Efficacy

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Introduction

Efficacy — in legal practice — denotes the capacity of a law, regulatory approval, remedy, order or piece of evidence to achieve its intended legal, regulatory or practical purpose. In India efficacy operates as both a substantive and procedural touchstone: courts and regulators assess whether a statute, administrative action, patent claim, drug approval, or evidentiary instrument actually delivers the results it claims to deliver. For practitioners, appreciating how courts probe “efficacy” changes how you frame pleadings, marshal technical proof, and structure interlocutory relief.

Core Legal Framework

There is no single statutory definition of “efficacy” in Indian law. The concept is woven into multiple statutory regimes and doctrines:

  • Patents Act, 1970 — Section 3(d)
  • Key text (emphasis added): “The following are not inventions within the meaning of this Act, … (d) the mere discovery of a new form of a known substance which does not result in the enhancement of the known efficacy of that substance…” Section 3(d) makes “therapeutic efficacy” a substantive threshold for patentability of incremental pharmaceutical innovations.
  • Drugs and Cosmetics Act, 1940 and Rules thereunder; New Drugs and Clinical Trials Rules, 2019
  • Regulatory approvals for drugs are granted only after demonstrations of safety and efficacy to the satisfaction of the Central Drugs Standard Control Organization (CDSCO) and state authorities. Clinical trial data and post-marketing surveillance inform efficacy assessments.
  • Indian Evidence Act, 1872
  • Section 45 (opinion of persons skilled in science, art or trade) is routinely invoked when determining the efficacy of a drug, device or technical process; Section 101 (burden of proof) is relevant to who must establish efficacy in disputes.
  • Constitution of India — Articles 14, 19, 21
  • Judicial review of statutes and executive action frequently evaluates whether the measure is effective and proportionate to its aim; the “efficacy” of a law in achieving a legitimate state objective forms part of reasonableness and proportionality analysis (see judicial pronouncements below).
  • Arbitration and Civil Procedure frameworks
  • The practical enforceability (efficacy) of an award or decree — its executability— is tested under the Arbitration and Conciliation Act, 1996 and the Code of Civil Procedure, 1908 (execution provisions).

Practical Application and Nuances

How “efficacy” surfaces in daily practice varies by context. Below are common contexts and concrete litigation/regulatory strategies.

  1. Patents and “therapeutic efficacy”
  2. Where it matters: Patent prosecution, patent oppositions, revocation proceedings.
  3. What to prove:
    • For a patentee: evidence that the claimed new form or compound demonstrates enhanced therapeutic efficacy compared to the prior art (head-to-head clinical data, pharmacodynamic or pharmacokinetic evidence, statistically significant outcomes).
    • For an opponent/generic: comparative clinical studies, meta-analyses, or absence of credible clinical benefit; demonstrate that alleged improvements are cosmetic (stability, shelf-life, process efficiency) and not therapeutic benefit.

  4. Practical example: In a revocation petition under Section 64 of the Patents Act, an opponent will seek production of clinical trial reports, regulatory filings, and expert affidavits. Cross-examination of the patentee’s experts on study design, endpoints and statistical power is decisive.

  5. Drug/device regulatory efficacy

  6. Where it matters: Challenges to approvals, interim injunctions restraining sale, post-marketing surveillance.
  7. What to raise:
    • For regulator approval challenges: demand underlying trial protocols, GCP compliance certificates, CDSCO assessment reports, adverse event reports, batch consistency data.
    • For interlocutory relief: courts balance public interest in access to medicine against prima facie concerns about efficacy/ safety. Production of raw trial data or appointment of technical committees is frequently sought.

  8. Practical example: When litigating against an approval, practitioners should plead specific defects (protocol deviations, inadequate endpoints, surrogate endpoints without proven correlation) rather than general assertions of “inefficacy”.

  9. Efficacy of legal remedies and orders

  10. Where it matters: Writ petitions, injunctions, execution proceedings.
  11. What to argue:
    • A writ petitioner must show that available statutory remedies are inadequate or ineffective (lack of efficacy); conversely, respondents will argue adequacy of alternative remedies.
    • In interim relief, courts ask whether the injunction will be efficacious (will it cure the harm without causing disproportionate public harm).

  12. Practical example: In a public interest litigation against a regulatory decision, demonstrate that the statutory appeal route is ill-suited to produce timely relief (delays, lack of technical review) so that writ jurisdiction is necessary.

  13. Efficacy of evidence

  14. Where it matters: Criminal prosecutions, civil suits involving technical subject-matter.
  15. What to present:
    • Expert reports under Section 45 Evidence Act, test reports, lab certificates, chain-of-custody, clinical endpoints, pharmacokinetic analyses, real-world evidence.
    • Keep in mind burden of proof rules: prosecution must prove guilt beyond reasonable doubt; claimant must discharge preponderance in civil matters.

  16. Practical example: When contesting a clinical claim, highlight methodological flaws (lack of randomisation, small sample, non-blinded outcomes) to undermine efficacy assertions.

