already face a daunting challenge of legal hurdles, health technology assessments conducted by the Canadian Agency for Drugs and Technologies in Health (CADTH), and price negotiations with the Pharmaceutical Alliance pan-Canadian (CPA). CADTH and APC are non-accountable, non-transparent organizations managed and funded by federal, provincial and territorial governments, and both have been harmonizing their processes for some years. Yet the alignment seems less about improving health care and more about rationing budgets by delaying or denying the introduction of new drugs in Canada, postponing their impact on government drug plans. .

Canada’s attractiveness as a market for drugs will further diminish if dramatically reduced prices are regulated. The impact will not only be felt by patients who want access to expensive drugs for rare diseases, but also by those who need drugs for common diseases. CADTH-pCPA processes already ensure that only about half of new drugs approved in Canada have government reimbursement coverage. In addition, when drugs are launched, the average time between marketing authorization and the price agreement negotiated by the pCPA is around two years.

The time was nearly 10 years for Kuvan, the first drug for phenylketonuria (PKU), a rare inherited condition that causes a buildup of amino acids that can lead to impaired brain function. PKU can be treated with inexpensive supplements, covered by provincial programs, as part of a low-protein diet that is unappetizing, difficult to maintain, and rarely provides optimal brain function. However, Kuvan offers a cheaper and more effective alternative for sufferers, but is covered with many restrictions by only four provincial drug plans. Although Kuvan is also accessible through a patient support program, new patients are no longer accepted.

Meanwhile, a more effective PKU drug for some patients, Palynziq, was approved in Europe and the United States nearly three and four years ago, respectively, but was only submitted in Canada in June. 2021 and not yet available. We understand that no Canadian patient support program will be provided for Palynziq, likely due to the bureaucratic experience with Kuvan and most provinces not covering it after 10 years.

The experience with Kuvan and Palynziq is a harbinger of what Canadians can expect if the PMPRB’s revised regulations are implemented as intended. If new drugs are introduced in Canada, their launch will be significantly delayed, and when their prices are significantly reduced by regulation, developers will be unwilling to negotiate discounts with provincial and private insurers. Patient support programs are unlikely to be available. The only Canadians who stand to gain would be those who don’t have drug coverage and who pay full price for their drugs, and they won’t benefit if the manufacturer decides the Canadian market isn’t worth it. . Delayed/denied access to cutting-edge new medicines will hurt Canadians.

Patients with diseases such as cancer, amyotrophic lateral sclerosis, cystic fibrosis, sickle cell disease and other life-threatening degenerative diseases do not have time to wait for delays in accessing drugs that could help them. Denying these patients access to meaningful therapeutic developments is unethical.

COVID-19 has demonstrated that Canada’s healthcare system is not working, despite having one of the highest healthcare spending rates among comparable high-income countries. Canada’s health care system is siloed, fragmented, underfunded and understaffed, leading to the rationing of services like specialty care, medical technologies and drugs. Canadians need a system that can meet future challenges without interrupting essential services and without adding to an already large backlog.

COVID-19 has also shown that Health Canada can move quickly to regulate new drugs – it approved the first vaccines in two months. Additionally, the federal government has recognized that the PMPRB’s review of COVID-19 vaccines and drugs would be an issue and has exempted them. It also allowed their use subject only to an interim order from the Minister of Health to reduce red tape. The changes needed to deliver COVID-19 vaccines and medications to Canadians are compelling evidence that the status quo is not working for timely patient access to medications that produce better outcomes.

To address the need for change, the Resilient Healthcare Coalition was created with a mission to create a faster, more agile healthcare system that is better able to absorb shocks like COVID-19. In a letter to Prime Minister Trudeau, the Coalition recommends that access to innovative health technologies, such as vaccines, drugs and diagnostics, be expanded and accelerated with an interim approval system for breakthrough drugs for diseases serious and potentially fatal. A similar campaign is underway in the United States demanding access within six months of marketing approval for a new drug for a serious or life-threatening condition.

How could we speed up access to drugs in Canada? Regulatory drug reviews conducted in the United States and Europe, where submissions are generally made earlier than in Canada, should be accepted by Health Canada through a mutual recognition process to allow for marketing approval faster. CADTH and pCPA must be bypassed for provisionally approved drugs, patient registries must be established (this will require improvements in health care data and access), and registration will be a condition of access to provisionally approved drugs.

We also need to develop standards to assess whether provisional approval should be converted to full acceptance and what penalties should be instituted for proponents who fail to comply with monitoring requirements within a specified time frame. A transparent process for withdrawing drugs that do not meet the standard is also required, although it is necessary to ensure that patients who have achieved beneficial results can continue treatment.

A major issue will be who should pay for provisionally approved drugs and the necessary evaluation requirements. These costs should be shared between drug developers and federal, provincial and territorial governments. The developer’s share would be greater due to their responsibility for data collection, but governments have to bear some expenses, including the work needed to improve registration and access to data. Cost sharing would also demonstrate a willingness on the part of governments and manufacturers to develop collaborative relationships that have been sorely lacking in Canada for decades. Cost-sharing may not be popular with governments, but it should be obvious to all Canadians that we need to radically improve access to advances in therapy.

A recent opinion poll showed that most Canadians believe that “meaningful reforms” are needed to improve the delivery and accessibility of health services. However, almost 60% of those polled did not have confidence in the ability of our political leaders to seriously assess the performance of the health system during the pandemic and to make creative and far-sighted change. Canadians must encourage their politicians to think beyond the current election cycle and show real leadership. If the provincial and territorial premiers are successful in getting the federal government to increase its share of health spending from 22% to 35%, it is crucial that the money be used for visionary change, not to maintain the status quo.

The current policy of Canadian governments appears to be to delay access until patents expire and generic competitors become available. It does not help patients or encourage innovators. Canadians need faster access to innovative medicines for serious and life-threatening illnesses.

The attitude of governments towards the biopharmaceutical industry must shift from antipathy to collaboration with the crucial aim of benefiting patients. Instead of wasting taxpayers’ money continuing with lawsuits to try to impose the PMPRB’s revised regulations, the funds should be used to improve the health care system. The federal government must prioritize rapid access to lifesaving new medicines by removing the barriers currently in place and replacing them with a nimble, faster system. It’s time to change policy.

Nigel Rawson is an independent researcher and researcher affiliated with the Canadian Health Policy Institute. John Adams is co-founder and CEO of Canadian PKU and Allied Disorders Inc. and volunteer chair of the board of Best Medicines Coalition. The opinions expressed are those of the authors and do not necessarily represent those of the organizations with which they collaborate.

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