Finding the balance

April 19, 2019

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The era of personalized medicine is arriving at a full gallop. In this brave new world, specialty medicines target different genetic profiles and patients take DNA tests to find out if they qualify for a treatment. An array of lab tests hones in on their immune profile, enabling scientists to tweak a drug so it speaks the same immune language. If all goes well, the patient gets better—much better.

How can we continue to support these sci-fi advances while keeping a lid on costs? How can we steer the innovation ship so it lands on fertile soil? Some ideas to consider:

Set innovation priorities: All innovation is not created equal. Some new treatments, while technologically impressive, may not have a benefit-to-cost ratio that justifies the investment. By the same token, some treatments have complicated protocols that can compromise patient adherence—and create massive who-pays-for-what confusion. We need to think beyond pure science and prioritize innovation that fits into the real world. How about focusing more on prevention? An HIV vaccine, for example, could end the epidemic once and for all and save incalculable health care costs.19

Connect pricing to value: The proposed new PMPRB framework will give consideration to value-based pricing, meaning that the cost-effectiveness of a treatment may factor into pricing determinations.18 In a similar vein, the Expensive Drugs for Rare Diseases (EDRD) working group is looking to incorporate real-world evidence into its evaluation of medications.20 Evidence not strong enough? The price comes down. That’s the idea, anyway. While value-based pricing comes with its own complications, it keeps industry accountable and oriented toward treatments that deliver the goods.

Leverage and evolve your PSPs: The patient support program (PSP) accompanying most specialty drugs is a valuable data source. PSPs facilitate the collection of data specific to a product and a disease state, thus having the potential to yield critical insights on bang for buck. PSPs also enable the collection of patient-reported outcomes (PROM) and other metrics that are increasingly valued as supports for payer decision-making.

Keep a finger on the pulse: Keep abreast of what’s happening and what’s about to happen in the specialty medicine ecosystem. Look for guidance on harnessing real-world evidence from the Canadian Association for Population Therapeutics.21 Explore opportunities afforded by collaborative research groups, such as the international partnership to explore the therapeutic potential of a new target for HIV cure and prevention, spearheaded by the NEOMed Institute in Montreal.22 The Centre for Commercialization of Regenerative Medicine’s (CCRM) new Centre for Cell and Vector Production in downtown Toronto, for its part, provides the specialized environment and expertise needed to develop cell therapies.23 Connect with thought leaders to zero in on the most salient clinical needs—and put them at the top of your R&D to-do list.


References

18.Martell A, Paperny AM. Exclusive: Facing crackdown in Canada, drugmakers offered billions in price cuts. Reuters, Feb. 6, 2019.Accessed March 2, 2-16 at https://ca.reuters.com/article/topNews/idCAKCN1PV0DH-OCATP

19.Pursuing a vaccine for HIV (podcast). Global Advocacy for HIV Prevention, May 2018. Accessed March 5, 2019 at https://www.avac.org/resource/pursuing-vaccine-hiv

20.Stakeholder Consultation: Supplemental Process for Complex/Specialized Drugs Background Document. EDRD communication. Accessed March 12, 2019 at http://www.raredisorders.ca/content/uploads/EDRD-supplemental-process-background_24Oct2018_Final.pdf

21.Taking Action on Real World Evidence: From Analysis to Impact. Canadian Association for Population Therapeutics (CAPT). Accessed March 12, 2019 at https://www.capt-actp.ca/?id=1

22.New Target for HIV Immune Therapy and Cure to be Explored by Trans-Atlantic Industry-Academia Collaboration. NeoMed communication, Feb. 20, 2019. New Accessed March 5, 2019 at http://neomed.ca/en/2019/02/20/new-target-hiv-immune-therapy-cure-explored-trans-atlantic-industry-academia-collaboration/

23.New facility for delivering cell and gene therapies is opening in Toronto in fall 2018. CCRM bulletin. Accessed March 7, 2019 at https://www.ccrm.ca/sites/default/files/CCVP%20Two-Page%20Article%20V10.pdf

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Wanted: More and better specialty data