  17. Administrative law and legislative efficacy

  18. Where it matters: Constitutional challenges to statutes and delegated legislation.
  19. What the court examines:
    • Whether the impugned measure is rationally connected to the legitimate object, whether it is arbitrary, and whether it is effective in achieving the legislative purpose. Principles of proportionality now form part of Indian jurisprudence (Maneka Gandhi and subsequent cases).
  20. Practical example: In challenge to a subsidy scheme, demonstrate either that the scheme is ineffective in achieving its stated social objective (empirical data) or conversely defend the scheme by producing implementation data showing outcomes.

Landmark Judgments

  • Novartis AG v. Union of India & Ors., (2013) 6 SCC 1 (Gleevec)
  • Principle: The Supreme Court interpreted Section 3(d) of the Patents Act to mean that “efficacy” in the pharmaceutical context must be understood as “therapeutic efficacy.” Mere improvements in physicochemical properties do not satisfy Section 3(d). Novartis’ claim for patent protection over an improved crystalline form of imatinib failed because it did not demonstrate enhanced therapeutic efficacy over the known substance.

  • Practical import: For patent drafters and litigators, therapeutic endpoints and comparative clinical evidence are indispensable where Section 3(d) is invoked.

  • Maneka Gandhi v. Union of India, AIR 1978 SC 597 (1978) (1 SCC 248)

  • Principle: Judicial review under Articles 14, 19, 21 requires that laws and executive action not be arbitrary; they must be just, fair and reasonable. Although not using the literal word “efficacy,” the Court endorsed scrutiny of whether measures achieve their legitimate aims in a proportionate manner.

  • Practical import: When litigating against a law or administrative action, frame arguments to show absence of rational connection or lack of effectiveness in attaining legitimate objectives. Empirical and expert evidence can be used to demonstrate ineffectiveness.

  • A.K. Kraipak v. Union of India, (1969) 2 SCC 262

  • Principle: Administrative decision-making must be free from arbitrariness and must follow fair procedure; effectiveness and reasonableness of delegated decisions are subject to judicial scrutiny.
  • Practical import: In challenges to executive decisions that claim to be efficacy-driven (e.g., public health orders), emphasize procedural defects and lack of reasoned assessment of efficacy.

Strategic Considerations for Practitioners

Practical strategies to marshal or attack “efficacy” issues:

  1. Build a multidisciplinary record
  2. Engage domain experts early (clinicians, pharmacologists, statisticians, regulatory specialists).

  3. Obtain and preserve primary data — trial protocols, CRFs (case report forms), raw results, regulatory correspondence. Courts place high value on primary datasets.

  4. Focus pleadings on specific, testable defects

  5. Avoid abstract assertions that a drug or measure is “ineffective.” Pinpoint protocol deviations, endpoints not clinically meaningful, absence of controls, bad comparator choice, lack of power, or selective reporting.

  6. Use expert affidavits strategically

  7. Experts under Section 45 can educate the court. Ensure expert affidavits explain methodology in non-technical yet precise terms, identify benchmarks (e.g., RCT standards, CONSORT compliance) and set out clear conclusions on efficacy.

  8. Seek technical fact-finding mechanisms

  9. Courts frequently appoint committees or monitoring boards. Promptly propose neutral, credible experts and suitable terms of reference; this can shape the fact-finding process rather than cede it to the opponent.

  10. Prepare for confidentiality and discovery issues

  11. Regulatory dossiers often contain confidential commercial information. Use confidentiality arrangements and specific judicial orders for inspection or in-camera review instead of wholesale public filings.

  12. Anticipate public interest balancing

  13. Where public health or access to medicine is involved, courts will weigh systemic efficacy against private exclusivity. For patentees seeking injunctive relief, prepare disproportionate-harm and public-interest arguments; for defendants, emphasize access and affordability concerns.

  14. Avoid overreliance on foreign approvals

  15. Foreign regulatory approvals are persuasive but not determinative in Indian adjudication. They can support efficacy assertions but must be backed by locally relevant data and compliance narratives.

Common pitfalls
– Treating “efficacy” as merely a label rather than an evidentiary standard.
– Failing to secure the underlying data before filing (risking inability to compel regulatory authorities later).
– Overloading courts with raw technical jargon; failing to translate technical proof into judicially usable metrics (effect size, confidence intervals, clinical significance).
– Ignoring procedural bars (limitation, alternative remedy) when claiming writ jurisdiction on efficacy grounds.

Checklist — Evidence to Prove (or Disprove) Efficacy
– Trial protocols and ethics approvals
– Randomisation and blinding records
– Primary outcome measures and statistical analysis plans
– Raw case report forms and datasets
– Adverse events and pharmacovigilance reports
– Comparative effectiveness studies or meta-analyses
– Regulatory evaluation reports and expert committee minutes
– Manufacturing consistency and bioequivalence studies (where relevant)

Conclusion

“Efficacy” in Indian legal practice is never a purely academic concept: it is a fact-driven, context-specific criterion that courts and regulators interrogate with technical rigour. Whether opposing a patent, challenging a drug approval, litigating the adequacy of a statutory remedy, or testing the weight of technical evidence, success depends on concrete, admissible proof and persuasive translation of technical findings into legal standards. Practitioners must marry legal strategy with robust scientific methodology, seek early expert input, preserve primary data and frame pleadings that invite focused judicial fact-finding rather than broad exhortations about “inefficacy.”