Personalised Treatment Delivery – aHUS Agenda Topic 4

aHUS patients are likely to have personal preferences for a treatment and as more options become available a personalised approach for how it is deliver is increasingly opened up.

 Is there an optimal way in which a complement inhibitor can be delivered to suit an individual’s need?
Our experts view is :

The delivery of a complement inhibitors, such as Eculizumab and Ravulizumab can be tailored to suit an individual’s needs in certain cases. Eculizumab or Ravulizumab are commonly administered via intravenous (IV) infusion, which requires either a visit to a healthcare facility or delivered at home. So some choices already exist. However, efforts have been made to develop more flexible and convenient delivery options. Here are some considerations for personalized delivery of complement inhibitors:

  1. Subcutaneous Administration: Some complement inhibitors have been developed for subcutaneous injection, which can be administered by the individual or a caregiver at home. This method offers more flexibility and reduces the need for frequent hospital visits.
  2. Dosing Schedule: The dosing schedule can be personalised adjusted to an individual’s specific needs, taking into account their response to treatment and any changes in their condition.
  3. Pediatric Patients: Considerations for dosing and administration may differ for pediatric patients, so treatment plans should be adjusted to meet the personalised needs of younger individuals.
  4. Patient Preferences: Patient preferences and lifestyles should be taken into account. Some individuals may prefer the convenience of at-home administration, while others may prefer regular clinic visits for treatment.
  5. Compliance and Adherence: Healthcare providers work with patients to ensure that they are adhering to their treatment regimen to achieve the best possible outcomes.

The personalized delivery of complement inhibitors is an important aspect of treatment for conditions like atypical hemolytic uremic syndrome (aHUS). The choice of administration method and dosing schedule should be made in consultation with healthcare professionals who can consider the individual’s needs and circumstances. It’s important for patients to have open and ongoing communication with their healthcare team to optimize their treatment plan.

There are also pill options in development to add to patient’s choice if clinical effectiveness is proven. So the answer to this agenda topic is “ Yes’ and there will is more choices on the way IF they prove to be at least as clinically effective , or not inferior as it is sometimes expressed. Then their marketing has to be approved by organisations like the FDA. If then approved, and it is by no means certain they would be, payers would have to agree to fund them so cost effectiveness would be important.

It would be interesting to have an idea about what patients’ collectively think about potential choices of “limabs, copans and repurposed lizumabs”. A “which and why” study by patients ( not pharma market researchers) for patients to provide understanding and empowerment for patients to talk with their HCPs about it.

This is one of a series of articles about the Global aHUS Patients Research Agenda which started at this link HERE.

Article No. 620

This Post Has 2 Comments

  1. Sukhbir Rangi

    Hi Len
    First of all, appreciate all your articles and helpful information
    But right now there is nothing on the market that can be given to a AHUS members that we can self administer .

    Unfortunately Alexian as dropped the plans to distribute sub cankerous Ultomoris

    Unless there is another drug out there, which I don’t believe there is, I know there is some in testing phases
    Example Crovalimab

    1. Len Woodward

      Thank you. Ravu and ecu is self administered at home by some patients with a fistular in the presence of a nurse. Ravu delivered by a subcutaneous mechanism was a potential treatment but it appears those plans have not come to fruition. So for now it is a case of waiting to firstly see the outcomes of trials of technologies like crovalimab and iptacopan. Then PNH trial results and FDA, EMA , Japan and UK market approvals will be indicative of what the future holds for aHUS patients.

Leave a Reply