How not to “Launch a rare disease drug ” or any other drug?
Today, I am anguished. I read an article from McKinsey, titled:
How to successfully launch a rare disease drug in a patient-centric world
The article is replete with grammatical & ‘dramatical’ mistakes; starting in the first line itself. I shall not focus on any of them; they are only “literal’.
The article is also replete with apathy, ignorance, exploitation of mental & physical condition of patients, their families, their physicians & groups. The article reeks of profiteering, crony capitalism and “n0-skin in the game” as Nassim Nicholas Taleb always points out.
The passively-voiced, prescriptively-toned, long-winded article is rubbish. I will not be courteous or polite in my criticism because I have never read a more “mean” article than this, ever.
The first of the 4 strategic pillars of “launch” that the article identifies is:
Our experience suggests that companies that launch rare disease treatments successfully excel in four areas.They show great commitment to the rare disease community, whose support is key.
The phrase “ They show” prescribes to develop a disingenuos relationship with the rare disease community. The prescription is “to show” rather than “to be”. This is a shameful approach to a very noble work of working dedicatedly & closely working with rare disease community. “Rare disease community” do not exist de-novo. They need to be developed by collating, by connecting individual, geographically distributed, helpless parents whose children affected by rare diseases are dying each day slowly for want of treatment and care. “Showing” committment is cheating with their emotions for the act of profiteering.
They continue:
Launch teams need to invest significant time with patients and caregivers early in the launch process to understand their journey and the barriers they might face in accessing treatment. This in itself can be a sign of commitment, but more can be done.
Again, prescription is to show “a sign of commitment” and not make a genuine commitment to the understanding of the mental and physical condition of patients. I am feeling ashamed at the “apathy’ in this statement. The words ‘launch teams’, ‘invest’, ‘launch process’ seek a clarification from the authors. The world has always been human-centric and these corporate jargon has no place in human relations. For example how about a company representative introducing as “ Hi! I am from the firm, a member of ‘launch team’ who is here to ‘invest’ time with you to elicit a ‘sign of commitment’ from you?” This jargon is deplorable and so is the basis of this prescription.
They further continue in same breath
For example, setting up a social media platform can prove valuable to patients who are geographically scattered, enabling them to share experiences and creating a sense of solidarity. Such a site is also a channel for dispersing information about an upcoming launch.
Both these prescriptions are unethical, non-compliant and plain illegal. As a representative and partner from the Pharmaceutical company I should and cannot set up a social media platform for patient groups. I can provide them guidance on doing so based on my general social know how and importance of digital channels in today’s world. In practice, mostly informal “WhatsApp” /chat groups via common physicians exist where entry is limited by awareness and not a lack of “sense of solidarity”. Further, using a rare disease community social media channel to “disperse information” about an “unapproved” product is unethical and non-compliant. Any information about upcoming product before launch is shared only on basis of solicitation of a physician/scientist and never ever on a social media channel.
Advocacy groups, on which patients often rely as their primary source of clinical information, are equally important partners. In the absence of market research, they can be engaged to help in the development of patient databases and surveys of patients’ needs, the design of clinical trials, and in finding and enrolling patients for those trials — a task that can be extremely challenging even if only a few dozen patients are required. In return, companies can show their commitment to advocacy groups by providing logistical support for fundraising and awareness-raising activities, or by financing studies that go beyond drug approval requirements — Phase IIIb and IV studies, ISTs, and outcome registries.
The entire paragraph is so grossly unethical, non-compliant and disingenous that I blinked twice after reading it. Advocacy groups cannot be used to develop patient database for the sheer point of respect of patient privacy, safety and pharma ethics. Advocacy groups do not have the expertise to provide inputs in clinical trial design. And “In return, companies can show their committment”; I rolled by eyes twice over this phrase. They seem to suggest to make the the pharma — Advocacy group relation a commercial/vendor relationship. This is beyond my reason and is nearing madness. I believe that the authors and editors were smoking something really different to come up with this sugegstion.
TAEs too are valuable, especially in building awareness of a disease. For many physicians, dedicating time and attention to a specific rare disease is a major career choice, and companies can back them, for instance, by involving them as investigators in clinical trials.
TAE’s are the real champions of rare disease patients. They are senior physicians who are in top triangle of Maslow’s hierachy of needs. They are well respected physicians who seek self-actualization and want to bring transformative changes in their patients whom they are seeing for years and providing supportive therapy while waiting for pharma firms to create treatments. I offer my highest salutes to such physicians who work hard for rare disease patients. They are where they are from YALE to Standford to AIIMS to Taiwan General hospital to Argentina only because of themself and not by “companies backing them up”.
Patient group identification
Identifying the largest possible patient group is a lengthy task
The entire paragraph is factually wrong. Wrong disease is referenced, test distribution is misquoted and sales force meeting physician for unapproved drugs is unethical but most of all the intentions of “identifying patients” are unethical and non-compliant. The patient is always managed by the treating physician who can approach company for scientific literature for specific cases, diagnostic test support and compassionate drug usage. Only selected team members ever interact with patient only after a rigrously worded contract and information release form.
Patient access
The focus of the section is for activities to be carried out once the product is approved for use in the country and the access pathways have been established. The author is thoroughly confused between “Experimental access” “Named patient access” and “humanitarian access” with no idea about how they help patients/countries who are at various stages of access and reimbursement pathways. The patient family and advocacy group fight for themselves and we need to support, educate, motivate them to continue on the path to access. The movie ‘The Extraordinary Measures’ shows what a strong family support can do for the patients of rare diseases.
Patient and caregivers’ support
_ _ _ _ _ A company can provide these support services internally or externally through advocacy groups or other third-party providers, depending on cost considerations and regulatory constraints
How tragically the basis of providing patient and caregiver support is to be based on cost/regulator and not the patients’ needs itself. The patients have occupied the backseat in the entire article. Their needs and condition are secondary to launch strategies. The pitiful strategy prescribed in this article should not only be criticized, it should be actively resisted and panned.
I was lost beyond this point. The article continues to provide a tactical description of executing this strategy which is so wrong that I do not want to waste my time criticizing it. I am anguished at the approach, mentality, language and intentions described to pursue the launch of a rare disease drug. We don’t need to Launch drugs with this thought process. We need to bring a patient-centric approach to our entire thinking which is completely missing in this article.
Important Notes/declarations:
- I have in past worked for Sanofi Genzyme, a compnay that makes drugs for rare diseases
- These views are completely my own and do not reflect or suggest any views of my Sanofi Genzyme or any other past/current employers
