Alnylam Issues 2nd Annual Patient Access Philosophy Report Highlighting Broad Access to the Company’s Approved Therapies

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today published two new reports: the second annual update on its Patient Access Philosophy and its first-ever Rare Disease Trend Report. Together, the reports illuminate both the strategies by which the company has been able to maximize access for its approved medicines around the world, and the barriers associated with coverage for rare disease therapies.

The Patient Access Philosophy Report

The 2020 Patient Access Philosophy Report reflects Alnylam’s progress advancing its stated 2017 Patient Access Philosophy. The 2020 report highlights the Company’s efforts to support patients in gaining access to needed medicines, both before and during the COVID-19 pandemic. It also provides updates on initiatives to deliver strong value to payers in the face of increasing barriers to access for rare disease medicines.

Highlights include:

Rapid, affordable access to treatment: For people experiencing debilitating diseases like hATTR amyloidosis, acute hepatic porphyria or primary hyperoxaluria type 1, waiting for a therapy that could change their lives is unacceptable. In collaboration with payers around the world, Alnylam has:

  • Realized greater than 98% coverage for ONPATTRO and nearly 94% for GIVLAARI across U.S. commercial and government insurers, including Medicare and Medicaid.
  • Ensured the majority of commercially insured U.S. patients enrolled in its patient services program faced little to no out of pocket costs:
    • 75% of enrolled ONPATTRO patients and 87% of enrolled GIVLAARI patients have zero cost sharing for the medicine.
  • Achieved reimbursed access to ONPATTRO and GIVLAARI an average of 11 months faster than other orphan drugs in major European countries.
  • Established availability of ONPATTRO in more than 20 countries through direct reimbursement or expanded access.

Advocating for covered use of home administration: Travel to an infusion center or hospital to receive treatment can be challenging for some patients, especially during the COVID-19 pandemic. To support patients, Alnylam has advocated for insurers to cover home infusion, and today:

  • 20% of U.S. commercial patients on ONPATTRO and GIVLAARI are receiving treatment in the home.
  • Seven countries in the EU and Canada offer home administration with reimbursement for our medicines.

Executed nearly 30 value-based agreements (VBAs) with U.S. payers (ONPATTRO - 19; GIVLAARI - 10): Alnylam has pro-actively engaged with payers on VBAs to link reimbursement of its medicines with product performance and value delivered. VBAs represent a commitment to work with payers on business innovations that address the needs of patients. The company has also pioneered additional components for its VBA framework, including:

  • Prevalence-Based Adjustment (PBA) for its ultra-rare orphan disease products, where the company has committed to lower the price of its medicine if the number of patients identified within a plan population exceeds the expected disease prevalence.
  • Patient-Need Adjustment (PNA), where the company provides payers with greater budget certainty for medicines administered across a broad range of patient age groups. The company is applying this innovative PNA component in VBAs for OXLUMO.

“Three years ago, we set out to achieve what mattered to us: getting our RNAi therapeutics to those who can benefit from them and do it in a way that delivers value,” said John Maraganore, PhD, Chief Executive Officer of Alnylam. “As an increasing number of medicines become available to treat people with rare genetic diseases, insurers face challenges paying for them. We’ve engaged in innovative agreements that tie payments to health outcomes, or provide rebates when plans encounter more patients or higher drug usage than expected. Our healthcare system needs more pragmatic approaches such as these to ensure patients obtain access to needed therapies and that real value is delivered for the cost. We are very proud of our progress, and plan to continue to innovate and collaborate to ensure that patients have access to our medicines.”

Alnylam Rare Disease Trend Report

Critical to Alnylam’s strong market access results is a desire to understand the priorities of payers. The company is issuing its first U.S. Rare Disease Trend Report, resulting from interviews with 30 payer and plan decision-makers about barriers to rare disease access over a five-year horizon. The findings reveal increasing concern about the sustainability of expenditures on rare disease medicines and potential strategies that may be implemented to manage costs. As a leader in the advancement of innovative medicines for rare diseases, the company plans to conduct this survey, and issue an updated version of the report, annually.

Key findings:

  • Managing rare disease drug utilization. Payers express concern about the increased use and number of rare disease therapies and the associated rising costs. Current focus areas for therapeutic utilization management include central nervous system and respiratory medicines. Future areas of management may include digestive and bleeding disorder therapies.
  • Healthcare economic evidence: Payers believe metrics beyond clinical efficacy, such as healthcare economic evidence, must play an increasing role in how we value medicines. However, they note there is currently a dearth of this type of evidence, particularly for emerging therapies.
  • Risk-sharing: Approximately 50% of payers engage in innovative payment models for rare disease medicines today, but this is limited largely to oncology. Payers express interest in pursuing more outcomes-based contracts, but they cite operational obstacles such as administrative burden from data collection, data privacy concerns, interoperability of electronic health records and clarity on outcomes to track.

“This is a forward-looking report about how our healthcare system must continue to evolve and consider the mid-and long-term value of rare disease therapies for the benefit of patients, families and communities,” commented Shirley Bachman, Vice President, US Market Access at Alnylam. “Through open dialogue and collaboration with payers, we endeavor to find contracting solutions that mitigate risk and offer greater cost predictability. This helps ensure that payers can plan effectively and have greater confidence in covering our therapies for patients who need them.”

To read more about Alnylam’s global market access progress, download the full Patient Access Philosophy Report here. For those interested in reading more insights from payers on delivering access to rare and ultra-rare disease medicines, the inaugural Rare Disease Trend Report can be found here.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit and engage with us on Twitter at @Alnylam or on LinkedIn.

Alnylam Forward Looking Statements

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam’s views with respect to its progress advancing its stated 2017 Patient Access Philosophy, its efforts to support patients in gaining access to needed medicines, both before and during the COVID-19 pandemic, and initiatives to deliver strong value to payers in the face of increasing barriers to access for rare disease medicines, the Company’s plans to conduct a Rare Disease Trend survey, and issue an updated version of its Rare Disease Trend Report, annually, and expectations regarding the advancement of its “Alnylam 2020” guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylam’s business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam’s ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its other marketed products; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO, GIVLAARI and OXLUMO; progress in continuing to establish an ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO, GIVLAARI and OXLUMO, and achieving net product revenues for ONPATTRO within its revised expected range during 2020; Alnylam’s ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam’s ability to establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures; as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.


Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)

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