Patient assistance programs

Assistance programs are critical for helping patients access healthcare when it is unaffordable. This includes uninsured or underinsured patients in high-income countries. Even when patients have insurance coverage for drug prescriptions, they may be unable to afford to pay for certain medicines.

To help support such patients, the Novartis Patient Assistance Foundation Inc. (NPAF) provides medicines at no cost for individuals who are experiencing financial hardship and have limited or no prescription drug coverage in the US. In 2020, NPAF served more than 107 000 patients, making available more than 72 medicines from our portfolio. Over the past five years, NPAF has provided medicines at no charge to more than 363 500 patients.

In 2020, NPAF began providing access to new Novartis medicines launched the same year, including Tabrecta for metastatic non-small cell lung cancer, Kesimpta for multiple sclerosis, inclisiran for managing cholesterol, and Ziextenzo for use in patients who are receiving chemotherapy and are at risk of infection.

At the start of 2020, NPAF focused on improving the patient experience. For example, NPAF reduced the time it took to process enrollment applications, providing patients more timely access to medicines. NPAF then had to adapt to the impact of COVID-19 on employment, travel restrictions and medication access. Due to quarantine and travel restrictions, patients faced challenges in submitting documentation for NPAF eligibility. Additionally, healthcare professionals (HCPs), who collect patient NPAF enrollment documents, had restricted access to patients. Many patients lost their jobs as well as insurance coverage for drug prescriptions, and many expressed concerns about their ability to continue accessing Novartis medicines.

In response, NPAF took measures to reduce the additional burden on patients and HCPs, working to ensure continuity of treatment. We quickly modified our processes for gathering and reviewing documentation in order to account for circumstances created by COVID-19. We expanded timeframes for refills, shipped patients a greater supply of medicines, and provided additional flexibility in the renewal process.

We also expanded NPAF’s Institutional Patient Assistance Program (IPAP) to include ophthalmology and cardiovascular products. Moreover, we widened the IPAP network of collaborating safety net clinics, which provide healthcare services to indigent populations. IPAP clinics receive Novartis medications directly, enroll patients in the program, and assist them with administrative processing. This support allows patients to walk in and receive medicines almost immediately, filling a critical gap in the healthcare system. We worked to ensure these clinics maintained continuity of care during the pandemic.

In LMICs, Novartis Oncology Access (NOA) makes medicines in its portfolio available through equitable pricing models. NOA pursues a partnership-based approach, sharing the cost of its medicines with government healthcare systems, charities and other payers, or directly with patients without healthcare coverage who are unable to pay for the full cost of their medication. In 2020, more than 33 000 patients in seven countries benefited from the Novartis Oncology portfolio in multiple disease areas.

Managed access programs

Physicians sometimes seek access to medical products that are not yet approved or available in their country to treat patients with serious or life-threatening conditions. Novartis Managed Access Programs (MAPs) address this need by making certain investigational or unapproved treatments available to eligible patients. Novartis has been widely acknowledged as a leading company when it comes to providing patients with access to medicines though MAPs.

Since 2017, Novartis has collaborated with an external Independent Bioethics Advisory Committee (IBAC), which provides analysis and recommendations on our guidelines and policies for the ethical conduct of clinical research, and on selected ethical challenges that may arise in clinical trials, development programs, managed access programs and other areas across Novartis. The IBAC includes bioethicists, clinicians, healthcare practitioners, patient advocates and other domain knowledge experts as required. We are one of a few companies that consults with an independent bioethics committee.

Novartis monitors patient support via MAPs through the Novartis Managed Access Center of Excellence. In 2020, we receivedand reviewed 10 670 MAP requests from physicians and we approved 94% of those requests from 82 countries and across 64 compounds. An additional estimate of 4 500 patients were supported via requests from healthcare institutions and governments. More than 14 000 patients are currently on treatment via MAPs.

COVID-19 was the leading indication for which Novartis received managed access requests in 2020, followed by metastatic breast cancer. Novartis provided medicines for treating COVID-19-related conditions to more than 7 000 patients, responding to unsolicited requests from physicians, healthcare institutions and governments. The initial demand came from Italy, with approximately 900 requests for medicines received within the first few weeks of the outbreak in that country. In response to the demand and urgency of the situation, we instituted an expedited review process that shortened MAP approval times from five working days to three or four hours, with medicines shipped within 24 hours. Novartis approved nearly 100% of requests for medicines to treat COVID-19 patients.

In 2019, the US Food and Drug Administration approved Piqray (alpelisib), a targeted treatment for advanced breast cancer. As part of our access strategy, through a global MAP we made the medicine available to patients with no alternative or comparable treatment options in countries where the product was yet to be approved or unavailable. In 2020, we received over 1 550 requests from 40 countries, and we approved 89%. To date, we have provided the medicine to more than 2 040 patients through managed access.

Despite supply constraints, in 2020 we fulfilled our commitment to make Zolgensma (onasemnogene abeparvovec), our one-time gene therapy approved to treat pediatric patients with spinal muscular atrophy, available to up to 100 patients via a global MAP. In 2020, 100 patients received Zolgensma in more than 20 countries where the therapy is not yet approved by regulatory authorities.

Further, through a MAP we continued to make Lamprene (clofazimine) available to more than 1 900 patients in 2020, mainly for nontuberculous mycobacterial infection.