A new paper published in The Journal of Haemophilia Practice advances the concept of shared decision making (SDM) between the hemophilia patient and their healthcare provider (HCP).
As a relatively new approach to care, SDM sheds the one-size-fits-all method associated with a more paternalistic model whereby patients and caregivers – the latter when the patient is a child or otherwise not able to make decisions – function in a mostly passive role, deferring to their providers on decisions about their care. Instead, SDM embraces the patient-as-partner style, which cultivates a truly reciprocal relationship.
This approach draws on both a patient’s lived experience and knowledge, and an HCP’s clinical expertise. It eschews what the authors describe as a “transactional interaction” for a more equitable and collegial HCP-patient dynamic in which both parties arrive at informed care decisions only after open, two-way discussions about patient/caregiver (P/C) goals, values, and desires.
While certain situations, such as a medical emergency scenario, may compel an HCP to make an immediate autonomous decision, there are several instances that represent excellent opportunities to initiate SDM; these include a new diagnosis, a non-life-threatening condition, and ongoing care for a chronic condition such as hemophilia.
Integral to the SDM process is a stepwise approach that starts with a planning phase, initiated after the P/C is presented with a new therapeutic/management option, or some other change in care that will necessitate decisions. Here is where the new dynamic is established, in which the P/C is introduced to the concept of choice and to the idea of being equal partners in their care.
The next phase is where the HCP engages the P/C about their values, preferences, and treatment goals. They are also presented with all the relevant management options, including risks, benefits, and potential alternatives. HCPs are encouraged to provide additional information and resources during this period to help inform the process.
The third step is the decision point, where the treatment choice is arrived at only after the HCP assures the P/C that there will be an opportunity to review the new treatment plan before it begins and that modifying, or even withdrawing from, the plan will always be an option.
There exist several established SDM models, including SHARE, which was developed by the U.S. Agency for Healthcare Research and Quality. The five-step model includes 1) Seeking the patient’s participation 2) Helping the patient explore and compare management options 3) Assessing the patient’s values and preferences 4) Reaching a decision with the patient, and 5) Evaluating the patient’s decision.
Whichever the chosen model, SDM works best when the flow of information and ideas between the HCP and P/C is transparent and untainted by overt or unconscious bias.
“Overt bias may be introduced by the practitioner who is more familiar with one treatment versus another. On the other hand, the patient may have a bias towards a therapy that is familiar to them and be reluctant to embrace a treatment that presents a new mechanism of action, for example. In both cases, education is vital. It is essential that the practitioner learns about and presents all options to the patient and family in a fair and balanced manner,” explained the authors. “Likewise, patients must be prepared to embrace new information with an open and thoughtful approach. Unconscious bias on the part of the practitioner can lead to false assumptions and negative outcomes for patients.
Because unconscious bias is inherently more difficult to exclude from interpersonal interactions, the authors encourage HCPs to proactively acquire the techniques and skills to temper their influence. Patient characteristics, including those of minority groups, should be given due consideration when employing SDM to develop new treatment plans. These considerations include race, ethnicity, culture, education, and knowledge base, plus potential patient biases that could inform decisions regarding care. Assumptions based on stereotypes, which may surface during this process, should be avoided.
Hemophilia care in the 21st century is fertile ground for growing the SDM model, as the availability of existing and emerging therapies offer ample opportunities for true HCP-P/C partnerships. Recent innovations in hemophilia therapies, from standard factor replacement products, to extended half-life products, to novel subcutaneous therapies, while they have undoubtedly improved patient outcomes, they also represent challenges. An expanded catalog of therapeutic choices, changes in treatment regimens, new modes of product administration, shifting measures of pharmacokinetics, compel both parties to not only cooperate more closely together but also learn together.
That said, the learning curve for both parties will only continue to steepen as novel pipeline therapies move towards possible authorization, including a pair of investigational subcutaneous, prophylactic therapies for hemophilia A and B, with and without inhibitors. The first utilizes small interfering RNA (siRNA) technology to target antithrombin (AT), a liver-generated clotting protein that plays a key role in the regulation of blood clots. siRNA molecules are segments of RNA that block or “silence” the activity of certain genes through RNA interference. It works by silencing the gene responsible for AT, which inhibits the protein’s anticoagulant function, which in turn increases coagulation in hemophilia patients. The second is a laboratory-engineered monoclonal antibody that initiates a series of chemical and molecular reactions that generates enough of activated factor X, in vivo, to restore proper clotting. Both technologies represent pathways to hemostasis restoration that forgo the need for regular FVIII or FIX replacement therapy.
Lastly, gene therapy as a one-time, paradigm-shifting treatment represents the closest thing to a curative therapy, if not an actual cure. While adeno-associated virus (AAV)-based gene therapies stand to have the most far-reaching impact for the person with hemophilia (PWH), they also elicit the most sweeping, open-ended series of questions relevant to long term efficacy, safety, and cost, among other topics. In short, the introduction of gene therapy into SDM discussion presents further opportunities for engagement, albeit with a unique set of challenges.
“Since gene therapy may present a significantly different treatment choice vs. a chronic therapy, a new set of risk and benefit considerations would need to be evaluated during the treatment decision process. For PWH accustomed to clotting factor-based treatments, gene therapy represents a major shift that would require both practitioners and PWH to adopt a new way of thinking about treatment beyond the previous known benefits and risks of clotting factor therapy. Both parties will need to think differently about what may be possible in order to focus on what matters most to PWH and their families.”
The SDM approach is not without its limitations as a lack of motivation on the part of either participant can cause the process to break down. Further, HCPs need to proceed carefully through the process to ensure that patients are not left overwhelmed or withdraw into a passive or disinterested stance, which is antithetical to effective SDM.
“SDM can be a powerful approach in the era of multiple therapeutic options for PWH. The focus of SDM is on understanding the patient’s treatment goals, and on assisting both the patient and practitioner to jointly arrive at a decision that takes into consideration the risks, benefits and alternatives to the proposed intervention on a case-by-case basis,” conclude the authors. “Considering the recent innovations in haemophilia and choices of approved therapies, SDM is a useful tool in determining treatments approaches, and may also set the stage for advocacy for access to novel therapies such as gene therapy.”
Valentino LA, Blanchette V, Negrier C, O’Mahoney B, Bias V, Sannie, Skinner MW. Personalising haemophilia management with shared decision making. J Haem Pract 2021; 8(1): 66-76..
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