The question of how to expedite patient access to emerging technologies and reduce costs has been on nearly everyone’s radar in 2017, including governments and the private sector.

Value frameworks to assess health technologies, more frequent pricing revisions, and the use of Real World Evidence (RWE) were among the most commonly discussed topics. In the US and Canada, approval and access to generic drugs was also high on the list.

Americas

In 2017, Canada took another step forward in its commitment to bringing down healthcare cost:

• After consultations with various stakeholders, the Canadian government proposed to amend drug pricing regulations that govern the Patented Medicine Prices Review Board (PMPRB), which protects consumers from excessive drug prices. The proposed amendments reduce the regulatory burden for generic drugmakers and equip the PMPRB with new tools to evaluate drug prices, including a much longer list of country comparators.
• The pan-Canadian Pharmaceutical Alliance (pCPA) moved towards establishing a policy framework for biosimilars that would enable more consistent price negotiations.

In the US, stakeholders focused their attention primarily on the implications of PDUFA VI, 21st Century Cures, the “Right to Try” Act, and the US generic drug program for patient access to the medicines they need. Other prominent topics were value frameworks for health technology assessment (HTA), the definition and application of RWE, and outcomes-based drug reimbursement agreements.

Key implications of PDUFA, 21st Century Cures, and the “Right to Try” Act:

• Both PDUFA VI and the 21st Century Cures Act provide pathways to expedited access to innovative treatments (Patient Access to Therapies and Information clause, Cures Act; Ensuring Sustained Success of Breakthrough Therapy Program clause, PDUFA VI).
• The “Right to Try” gives patients with terminal illnesses the right to access experimental treatments that have demonstrated a level of safety.

Important milestones in the US generic drug program include:

• The FDA’s Drug Competition Action Plan to increase competition and improve patient access to affordable drugs.
• The reauthorization of the Generic Drug User Fee Amendments (GDUFA), which will provide important funding to the FDA generic drug program, advancing generic drug approvals.

Catering to the need for continued discussions among the different stakeholders, DIA and the Boston Consulting Group (BCG) held several Market Access Roundtables to develop a common set of standards for future market access competencies.

The focus on value frameworks to assess health technologies has led to a surge in HTA frameworks by non-governmental actors like ICER (Institute for Clinical and Economic Review), NCCN (National Comprehensive Cancer Network), ASCO (American Society of Clinical Oncology), the Memorial Sloan Kettering Cancer Center, and the American Heart Association. There has also been an increased push for using RWE to inform the development and implementation of these frameworks.

Arguably one of the most remarkable outcomes-based agreements for treatments with hefty price-tags was the deal struck between Novartis and the Centers for Medicare and Medicaid Services (CMS), offering patients a money-back guarantee if the drug maker’s new, personalized cancer treatment is unsuccessful.

Asia

Some of the Asian markets echo challenges similar to those in Europe and in the US:

• Intense debates in Japan centered on HTA assessments and more frequent pricing revisions, all matters still awaiting final resolution at the end of the year. The country also uses a R&D cost-based model in line with WHO’s Fair Pricing Initiative that does not use any comparators to determine price.
• In China, the Ministry of Human Resources and Social Security (MoHRSS) has updated the China National Reimbursement Drug List (NRDL) as part of the country’s wide-ranging health reform, improving both availability and affordability of needed drugs, including those produced by foreign firms. The NRDL also includes a cost negotiation process for 44 nationally and internationally produced high-priced drugs.

Europe

As healthcare systems are increasingly socio-economically vulnerable across the EU, sustaining patient access to high quality care has become a high priority. To that end

• pharmaceutical companies can use scientific advice from HTA Bodies (HTABs) to generate the evidence needed by payers for decisions on treatment costs and reimbursement;
• the European Medicines Agency (EMA), HTA bodies, and European payer organizations have joined forces to address clinical research deficiencies and streamline pricing and reimbursement negotiations to boost timely and affordable access to new healthcare products; and
• ADAPT-SMART, a project by the Innovative Medicines Initiative (IMI) to provide early access to innovative drugs, continued to look into challenges and solutions to early access, including issues around harnessing RWD in Central and Eastern Europe.