All major players in the pharmaceutical industry are multi-national organizations. Patient organizations, on the other hand, rarely cross borders. Maybe it’s time they should. Certainly the pace of change in our industry is heading in that direction. The barriers to a global patient organization include disconnected national regulatory standards; language gaps; cultural biases; lack of institutional support and funding; and the way that disease is managed and treated by the practice professions. The language of science is standardized and to a large degree universal, while the language of being sick is spoken in many different tongues. A case in point is Germany, which like many other European countries has instituted sweeping austerity measures that affect the delivery of healthcare and access to medicines, in an attempt to bring down costs and ultimately, trim the fiscal deficit. Implicitly, the gauntlet has been laid down on patients, who need to make their voices heard, if they want access to new therapies.
“I believe that patients in Germany would want to have a drug that keeps them alive longer,” Simon Clowes, global product vice president, AstraZeneca, and leader of a cross-functional team for the acute coronary syndrome (ACS) drug Brilique, told PharmExec. No one should have to guess about whether patients want the drug.
In the case of Brilique (ticagrelor), AstraZeneca did what many other companies still won’t do; they conducted a head-to-head study against the standard of care, in this case, the blockbuster drug Plavix (clopidogrel), combined with aspirin. According to results from the company’s PLATO trials, Brilique (also combined with aspirin) came out on top in terms of a decrease in heart attack frequency and an increase in overall survival, at least for the Non ST-Elevation Myocardial Infarction and Unstable Angina (NSTEMI/UA) forms of ACS, which represents 72% of the ACS patient population in Germany, according to AstraZeneca.
It follows that ACS patients in Germany would indeed want to weigh in on the regulatory process, and engage with AstraZeneca as well. After all, it’s fundamentally a question of value and price, also known as a cost/benefit analysis. Manufacturers want high margins, and governments want a low price; patients want affordable access to drugs that work, with minimal side effects. In Germany, professional associations of specialist physicians are active in policy debates, but lack real clout in advancing the patient perspective against that of the all powerful sick fund payers. Contrast this to the US, where an ad hoc community-based patients group, Mended Hearts, succeeded in persuading the powerful American Heart Association to develop and then jointly publicize important new clinical guidelines that expand patient access to medicines with a proven preventive impact on cardiovascular death, stroke and other chronic heart conditions.
Speaking on the Germany process – Brilique has received a positive preliminary benefit assessment, but awaits a final assessment from the Federal Joint Commission (G-BA) – Clowes describes the preliminary stage as “a bit like NICE,” in that NICE has an open meeting where everyone, patient groups included, can make the case for or against reimbursement. NICE, incidentally, announced its final support for Brilique today, which means there is an obligation for local trusts to reimburse the drug through the NHS. Clowes is unsure about the extent to which patient organizations participate in the German process. In fact, he wasn’t sure if ACS patient orgs exist in Germany, or if AstraZeneca has been in touch with them. And yet, Germany is the largest pharmaceutical market in Europe. In a follow-up email, Clowes’ German colleagues wrote that in general, “selected patient organizations are involved in the Federal Joint Committee (G-BA), where they have the right of petition but are not allowed to vote in the committee. Patient organizations serve in a consultative capacity when it comes to the final decision of the added value during the assessment process.” Germany’s system is overtly legalistic, with a separate judicial process to decide key policy and regulatory questions on social welfare, including health – a process that largely ignores the patient in favor of the three “social partners” of government, business and labor. Despite a robust and outspoken physician community, patients are much less visible in shaping the the drug licensing and regulatory process than is the case in the US, the UK, Australia or Canada, for example.
This should change. Patient organizations have to look more to the regional level and seek out underrepresented populations across national borders to strengthen their influence. This is beginning to take place in Europe, under the sponsorship of the 27 nation European Union. It has been noted that “going global” carries an element of risk in that patient groups might lose their focus on the individual disease state or condition that is relevant to patients and veer off into policy discussions on which there is no consensus and could leave groups politically exposed. Nevertheless, considering the level of influence that regulators in Germany and the UK have over global pricing and reimbursement, and ultimately, access, it becomes crucial for patients to participate in the process as much as possible. It could mean the difference between life and death.
Patient engagement is often in the best interest of industry, but does overt encouragement to expand these organizations mesh with industry’s global strategy?
For an analysis of Germany’s AMNOG drug reimbursement system, which went into effect on January 1 of this year, and more on AstraZeneca’s adventures with Brilique see PharmExec’s forthcoming November issue.