Embedding Patient-Centricity by Collaborating with Patients to Transform the Rare Disease Ecosystem.

What is patient-centricity? In some contexts, it has been associated with targeting therapies based on biomarkers or enabling healthcare access. There has been a surge in patient-centricity publications, and in many cases for the biopharmaceutical industry, patient engagement is used to endorse pre-held assumptions at a specific moment in time. Rarely is patient engagement used to drive business decisions. Here we describe an innovative partnership between Alexion, AstraZeneca Rare Disease and patients that allowed a deeper understanding of the biopharmaceutical stakeholder ecosystem and an empathic understanding of each patient's and caregiver's lived experience. Alexion's decision to build patient-centricity frameworks resulted in the formation of two unique organisation design platforms: STAR (Solutions To Accelerate Results for patients) and LEAP (Learn, Evolve, Activate and deliver for Patients) Immersive Simulations. These interconnected programmes required cultural, global, and organisational shifts. STAR generates global patient insights that are embedded in drug candidate and product strategies while helping to establish enterprise foundational alignment and external stakeholder engagement plans. LEAP Immersive Simulations produce detailed country-level patient and stakeholder insights that contribute to an empathetic understanding of each patient's lived experience, support country medicine launches and provide ideas to have a positive impact along the patient journey. Combined, they deliver integrated, cross-functional insights, patient-centric decision making, an aligned patient journey, and 360° stakeholder activation. Throughout these processes, the patient is empowered to dictate their needs and validate the proposed solutions. This is not a patient engagement survey. This is a partnership where the patient co-authors strategies and solutions.

Safety and efficacy of elbasvir/grazoprevir in patients infected with hepatitis C virus genotype 4 in Qassim region of Saudi Arabia.

Objective: Untreated hepatitis C virus (HCV) infection can lead to cirrhosis, hepatocellular cancer, and even death and also increases liver diseases. The elbasvir/grazoprevir (EBR-GZR) treatment regimen when given in HCV genotype (GT) 1 and GT4 infection for 8 or 12 weeks showed a high sustained virolgical response (SVR) rates in different populations. This study was assessed the effectiveness as well as safety of EBR-GZR in 12 week treatment regimen in HCV GT4-infected treatment-naïve Saudi patients. Methods: This study conducted from June 2017 and December 2020 on Saudi HCV patients infected with GT4. Treatment protocol was given for 12 weeks in treatment-naive HCV GT4 infected cirrhotic and non-cirrhotic participants which were later followed for a total of 24 weeks for safety and efficacy of EBR-GZR. Results: We analyzed data of 54 participants with HCV GT 4 infection. Mean age was (53.46 ± 14.94) Treatment regimen was given to 14 cirrhotic (F4) and 40 non-cirrhotic (F0-F3). SVR was seen in 98.1% of participants with tolerable side effects and improved model for end stage liver disease (MELD) scores as fall in percentage seen from 18.5% to 14.8% in participants with MELD > 10. Conclusion: This retrospective study confirms that EBR-GZR for 12 weeks is a safe and effective treatment regimen in HCV GT4 patients in studied Saudi population. Treatment completion was followed by high SVR12 rates with improvement in prognostic markers of liver disease in participants with compensated cirrhosis. In short, the EBR-GZR combination showed efficacy in achieving the SVR12 in Child-Pugh B cirrhotic and non-cirrhotic population with a favorable safety profile.

Susceptibility profile of methicillin-resistant Staphylococcus aureus to linezolid in clinical isolates.

OBJECTIVE: To determine the resistance and sensitivity pattern of methicillin-resistant Staphylococcus aureus (MRSA) isolates to linezolid (LZD) along with its prevalence in a tertiary care hospital of Karachi, Pakistan. MATERIALS AND METHODS: A cross-sectional study was carried out. This study lasted for about 1 year. Prevalence and sensitivity of LZD, vancomycin, and oxacillin was tested against isolates of MRSA. RESULTS: Out of total 369 specimens 165 were found to be MRSA making the prevalence in our study 44.7%. All of the isolates which were tested positive for MRSA were susceptible to LZD and no resistance was noted when compared with previous studies performed in Europe and USA. CONCLUSION: Stringent implementation of infection control measures along with screening for resistance in patients on prolonged LZD therapy or who previously went under LZD therapy should be performed, coupled with judicious usage of the aforementioned antibiotic should be undertaken, as sufficient data is not available at this point for the clinical spectrum of LZD resistant S. aureus, antimicrobial resistance.

Fatal primary meningoencephalitis caused by Naegleria fowleri.

Naegleria fowleri is a free living parasite which habitats in fresh water reservoirs. It causes a fatal nervous system infection known as primary amoebic meningoencephalitis by invading through cribriform plate of nose and gaining entry into brain. We report a case of primary amoebic meningoencephalitis caused by Naegleria fowleri in Karachi, Pakistan, in a 42 years old male poultry farm worker having no history of swimming. Clinical course was fulminant and death occurred within one week of hospital admission. Naegleria fowleri was detected by wet mount technique in the sample of cerebrospinal fluid collected by lumbar puncture of patient. This is a serious problem and requires immediate steps to prevent general population to get affected by this lethal neurological infection.